[Senate Report 109-129]
[From the U.S. Government Publishing Office]



                                                       Calendar No. 156
109th Congress                                                   Report
                                 SENATE
 1st Session                                                    109-129

======================================================================



 
             STEM CELL THERAPEUTIC AND RESEARCH ACT OF 2005

                                _______
                                

                August 31, 2005.--Ordered to be printed

   Filed, under authority of the order of the Senate of July 29, 2005

                                _______
                                

Mr. Enzi, from the Committee on Health, Education, Labor, and Pensions, 
                        submitted the following

                              R E P O R T

                         [To accompany S. 1317]

    The Committee on Health, Education, Labor, and Pensions, to 
which was referred the bill (S. 1317) to provide for the 
collection and maintenance of cord blood units for the 
treatment of patients and research, and to amend the Public 
Health Service Act to authorize the Bone Marrow and Cord Blood 
Cell Transplantation Program to increase the number of 
transplants for recipients suitably matched to donors of bone 
marrow and cord blood, having considered the same, reports 
favorably thereon with an amendment in the nature of a 
substitute and recommends that the bill (as amended) do pass.

                                CONTENTS

                                                                   Page
  I. Purpose and Summary of the Bill..................................1
 II. Background and Need for Legislation..............................3
III. Legislative History and Committee Action.........................4
 IV. Explanation of Bill and Committee Views..........................5
  V. Cost Estimate...................................................10
 VI. Application of Law to the Legislative Branch....................12
VII. Regulatory Impact Statement.....................................12
VIII.Section-by-Section Analysis.....................................12

 IX. Changes in Existing Law.........................................14

                   I. Purpose and Summary of the Bill

    S. 1317, the Stem Cell Therapeutic and Research Act of 
2005, does the following:
           Reauthorizes and expands the National Bone 
        Marrow Donor Registry (NBMDR), renaming it the C.W. 
        Bill Young Cell Transplantation Program (Program) to 
        reflect its broadened scope of services, then providing 
        those services through a mechanism of contracts which 
        address bone marrow/peripheral blood stem cells and 
        cord blood separately;
           Requires the Secretary of Health and Human 
        Services (Secretary) to provide structure and funds for 
        the collection and maintenance of units of cord blood, 
        which are to be used for treatment of patients with 
        transplant-amenable diseases and for research; and
           Amends the Public Health Service Act (PHSA), 
        to increase the number of bone marrow and cord blood 
        transplants performed, funding this function 
        separately.
    This legislation builds upon the firm foundation laid for 
stem cell therapy and research over the last two decades by the 
work of the National Marrow Donor Program (NMDP) and cord blood 
banks with bone marrow/peripheral blood stem cells and cord 
blood. This pioneering work of the NBMDR, mandated and funded 
by Congress, was ably carried out by NMDP under the direction 
of the Health Resources and Services Administration. In 
particular, Congress recognizes that the benefits, to date, of 
these life-saving treatments and the future realization of 
their therapeutic potential, could not occur without the past 
efforts by the NMDP, cord blood banks and others to facilitate 
and coordinate bone marrow/peripheral blood and cord blood 
therapies.
    A principal effect of this legislation is to direct the 
Secretary to establish standards, including institutional 
accreditation criteria, for the collection, processing, storage 
and distribution of cord blood, as well as fund a substantial 
increase in the number of cord blood units banked (inventory 
goal = 150,000 new units), for use by unrelated recipients 
under the auspices of a national initiative, the Program. These 
changes should considerably expand and facilitate use of cord 
blood for therapy and research, meeting the needs of 80-90 
percent of patients who require stem cell transplantation as an 
optimal therapy for more than 70 diseases.
    A demonstration project is also authorized, which makes 
directed donation possible through qualified cord blood banks 
in those instances when need for cord blood transplantation can 
reasonably be anticipated for first-degree family members. In 
addition, the bill establishes parallel or joint administrative 
functions for all types of transplant material--bone marrow/
peripheral blood stem cells and cord blood--that should 
increase their use: donor recruitment, particularly amongst 
underrepresented populations; single point access to transplant 
material for providers; patient advocacy and case management 
activities; and an Advisory Council of medical experts and 
other stakeholders to offer recommendations to the Secretary on 
all aspects related to the Program. Finally, the bill mandates 
establishment of a comprehensive clinical outcomes database--
the Stem Cell Therapeutic Outcomes Database--for all stem cell 
therapeutics, enhancing the safety and efficacy of these 
treatments by providing access to all authorized healthcare 
providers and qualified researchers. This legislation 
intentionally places additional emphasis on cord blood 
therapies, predicating new initiatives on current programs; in 
doing so, however, it affirms the value of and continues full 
support for work done, to date, with all types of stem cell 
therapy: bone marrow/peripheral blood and cord blood.
    All stem cells have the potential to divide and multiply, 
so as to ``self-renew'' the population of stem cells, and also 
give rise to more specialized cells as seen in adult tissues 
through differentiation. All stem cells share these basic 
properties; however, what stratifies them is the range of cell 
types, tissues, and organs that they have the ability to 
create. Adult stem cells like the hematopoietic stem cells 
found in bone marrow/peripheral blood and cord blood can 
repopulate the wide array of blood cell lines--say in a patient 
in treatment for leukemia--but currently have limited potential 
to develop into non-blood cells. As such, their therapeutic 
potential may be significant for a circumscribed range of 
disorders, but is likely to be extremely limited for disorders 
outside this range.

              II. Background and Need for the Legislation

    Human bone marrow and peripheral blood stem cell 
transplantation are well-established application of stem cell 
therapy. Human cord blood transplantation represents a variant 
of this therapy, in which blood, taken from the placenta and 
umbilical cord after birth of the baby and following separation 
of the cord, is used as the transplant material rather than 
bone marrow. All forms of stem cell transplantation can be life 
saving. Stem cell transplantation utilizing cord blood as the 
source material adds a third treatment option to the 
therapeutic armamentarium; providers can now balance the unique 
advantages and disadvantages of bone marrow/peripheral blood 
stem cells and cord blood to choose the optimal source material 
for transplantation in their adult and child patients. Note 
that, with each variant of therapy, the stem cells used for 
transplantation are derived from biological material which 
completely lacks all capacity for human life. In addition, all 
types of source material--bone marrow, peripheral blood stem 
cells and cord blood--are of value to the ongoing research that 
broadens the clinical applications of stem cell therapy.
    The National Marrow Donor Program (NMDP), which has 
implemented the NBMDR, pioneered several of the features of 
this legislation, including the following: clinical registry of 
marrow donors, to facilitate timely identification of suitable 
transplant material (> 5.5 M adults), including routine 
assessment of the search function; recruitment of marrow 
donors, especially in underserved populations; development and 
maintenance of medical emergency contingency response capacity 
to a radiation disaster; and patient advocacy and case 
management services. Over 20,000 bone marrow/peripheral blood 
stem cell and cord bood transplants have taken place to date. 
The NMDP has demonstrably advanced the application of bone 
marrow transplantation. This legislation would modify and 
expand established programs, providing the Federal support that 
is necessary in order to more fully realize the potential of 
cord blood as a source material for stem cell transplantation.
    Particular issues to be addressed by the legislation 
include the following:
           The inventory of cord blood now available 
        falls far short of the estimated need (150,000 new 
        units);
           Lack of consistent standards for donor 
        identification, collection and storage of cord blood 
        have made some of the current inventory unsuitable for 
        treatment or, possibly, for research;
           A common, comprehensive, readily accessible 
        information system, designed to facilitate timely 
        identification of compatible transplant material, would 
        be of singular benefit to patients and providers alike;
           A database which records pertinent clinical 
        outcome measures would be of particular value to this 
        developing therapy; and
           Patients require informational/educational, 
        advocacy and cases management services, which may be 
        duplicated or unmet.
    Technologies pertinent to bone marrow/peripheral blood stem 
cell and cord blood collection often differ remarkably; 
however, given the complimentary nature of these stem cell 
therapies, it makes sense to combine federal support for them 
into a single Program, rather than reauthorizing the NBMDR and 
passing new legislation pertaining to cord blood alone. Both 
economies of scale and decreased duplication of services (e.g. 
patient education and advocacy, case management, and clinical 
outcomes database) should help reduce the costs of broadening 
support to include cord blood-related considerations. Given the 
complexities and rapid developments in these areas of stem cell 
therapy, the Advisory Council will provide the Secretary with a 
consolidated yet comprehensive source of expert, unbiased 
analysis and recommendations.
    A small proportion (> 5%) of the Program's funding is 
devoted to a demonstration project that allows for directed 
donation of cord blood for the benefit of first-degree 
relatives with conditions amenable to cord blood 
transplantation therapy. Cord blood units banked for this 
purpose must adhere to public standards and, if not used, will 
revert to the public inventory. Application for the 
demonstration project is at the discretion of the individual 
cord blood banks and is not a prerequisite for receiving the 
award of a contract for broader cord blood services.
    HIPAA regulations are sufficient to guarantee proper use of 
both the single portal of access to transplant material and to 
the outcomes database.
    Federal funding is necessary to create an inventory of cord 
blood units sufficient to meet estimated current patient needs. 
Funds authorized and appropriated for this purpose should be 
available only until the inventory goal specified is met by 
U.S. public cord blood banks. These funds are not intended to 
pay for individual clinical treatment that involves the use of 
cord blood; in particular, they are not intended to substitute 
for treatment-related reimbursement available through other 
federal programs (e.g. Medicaid/SCHIP, or through private 
insurance). The legislation also authorizes additional 
appropriations in support of the C.W. Bill Young Cell 
Transplantation Program; these latter funds are completely 
separate and distinct from the former and are not time-limited.

             III. Legislative History and Committee Action

    The Cord Blood Stem Cell Act of 2003 (H.R. 2852) was 
introduced on July 24, 2003 by Representative Chris Smith and 
35 co-sponsors, with the Senate version of the bill, S. 1717, 
introduced by Senators Orrin Hatch, Christopher Dodd, Arlen 
Specter and Sam Brownback on October 3, 2003. S. 1717/H.R. 2852 
directed the Secretary of Health and Human Services, through 
the Administrator of the Health Resources and Services 
Administration, to enter into contracts with qualified cord 
blood stem cell banks to assist in the establishment, provision 
and maintenance of a national Cord Blood Stem Cell Bank Network 
of at least 150,000 units of human cord blood stem cells. The 
legislation also established qualification requirements for 
participating banks and created a registry to allow patients 
and physicians to identify and obtain appropriate cord blood 
transplant material from the Network.
    In the 109th Congress, Senator Hatch re-introduced this 
cord blood bill as S. 681 on March 17, 2005, with Senators 
Christopher Dodd, Arlen Specter, Sam Brownback and Tom Harkin. 
Subsequently, in response to the Institute of Medicine's 
report, ``Cord Blood: Establishing a National Hematopoietic 
Stem Cell Bank Program'', as well as the House bill (H.R. 
2520), introduced by Representative Chris Smith and co-
sponsored by 78 other legislators, Senator Hatch, along with 
Senators Richard Burr, Christopher Dodd, John Ensign, and Jack 
Reed, began work on a revised bill that, like H.R. 2520, linked 
the creation of a national cord blood bank network with the 
reauthorization of the National Marrow Donor Program and on 
June 27, 2005, they introduced S. 1317, the Bone Marrow and 
Cord Blood Therapy and Research Act of 2005. S. 1317 provides 
for the collection and maintenance of cord blood units, for the 
treatment of patients and research, and to amend the PHSA to 
authorize creation of the Program, to increase the number of 
transplants for recipients suitably matched to donors of bone 
marrow and cord blood; this subsequent legislation incorporated 
key elements of the authoritative Institute of Medicine (IOM) 
report, ``Cord Blood: Establishing a National Hematopoietic 
Stem Cell Bank Program (2005)''. On June 29, 2005, the 
committee, meeting in executive session, modified the bill by 
approving a manager's amendment (Senator Enzi, for himself and 
Senators Hatch, Dodd, Burr, Reed, DeWine, Mikulski, Frist, 
Clinton, Enzi, Roberts, and Murray), then favorably ordered the 
bill reported.

              IV. Explanation of Bill and Committee Views

    This legislation builds upon the firm foundation 
established by the NMDP, which has advanced the public health 
by facilitating the application of one form of stem cell 
therapy--bone marrow transplantation--to the care of patients 
with a variety of malignant, metabolic and other diseases. It 
also draws upon the strength and experience of the long-
established blood banking profession in defining standards for 
collection and storage of blood products and accreditation of 
facilities. Each will do much to promote the use of this 
analogous therapy--cord blood transplantation. Yet the 
legislation acknowledges that more needs to be done in the area 
of cord blood transplantation.
    The committee, in its deliberations on S. 1317, placed 
particular emphasis on several aspects of the legislation and 
wants to state clearly the following points of consensus.
    Cord blood units collected through the program, but which 
subsequently prove unsuitable for use as transplant material, 
are still of great value for research purposes. For example, 
units that are collected and cryopreserved, but which later 
test positive on initial screening for infectious agents, as 
well as units with insufficient cell counts, can nevertheless 
be used for quality assurance testing and for ex vivo 
expansion, adoptive immunotherapy or graft engineering trials. 
The committee supports the use of some cord blood units 
collected under the auspices of the Program for research.
    It is not the intent of the committee that all clinical 
expenses pertaining to cord blood transplantation should be 
borne by the Program. It is expected that all pertinent public 
and private insurance reimbursement for clinical expenses 
related to cord blood transplantation will be fully utilized; 
in particular, no funds are available through the legislation 
for this purpose. This pertains to both public and private cord 
blood banks and includes both directed and random donation of 
cord blood. Cord blood units are owned by the cord blood banks 
to which they were donated. Eventual financial self-sufficiency 
regarding the inventory provisions of the legislation is 
desired and anticipated.
    The committee believes strongly in the value of 
Congressional oversight of the programs it authorizes and 
considers thoughtful, candid, timely reports to Congress to be 
an important part of this process. Thus, it anticipates that 
the reports mandated by this legislation will contain at least 
the following features:
           Related Cord Blood Donors Demonstration 
        Project--
                  Number of cord blood banks participating.
                  Number of cord blood units banked.
                  Number of units used for transplantation and 
                for research transplant or research results.
                  Amount of money spent by the bank in support 
                of this project.
           Advisory Council--
                  Recommendations made to the Secretary on all 
                matters related to the Program.
                  Actions taken by the Program on those 
                recommendations.
    The committee recognizes that cord blood transplantation is 
a promising, but emerging, therapy and that they have a unique 
opportunity to advance the science and practice through 
establishment of the Stem Cells Therapeutic Outcomes Database. 
Currently, the Bone Marrow Scientific Registry (BMSR) compiles 
outcomes data on transplant patients. Given the creation of the 
cord blood program, the BMSR is expanded and renamed the Stem 
Cells Therapeutic Outcomes Database. While the Advisory Council 
must play a leading role in its expansion, the database should 
contain at least the following elements: clinical diagnosis; 
donor selection criteria/matching considerations; preparatory 
regimen; transplant procedure; dosages; results; post-
transplant procedures; adverse events; and long-term follow-up.
    The committee thinks it is of pre-eminent importance that 
the Secretary promptly develops a scientifically sound 
definition of a ``high-quality cord blood unit'' and 
anticipates that the Advisory Council will play a prominent 
role in this process. Such a definition is likely to include 
quantitative measures of cell counts and viability, HLA typing 
resolution, match algorithms, and agreement on outcomes 
measurements; it is also likely to address quality testing, 
selection criteria, collection and transportation standards, 
confidentiality and integration of files, searches and general 
procedures. We feel that additional features of this 
legislation will facilitate the establishment of optimal 
standards:
           FDA participation on the Advisory Council;
           Establishment by the FDA of licensure 
        requirements for cord blood units;
           Continued authority of current FDA, other 
        Federal Government and state regulations; and
           Institutional accreditation.
    The committee understands that the actual practice of 
clinical medicine is and should be a highly individualized and 
personalized interaction between provider and patient. Nothing 
in this legislation should be construed, then, to limit the 
provider's right and responsibility to explore any and all 
sources of transplant material for their patient. This 
legislation is intended to help physicians to determine which 
source of stem cell material is best for their patient. 
Incumbent on the program, then, is the duty to present all 
pertinent information clearly and without bias or conflict of 
interest.
    To ensure equitable and efficient operation of the program, 
however, it is likely that the Secretary, in consultation with 
the Advisory Council and subject to public comment, will 
develop guidelines for participation, which should include at 
least the following:
           Unit reservation policies for cord blood 
        units;
           Informed consent policies for cord blood 
        donation;
           Accreditation requirements for public and 
        private cord blood banks; and
           Standardized data requirements for the 
        outcomes database and the single portal of access.
    The committee recognizes that health information technology 
has revolutionized the management of medical data. Thus, as per 
the IOM report recommendation, the committee intends that the 
statute be neutral regarding whether centralized (``real'') or 
decentralized (``virtual'') models of management are employed. 
It anticipates that the competitive process of contracting will 
encourage innovative solutions to the needs of the Program, 
especially those unique to the transplantation of cord blood. 
In determining that standard dataset, the Secretary should 
consider HLA typing, size of the cord blood unit, sex, and 
blood type of the donor.
    The requirements of the bone marrow and cord blood programs 
may differ; therefore, the Secretary is required to conduct a 
competition for the initial establishment of this cord blood 
function of the Program separate from that for the bone marrow/
peripheral blood stem cell function. The contracting 
competition should be transparent and without preconceptions; 
in particular, it should not exclude, de novo, any applicant or 
any particular model from the process.
    We emphasize, however, that any successful solution must 
provide for a single portal of entry for provider and patient 
alike, one that is accurate, comprehensive, understandable, 
easy to use and secure; in particular, the provider must have 
sufficient information readily available to choose 
intelligently and objectively amongst the various stem cell 
transplant materials available. A standardized, electronic 
format for presentation of the data to provider and patient 
will considerably facilitate this use.
    The committee expects that, with passage of this 
legislation, consideration of cord blood banking will be given, 
as a matter of routine, in all of the organ donation education 
and awareness campaigns currently sponsored by the Secretary. 
Furthermore, the committee anticipates that, as with other 
donor organ procurement programs, the Secretary will consider 
carefully how best to promote donation of cord blood.
    The committee strongly supports the IOM report 
recommendation that women be provided with a balanced 
perspective and clear information in order to participate, 
actively and knowledgably, in the choice of whether or how to 
donate cord blood. Informed consent is likely to include, at 
least, consideration of the following options: public donation 
or private storage; and disposal. Pertinent cost information 
must be conveyed to the potential donor. The discussion must be 
informed, objective, non-coercive and culturally sensitive; 
timing may be a crucial factor. Experience with other types of 
organ transplant may be instructive and should be considered in 
designing the approach to be used for cord blood donation.
    Medical contingency response to a terrorist emergency is 
particularly pertinent to cord blood as the source of stem cell 
transplant material most likely to be immediately available. It 
is the committee's expectation that the Secretary will, through 
the contracting process, address developing the capacity for 
urgent, large scale searches and attendant logistic 
considerations, including transportation and security. In 
addition, cord blood banks must be prepared to increase 
collection in order to maintain their public inventory in the 
case of an emergency.
    It is the committee's expectation that those Program 
functions which remain under the direct oversight of the 
Secretary, i.e. Patient Advocacy and Case Management, single 
point of access, and the Stem Cell Therapeutic Outcomes 
Database, will operate in a objective and unbiased manner, free 
from conflicts of interest, such that they can support use of 
the stem cell source material that is most appropriate for the 
individual patient.
    The legislation does not presume any fixed, pre-determined 
distribution of funds appropriated amongst the various 
functions of the program, save for the limit established for 
the demonstrations programs; rather, that is left to the 
Secretary's determination, providing the Secretary with the 
flexibility necessary to meet the pre-eminent goal of better 
patient care through stem cell transplantation therapy. 
However, in providing this flexibility, the committee does not 
intend to imply that the funds should be divided equally 
between the operators of the bone marrow and cord blood 
functions. When determining how to allocate funds under this 
program, the Secretary should ensure that his actions do 
nothing that would put the bone marrow functions of the Program 
at risk. The allocation of funds between section 3 and the Stem 
Cell Therapeutic Outcomes Database should be proportional to 
the relative functions of each. By combining the funding for 
emergency contingency planning with other program operations, 
the committee does not intend to diminish the importance of 
providing sufficient funds for the Program's activities related 
to national security and encourages the Secretary to ensure 
that the emergency contingency planning activities are 
adequately funded.
    The committee anticipated that the funding authorized for 
establishing and strengthening the cord blood unit inventory 
will be devoted primarily to defraying the start-up expenses, 
including developing the expanded inventory in an optimal 
fashion. While we feel that such activities clearly have the 
potential to be self-supporting in time, we also recognize that 
sufficient funding over an adequate period of time will be 
necessary for these activities to realize their full potential. 
It is the committee's expectation that the Secretary will 
closely scrutinize all costs related to this legislation, so 
that tax dollars are spent judiciously to achieve the maximum 
effect.
    The legislation neither requires nor precludes the 
Secretary from developing new regulations, either temporary or 
permanent, governing the program; however, the committee 
expects that, if passed, the legislation will be implemented 
promptly.
    The committee recognizes that some existing blood banks may 
have particular expertise in managing private or directed 
donation of cord blood, i.e. donations made expressly for the 
benefit of a first degree family member with a disease amenable 
to treatment with cord blood transplantation. In order to take 
advantage of this expertise, the committee interprets the 
legislation to permit applications from such blood banks for 
participation in the demonstration project only. These blood 
banks would not be required to provide analogous donor 
services, nor would they be required to submit an application 
for a more comprehensive contract. Blood banks submitting an 
application for the demonstration project only will, however, 
still need to meet all quality, accreditation and research 
requirements specified in the legislation. Funding for 
contracts awarded to these blood banks for a demonstration 
project would be apportioned from the same > 5 percent of the 
authorization of appropriations that funds demonstration 
projects awarded to blood banks under a more comprehensive 
contract. All qualified cord blood units donated through any 
form of the demonstration project which are not utilized by the 
intended recipient will revert to the public inventory 
established by the C.W. Bill Young Cell Transplantation 
Program.
    The Secretary has consistently required that the operator 
of the National Bone Marrow Donor Registry (NMBDR) conduct 
research to analyze data pertaining to blood stem cell 
transplantation. As part of previous contracts, the Secretary 
requires the contractor to establish and maintain a program of 
analysis and research regarding the operation of the Registry 
and persons' decisions regarding enrolling in the Registry, 
using data in the National Registry of Unrelated Marrow 
Transplants (renamed, in S. 1317, the Stem Cell Therapeutics 
Outcomes Database). Research priorities are established 
collaboratively with Registry committees possessing expertise 
in research and histocompatibility. The Secretary also requires 
the contractor to ensure that research conducted or sponsored 
by the Registry results in peer-reviewed published articles 
whenever possible.
    The committee recognizes the important research the NMDP 
has accomplished while operating the Registry. We support its 
goal to systematically remove all barriers to transplant and to 
increase the likelihood of finding a match for all who could 
potentially benefit from transplantation. There, S. 1317 
includes language authorizing the Secretary to continue having 
the contractor engage in the type of research projects it has 
historically required. The committee seeks to ensure that the 
contractor maintains the relationships the NMDP has established 
with other research organizations, donor centers, leading 
investigators, and the government. The committee seeks to 
formalize the existing research aspects of the program.

                            V. Cost Estimate

                                     U.S. Congress,
                               Congressional Budget Office,
                                     Washington, DC, July 14, 2005.
Hon. Mike Enzi,
Chairman, Committee on Health, Education, Labor, and Pensions,
U.S. Senate, Washington, DC.
    Dear Mr. Chairman: The Congressional Budget Office has 
prepared the enclosed cost estimate for S. 1317, the Stem Cell 
Therapeutic and Research Act of 2005.
    If you wish further details on this estimate, we will be 
pleased to provide them. The CBO staff contact is Tom Bradley.
            Sincerely,
                                       Douglas Holtz-Eakin,
                                                          Director.
    Enclosure.

S. 1317--Stem Cell Therapeutic and Research Act of 2005

    Summary: S. 1317 would amend the Public Health Service Act 
to provide for the collection and storage of umbilical cord 
blood and to authorize establishment of a program to increase 
the number of transplants of bone marrow and cord blood.
    CBO estimates that implementing S. 1317 would cost $3 
million in 2006 and $212 million over the 2006-2010 period, 
subject to the appropriation of the authorized amounts. 
Enacting S. 1317 could affect direct spending. This estimate 
assumes that S. 1317 will be enacted near the end of fiscal 
year 2005, in which case, CBO estimates the bill would not have 
a significant effect on direct spending. (If the bill is 
enacted by early August, CBO estimates that S. 1317 would shift 
outlays of $2 million from 2005 to 2006, thereby reducing 
direct spending by $2 million in 2005 and increasing direct 
spending by $2 million in 2006.) S. 1317 would have no effect 
on revenues.
    S. 1317 contains no intergovernmental or private-sector 
mandates as defined in the Unfunded Mandates Reform Act (UMRA) 
and would impose no costs on state, local, or tribal 
governments.
    Estimated cost to the Federal Government: The estimated 
budgetary impact of S. 1317 is shown in the following table. 
The costs of this legislation fall within budget function 550 
(health).

----------------------------------------------------------------------------------------------------------------
                                                                     By fiscal year, in millions of dollars--
                                                                 -----------------------------------------------
                                                                   2005    2006    2007    2008    2009    2010
----------------------------------------------------------------------------------------------------------------
                                        SPENDING SUBJECT TO APPROPRIATION

Spending Under Current Law:
    Budget Authority \1\........................................      10       0       0       0       0       0
    Estimated Outlays...........................................       3      10       6       0       0       0
Proposed Changes:
    Authorization Level.........................................       0      49      53      53      53      53
    Estimated Outlays...........................................       0      15      43      50      52      52
Spending Under S. 1317:
    Authorization Level \1\.....................................      10      49      53      53      53      53
    Estimated Outlays...........................................       3      25      49      50      53      53
----------------------------------------------------------------------------------------------------------------
\1\ The 2005 level is the amount appropriated in that year for the National Cord Blood Stem Cell Bank program.

    Basis of estimate: For this estimate, CBO assumes that S. 
1317 will be enacted in the fall of 2005, that the authorized 
amounts will be appropriated for each year, and that outlays 
will follow historical spending patterns for similar activities 
administered by the Health Resources and Services 
Administration (HRSA).

Spending subject to appropriation

    S. 1317 would require the Secretary of Health and Human 
Services to enter into contracts with cord-blood banks to 
establish and maintain an inventory of cord blood for 
transplantation. The bill also would require the Secretary to 
establish the C.W. Bill Young Cell Transplantation Program, 
with the purpose of increasing the number of transplants of 
bone marrow and cord blood.
    The bill would authorize the appropriation of $49 million 
in 2006 and $53 million a year in 2007 through 2010. (For the 
program to establish an inventory of cord blood, the bill also 
would make available through 2007 the amounts appropriated in 
2004 and 2005 for similar purposes--the effect of that 
provision is discussed below, under ``Direct Spending.'') CBO 
estimates that implementing S. 1317 would cost $15 million in 
2006 and $212 million over the 2006-2010 period, assuming 
appropriation of the authorized amounts.
    Cord Blood Inventory. The bill would authorize the 
Secretary to contract with qualified cord-blood banks to 
collect and maintain an inventory of 150,000 units of cord 
blood, and to make those units available for transplantation to 
recipients who are not related to the donor. In addition, the 
Secretary would conduct a demonstration program in which those 
cord-blood banks would collect and maintain cord-blood units 
donated by a family member for directed transplantation to a 
relative diagnosed with a condition that will benefit from 
transplantation.
    C.W. Bill Young Cell Transplantation Program. The bill 
would require the Secretary to establish an advisory council to 
make recommendations concerning the design and operation of a 
program to encourage the donation of bone marrow and cord blood 
and to facilitate the matching and distribution of those 
substances for transplantation. The bill also would require the 
Secretary to contract with one or more entities to carry out 
those activities.

Direct spending

    In both 2004 and 2005, the Congress appropriated $10 
million to ramain available until expended to establish a 
National Cord Blood Stem Cell Bank program within HRSA. The 
agency contracted with the Institute of medicine (IOM) to 
conduct a study and recommend a structure for that program. 
That study was completed in April 2005, and HRSA currently is 
engaged in the process of implementing the IOM's 
recommendations. Under current law, CBO estimates that HRSA 
will spend $3 million in 2005, $10 million in 2006, and $6 
million in 2007 on the National Cord Blood Stem Cell Bank 
program.
    The bill would make available for the program to collect 
and maintain an inventory of cord blood the funds appropriated 
in 2004 and 2005 for the National Cord Blood Stem Cell Bank 
program. Those funds would be available through 2007.
    Assuming enactment near the end of the fiscal year, CBO 
estimates that S. 1317 would not have a significant effect on 
the rate as which the previously appropriated funds will be 
spent. Therefore, CBO estimates that enacting S. 1317 would not 
have a significant effect on spending.
    However, if the bill is enacted by early August, CBO 
expects that HRSA would postpone the expenditure of about $2 
million from fiscal year 2005 to 2006, thereby reducing outlays 
by $2 million in 2005 and increasing outlays by $2 million in 
2006. Any such changes in the use of funds previously 
appropriated would constitute a change in direct spending. 
(That potential timing shift would have no net effect on 
outlays over the 2005-2006 period, and there would be no effect 
on budget authority.)
    Intergovernmental and private-sector impact: S. 1317 
contains no intergovernmental or private-sector mandates as 
defined in UMRA and would impose no costs on State, local, or 
tribal governments.
    Estimate prepared by: Federal Costs: Tom Bradley. Impact on 
State, Local, and Tribal Governments: Leo Lex. Impact on the 
Private Sector: Jennifer Doleac.
    Estimate approved by: Peter H. Fontaine, Deputy Assistant 
Director for Budget Analysis.

            VI. Application of Law to the Legislative Branch

    S. 1317 provides for the collection and maintenance of cord 
blood units for the treatment of patients and research. S. 1317 
also amends the PHSA to authorize the program, which will 
subsume and reauthorize the current NBDMR and add a new cord 
blood program.

                    VII. Regulatory Impact Statement

    This legislation requires the Secretary to develop 
standards that regulate the collection and storage of cord 
blood for undirected public use and for directed donation on 
behalf of a first-degree relative. It also requires the 
Secretary to identify a mechanism through which facilities can 
be accredited to perform these functions. The committee has 
determined that the bill will not have a significant regulatory 
impact.

                   VIII. Section-by-Section Analysis


Sec. 1. Short title

    The Stem Cell Therapeutic and Research Act of 2005.

Sec. 2. Cord blood inventory

    Instructs the Secretary of Health and Human Services (HHS) 
to enter into one time contracts with qualified cord blood 
banks in order to create and maintain a national inventory of 
150,000 new high quality cord blood units suitable for 
transplantation into unrelated recipients. In order to be 
considered qualified, a cord blood bank must meet certain 
quality standards, including accreditation by an entity 
recognized by the Secretary.
    Establishes a three year demonstration program, under which 
qualified banks can use a portion of funding for the collection 
and storage of cord blood units for a family where a first-
degree relative has been diagnosed with a condition that will 
benefit from transplantation. That donated unit is then 
reserved for use by that family until it is used or no longer 
needed, at which point it is released into the public 
inventory. Funding for this demonstration project is capped at 
five percent of appropriations, and units collected do not 
count toward the goal of 150,000.
    Specifies that previously appropriated funds shall remain 
available until the end of fiscal year 2007, and authorizes new 
appropriations of $15 million in each of fiscal years 2007-
2010.

Sec. 3. C.W. Bill Young Cell Transplantation Program

                         379--NATIONAL PROGRAM

    Establishes a transplantation program whose mission is to 
increase the amount of transplant material, both bone marrow 
and cord blood, available for use by unrelated patients. Toward 
that end, the Secretary of HHS may award a separate contract to 
perform each of the program's main functions--i.e., a national 
cord blood bank network and a national bone marrow registry 
program.
    Establishes an Advisory Council to advise the Secretary on 
how best to carry out program activities.
    Requires the Secretary to recognize one or more 
accreditation entities to accredit cord blood banks and ensure 
that they are meeting quality standards.
    Bone marrow functions: Details the responsibilities of the 
bone marrow entity within the Program. Language is very similar 
to existing statute that established the National Bone Marrow 
Donor Registry. Functions of the bone marrow program include 
operating a system to match patients to donors, recruiting 
donors (with an emphasis on ethnic and racial minorities), 
maintaining medical emergency contingency response 
capabilities, carrying out informational and educational 
activities, providing patient advocacy and case management, 
analyzing data on the functioning of the search process, and 
facilitating non-clinical research to improve transplantation.
    Cord blood functions: Details the responsibilities of the 
cord blood entity within the Program. Functions are similar to 
those listed for bone marrow, with some minor changes as 
appropriate.
    Single point of access; standard data: Requires the 
Secretary to ensure that health care professionals and patients 
are able to search electronically for and facilitate access to, 
in the manner and to the extent defined by the Secretary and 
consistent with the bone marrow and cord blood functions for 
operating a system for identifying, matching, and facilitating 
the distribution of bone marrow or cord blood that is suitably 
matched to candidate patients, cells from bone marrow and cord 
blood units through a single point of access. Mandates that the 
Secretary shall require all recipients of contracts under this 
section to make available a standard dataset for the single 
point of access in a standardized electronic format that 
enables transplant physicians to compare among and between bone 
marrow donors and cord blood units to ensure the best possible 
match for patients.
    Bone marrow recruitment; priorities; information and 
education: Requires the program to carry out activities for the 
recruitment and education of bone marrow donors and the general 
public, with an emphasis on increasing donation within 
underrepresented populations.
    Bone marrow criteria, standards, and procedures: 
Reauthorizes existing statute, with slight modifications, 
regarding the quality standards for entities involved bone 
marrow transplantation. [Note: Parallel language does not exist 
for cord blood, as quality assurance will be the primary 
responsibility of the accreditation entities and the FDA.]
    Cord blood recruitment; priorities; information and 
education: Parallels above bone marrow language.
    Patient advocacy and case management for bone marrow and 
cord blood: Establishes a single office to assist physicians, 
patients, donors, and families for whom the program has been 
requested to conduct a search for bone marrow and/or cord 
blood.

             379A--STEM CELL THERAPEUTIC OUTCOMES DATABASE

    Requires the Secretary to contract for the establishment of 
an outcomes database (presumably built on the existing Bone 
Marrow Scientific Registry), which will collect and maintain 
information on outcomes for all patients who have received 
treatments with stem cell therapeutics products.

                 379B--AUTHORIZATION OF APPROPRIATIONS

    Authorizes appropriations of $34 million in fiscal year 
2006 and $38 million in each of fiscal year 2007-2010.

                      IX. Changes in Existing Law

    In compliance with rule XXVI paragraph 12 of the Standing 
Rules of the Senate, the following provides a print of the 
statute or the part or section thereof to be amended or 
replaced (existing law proposed to be omitted is enclosed in 
black brackets, new matter is printed in italic, existing law 
in which no change is proposed is shown in roman):

PUBLIC HEALTH SERVICE ACT

           *       *       *       *       *       *       *


  PART I--[NATIONAL BONE MARROW DONOR REGISTRY] C.W. BILL YOUNG CELL 
                        TRANSPLANTATION PROGRAM


[SEC. 379. NATIONAL REGISTRY.

    [(a) Establishment.--The Secretary shall by contract 
establish and maintain a National Bone Marrow Donor Registry 
(referred to in this part as the ``Registry'') that has the 
purpose of increasing the number of transplants for recipients 
suitably matched to biologically unrelated donors of bone 
marrow, and that meets the requirements of this section. The 
Registry shall be under the general supervision of the 
Secretary, and under the direction of a board of directors 
meeting the following requirements:
          [(1) Each member of the board shall serve for a term 
        of 2 years, and each such member may serve as many as 3 
        consecutive 2-year terms, except that such limitations 
        shall not apply to the Chair of the board (or the 
        Chair-elect) or to the member of the board who most 
        recently served as the Chair.
          [(2) A member of the board may continue to serve 
        after the expiration of the term of such member until a 
        successor is appointed.
          [(3) In order to ensure the continuity of the board, 
        the board shall be appointed so that each year the 
        terms of approximately one-third of the members of the 
        board expire.
          [(4) The membership of the board shall include 
        representatives of marrow donor centers and marrow 
        transplant centers; recipients of a bone marrow 
        transplant; persons who require or have required such a 
        transplant; family members of such a recipient or 
        family members of a patient who has requested the 
        assistance of the Registry in searching for an 
        unrelated donor of bone marrow; persons with expertise 
        in the social sciences; and members of the general 
        public; and in addition nonvoting representatives from 
        the Naval Medical Research and Development Command and 
        from the Division of Organ Transplantation of the 
        Health Resources and Services Administration.
    [(b) Functions.--The Registry shall--
          [(1) establish a system for finding marrow donors 
        suitably matched to unrelated recipients for bone 
        marrow transplantation:
          [(2) carry out a program for the recruitment of bone 
        marrow donors in accordance with subsection (c), 
        including with respect to increasing the representation 
        of racial and ethnic minority groups (including persons 
        of mixed ancestry) in the enrollment of the Registry;
          [(3) carry out informational and educational 
        activities in accordance with subsection (c);
          [(4) annually update information to account for 
        changes in the status of individuals as potential 
        donors of bone marrow;
          [(5) provide for a system of patient advocacy through 
        the office established under subsection (d);
          [(6) provide case management services for any 
        potential donor of bone marrow to whom the Registry has 
        provided a notice that the potential donor may be 
        suitably matched to a particular patient (which 
        services shall be provided through a mechanism other 
        than the system of patient advocacy under subsection 
        (d)), and conduct surveys of donors and potential 
        donors to determine the extent of satisfaction with 
        such services and to identify ways in which the 
        services can be improved;
          [(7) with respect to searches for unrelated donors of 
        bone marrow that are conducted through the system under 
        paragraph (1), collect and analyze and publish data on 
        the number and percentage of patients at each of the 
        various stages of the search process, including data 
        regarding the furthest stage reached; the number and 
        percentage of patients who are unable to complete the 
        search process, and the reasons underlying such 
        circumstances; and comparisons of transplant centers 
        regarding search and other costs that prior to 
        transplantation are charged to patients by transplant 
        centers; and
          [(8) support studies and demonstration projects for 
        the purpose of increasing the number of individuals, 
        especially minorities, who are willing to be marrow 
        donors.
    [(c) Recruitment; Priorities; Information and Education.--
          [(1) Recruitment; priorities.--The Registry shall 
        carry out a program for the recruitment of bone marrow 
        donors. Such program shall identify populations that 
        are underrepresented among potential donors enrolled 
        with the Registry. In the case of populations that are 
        identified under the proceeding sentence:
                  [(A) The Registry shall give priority to 
                carrying out activities under this part to 
                increase representation for such populations in 
                order to enable a member of such a population, 
                to the extent practicable, to have a 
                probability of finding a suitable unrelated 
                donor that is comparable to the probability 
                that an individual would have.
                  [(B) The Registry shall consider racial and 
                ethnic minority groups (including persons of 
                mixed ancestry) to be populations that have 
                been identified for purposes of this paragraph, 
                and shall carry out subparagraph (A) with 
                respect to such populations.
          [(2) Information and education regarding recruitment; 
        testing and enrollment.--
                  [(A) In general.--In carrying out the program 
                under paragraph (1), the Registry shall carry 
                out informational and educational activities 
                for purposes of recruiting individuals to serve 
                as donors of bone marrow, and shall test and 
                enroll with the Registry potential donors. Such 
                information and educational activities shall 
                include the following:
                          [(i) Making information available to 
                        the general public, including 
                        information describing the needs of 
                        patients with respect to donors of bone 
                        marrow.
                          [(ii) Educating and providing 
                        information to individuals who are 
                        willing to serve as potential donors, 
                        including providing updates.
                          [(iii) Training individuals in 
                        requesting individuals to serve as 
                        potential donors.
                  [(B) Priorities.--In carrying out 
                informational and educational activities under 
                subparagraph (A), the Registry shall give 
                priority to recruiting individuals to serve as 
                donors of bone marrow for populations that are 
                identified under paragraph (1).
          [(3) Transplantation as treatment option.--In 
        addition to activities regarding recruitment, the 
        program under paragraph (1) shall provide information 
        to physicians, other health care professionals, and the 
        public regarding the availability, as a potential 
        treatment option, of receiving a transplant of bone 
        marrow from an unrelated donor.
    [(d) Patient Advocacy; Case Management.--
          [(1) In general.--The Registry shall establish and 
        maintain an office of patient advocacy (in this 
        subsection referred to as the ``Office'').
          [(2) General functions.--The Office shall meet the 
        following requirements:
                  [(A) The Office shall be headed by a 
                director.
                  [(B) The Office shall operate a system for 
                patient advocacy, which shall be separate from 
                mechanisms for donor advocacy, and which shall 
                serve patients for whom the Registry is 
                conducting, or has been requested to conduct, a 
                search for an unrelated donor of bone marrow.
                  [(C) In the case of such a patient, the 
                Office shall serve as an advocate for the 
                patient by directly providing to the patient 
                (or family members, physicians, or other 
                individuals acting on behalf of the patient) 
                individualized services with respect to 
                efficiently utilizing the system under 
                subsection (b)(1) to conduct an ongoing search 
                for a donor.
                  [(D) In carrying out subparagraph (C), the 
                Office shall monitor the system under 
                subsection (b)(1) to determine whether the 
                search needs of the patient involved are being 
                met, including with respect to the following:
                          [(i) Periodically providing to the 
                        patient (or an individual acting on 
                        behalf of the patient) information 
                        regarding donors who are suitability 
                        matched to the patient, and other 
                        information regarding the progress 
                        being made in the search.
                          [(ii) Informing the patient (or such 
                        other individual) if the search has 
                        been interrupted or discontinued.
                          [(iii) Identifying and resolving 
                        problems in the search, to the extent 
                        practicable.
                  [(E) In carrying out subparagraph (C), the 
                Office shall monitor the system under 
                subsection (b)(1) to determine whether the 
                Registry, donor centers, transplant centers, 
                and other entities participating in the 
                Registry program are complying with standards 
                issued under subsection (e)(4) for the system 
                for patient advocacy under this subsection.
                  [(F) The Office shall ensure that the 
                following data are made available to patients:
                          [(i) The resources available through 
                        the Registry.
                          [(ii) A comparison of transplant 
                        centers regarding search and other 
                        costs that prior to transplantation are 
                        charged to patients by transplant 
                        centers.
                          [(iii) A list of donor registries, 
                        transplant centers, and other entities 
                        that meet the applicable standards, 
                        criteria, and procedures under 
                        subsection (e).
                          [(iv) The posttransplant outcomes for 
                        individual transplant centers.
                          [(v) Such other information as the 
                        Registry determines to be appropriate.
                  [(G) The Office shall conduct surveys of 
                patients (or family members, physicians, or 
                other individuals acting on behalf of patients) 
                to determine the extent of satisfaction with 
                the system for patient advocacy under this 
                subsection, and to identify ways in which the 
                system can be improved.
          [(3) Case management.--
                  [(A) In general.--In serving as an advocate 
                for a patient under paragraph (2), the Office 
                shall provide individualized case management 
                services directly to the patient (or family 
                members, physicians, or other individuals 
                acting on behalf of the patient), including--
                          [(i) individualized case assessment; 
                        and
                          [(ii) the functions described in 
                        paragraph (2)(D) (relating to progress 
                        in the search process).
                  [(B) Postsearch functions.--In addition to 
                the case management services described in 
                paragraph (1) for patients, the Office may, on 
                behalf of patients who have completed the 
                search for an unrelated donor, provide 
                information and education on the process of 
                receiving a transplant of bone marrow, 
                including the posttransplant process.
      [(e) Criteria, Standards, and Procedures.--Not later than 
180 days after the date of enactment of this part, the 
Secretary shall establish and enforce, for entities 
participating in the program, including the Registry, 
individual marrow donor centers, marrow donor registries, 
marrow collection centers, and marrow transplant centers--
          [(1) quality standards and standards for tissue 
        typing, obtaining the informed consent of donors, and 
        providing patient advocacy;
          [(2) donor selection criteria, based on established 
        medical criteria, to protect both the donor and the 
        recipient and to prevent the transmission of 
        potentially harmful infectious diseases such as the 
        viruses that cause hepatitis and the etiologic agent 
        for Acquired Immune Deficiency Syndrome; 
          [(3) procedures to ensure the proper collection and 
        transportation of the marrow; 
          [(4) standards for the system for patient advocacy 
        operated under subsection (d), including standards 
        requiring the provision of appropriate information (at 
        the start of the search process and throughout the 
        process) to patients and their families Physicians;
          [(5) standards that--
                  [(A) require the establishment of a system of 
                strict confidentiality of records relating to 
                the identity, address, HLA type, and managing 
                marrow donor center for marrow donors and 
                potential marrow donors; and
                  [(B) prescribe the purposes for which the 
                records described in subparagraph (A) may be 
                disclosed, and the circumstances and extent of 
                the disclosure; and
          [(6) in the case of a marrow donor center or marrow 
        donor registry participating in the program, procedures 
        to ensure the establishment of a method for integrating 
        donor files, searches, and general procedures of the 
        center or registry with the Registry.
    [(f) Comment Procedures.--The Secretary shall establish and 
provide information to the public on procedures, which may 
include establishment of a policy advisory committee, under 
which the Secretary shall receive and consider comments from 
interested persons relating to the manner in which the Registry 
is carrying out the duties of the Registry under subsection (b) 
and complying with the criteria, standards, and procedures 
described in subsection (e).
    [(g) Consultation.--The Secretary shall consult with the 
board of the directors of the Registry and the bone marrow 
donor program of the Department of the Navy in developing 
policies affecting the Registry.
    [(h) Application.--To be eligible to enter into a contract 
under section, an entity shall submit to the Secretary and 
obtain approval of an application at such time, in such manner, 
and containing such information as the Secretary shall by 
regulation prescribe.
    [(i) Eligibility.--Entities eligible to receive a contract 
under this section shall include private nonprofit entities.
    [(j) Records.--
          [(1) Recordkeeping.--Each recipient of a contract or 
        subcontract under subsection (a) shall keep such 
        records as the Secretary shall prescribe, including 
        records that fully disclose the amount and disposition 
        by the recipient of the proceeds of the contract, the 
        total cost of the undertaking in connection with which 
        the contract was made, and the amount of the portion of 
        the cost of the undertaking supplied by other sources, 
        and such other records as will facilitate an effective 
        audit.
          [(2) Examination of records.--The Secretary and the   
        Comptroller General of the United States shall have 
        access to any books, documents, papers, and records of 
        the recipient of a contract or subcontract entered into 
        under this section that are pertinent to the contract, 
        for the purpose of conducting audits and examinations.
    [(k) Penalties for Disclosure.--Any person who discloses 
the content of any record referred to in subsection (e)(5)(A) 
without the prior written consent of the donor or potential 
donor with respect  to whom the record is maintained, or in 
violation of the standards described in subsection (e)(5)(B), 
shall be imprisoned for not more  than 2 years or fined in 
accordance with title 18, United States Code or both.
    [(l) Annual Report Regarding Pretransplant Costs.--The  
Registry shall annually submit to the Secretary the data 
collected under subsection (b)(7) on comparisons of transplant 
centers regarding search and other costs that prior to 
transplantation are charged to patients by transplant centers. 
The data shall be submitted to the Secretary through inclusion 
in the annual report required in section 379A(c).]

SEC. 379. NATIONAL PROGRAM.

    (a) Establishment.--The Secretary, acting through the 
Administrator of the Health Resources and Services 
Administration, shall by one or more contracts establish and 
maintain a C.W. Bill Young Cell Transplantation Program 
(referred to in this section as the ``Program''), successor to 
the National Bone Marrow Donor Registry, that has the purpose 
of increasing the number of transplants for recipients suitably 
matched to biologically unrelated donors of bone marrow and 
cord blood, and that meets the requirements of this section. 
The Secretary may award a separate contract to perform each of 
the major functions of the Program described in paragraphs (1) 
and (2) of subsection (d) if deemed necessary by the Secretary 
to operate an effective and efficient system that is in the 
best interest of patients. The Secretary shall conduct a 
separate competition for the initial establishment of the cord 
blood functions of the Program. The Program shall be under the 
general supervision of the Secretary. The Secretary shall 
establish an Advisory Council to advise, assist, consult with, 
and make recommendations to the Secretary on matters related to 
the activities carried out by the Program. The members of the 
Advisory Council shall be appointed in accordance with the 
following:
          (1) Each member of the Advisory Council shall serve 
        for a term of 2 years, and each such member may serve 
        as many as 3 consecutive 2-year terms, except that--
                  (A) such limitations shall not apply to the 
                Chair of the Advisory Council (or the Chair-
                elect) or to the member of the Advisory Council 
                who most recently served as the Chair; and
                  (B) 1 additional consecutive 2-year term may 
                be served by any member of the Advisory Council 
                who has no employment, governance, or financial 
                affiliation with any donor center, recruitment 
                organization, transplant center, or cord blood 
                bank.
          (2) A member of the Advisory Council may continue to 
        serve after the expiration of the term of such member 
        until a successor is appointed.
          (3) In order to ensure the continuity of the Advisory 
        Council, the Advisory Council shall be appointed so 
        that each year the terms of approximately one-third of 
        the members of the Advisory Council expire.
          (4) The membership of the Advisory Council--
                  (A) shall include as voting members a 
                balanced number of representatives including 
                representatives of marrow donor centers and 
                marrow transplant centers, representatives of 
                cord blood banks and participating birthing 
                hospitals, recipients of a bone marrow 
                transplant, recipients of a cord blood 
                transplant, persons who require such 
                transplants, family members of such a recipient 
                or family members of a patient who has 
                requested the assistance of the Program in 
                searching for an unrelated donor of bone marrow 
                or cord blood, persons with expertise in bone 
                marrow and cord blood transplantation, persons 
                with expertise in typing, matching, and 
                transplant outcome data analysis, persons with 
                expertise in the social sciences, basic 
                scientists with expertise in the biology of 
                adult stem cells, and members of the general 
                public; and
                  (B) shall include as nonvoting members 
                representatives from the Department of Defense 
                Marrow Donor Recruitment and Research Program 
                operated by the Department of the Navy, the 
                Division of Transplantation of the Health 
                Resources and Services Administration, the Food 
                and Drug Administration, and the National 
                Institutes of Health.
          (5) Members of the Advisory Council shall be chosen 
        so as to ensure objectivity and balance and reduce the 
        potential for conflicts of interest. The Secretary 
        shall establish bylaws and procedures--
                  (A) to prohibit any member of the Advisory 
                Council who has an employment, governance, or 
                financial affiliation with a donor center, 
                recruitment organization, transplant center, or 
                cord blood bank from participating in any 
                decision that materially affects the center, 
                recruitment organization, transplant center, or 
                cord blood bank; and
                  (B) to limit the number of members of the 
                Advisory Council with any such affiliation.
          (6) The Secretary, acting through the Advisory 
        Council, shall submit to the Congress--
                  (A) an annual report on the activities 
                carried out under this section; and
                  (B) not later than 6 months after the date of 
                the enactment of the Stem Cell Therapeutic and 
                Research Act of 2005, a report of 
                recommendations on the scientific factors 
                necessary to define a cord blood unit as a 
                high-quality unit.
    (b) Accreditation.--The Secretary shall, through a public 
process, recognize one or more accreditation entities for the 
accreditation of cord blood banks.
    (c) Informed Consent.--The Secretary shall, through a 
public process, examine issues of informed consent, including--
          (1) the appropriate timing of such consent; and
          (2) the information provided to the maternal donor 
        regarding all of her medically appropriate cord blood 
        options.
Based on such examination, the Secretary shall require that the 
standards used by the accreditation entities recognized under 
subsection (b) ensure that a cord blood unit is acquired with 
the informed consent of the maternal donor.
    (d) Functions.--
          (1) Bone marrow functions.--With respect to bone 
        marrow, the Program shall--
                  (A) operate a system for identifying, 
                matching, and facilitating the distribution of 
                bone marrow that is suitably matched to 
                candidate patients;
                  (B) consistent with paragraph (3), permit 
                transplant physicians, other appropriate health 
                care professionals, and patients to search by 
                means of electronic access all available bone 
                marrow donors listed in the Program;
                  (C) carry out a program for the recruitment 
                of bone marrow donors in accordance with 
                subsection (e), including with respect to 
                increasing the representation of racial and 
                ethnic minority groups (including persons of 
                mixed ancestry) in the enrollment of the 
                Program;
                  (D) maintain and expand medical contingency 
                response capabilities, in coordination with 
                Federal programs, to prepare for and respond 
                effectively to biological, chemical, or 
                radiological attacks, and other public health 
                emergencies that can damage marrow, so that the 
                capability of supporting patients with marrow 
                damage from disease can be used to support 
                casualties with marrow damage;
                  (E) carry out informational and educational 
                activities in accordance with subsection (e);
                  (F) at least annually update information to 
                account for changes in the status of 
                individuals as potential donors of bone marrow;
                  (G) provide for a system of patient advocacy 
                through the office established under subsection 
                (h);
                  (H) provide case management services for any 
                potential donor of bone marrow to whom the 
                Program has provided a notice that the 
                potential donor may be suitably matched to a 
                particular patient through the office 
                established under subsection (h);
                  (I) with respect to searches for unrelated 
                donors of bone marrow that are conducted 
                through the system under subparagraph (A), 
                collect, analyze, and publish data in a 
                standardized electronic format on the number 
                and percentage of patients at each of the 
                various stages of the search process, including 
                data regarding the furthest stage reached, the 
                number and percentage of patients who are 
                unable to complete the search process, and the 
                reasons underlying such circumstances;
                  (J) support studies and demonstration and 
                outreach projects for the purpose of increasing 
                the number of individuals who are willing to be 
                marrow donors to ensure a genetically diverse 
                donor pool; and
                  (K) facilitate research with the appropriate 
                Federal agencies to improve the availability, 
                efficiency, safety, and cost of transplants 
                from unrelated donors and the effectiveness of 
                Program operations.
          (2) Cord blood functions.--With respect to cord 
        blood, the Program shall--
                  (A) operate a system for identifying, 
                matching, and facilitating the distribution of 
                donated cord blood units that are suitably 
                matched to candidate patients and meet all 
                applicable Federal and State regulations 
                (including informed consent and Food and Drug 
                Administration regulations) from a qualified 
                cord blood bank;
                  (B) consistent with paragraph (3), allow 
                transplant physicians, other appropriate health 
                care professionals, and patients to search by 
                means of electronic access all available cord 
                blood units made available through the Program;
                  (C) allow transplant physicians and other 
                appropriate health care professionals to 
                reserve, as defined by the Secretary, a cord 
                blood unit for transplantation;
                  (D) support studies and demonstration and 
                outreach projects for the purpose of increasing 
                cord blood donation to ensure a genetically 
                diverse collection of cord blood units;
                  (E) provide for a system of patient advocacy 
                through the office established under subsection 
                (h);
                  (F) coordinate with the qualified cord blood 
                banks to support informational and educational 
                activities in accordance with subsection (g);
                  (G) maintain and expand medical contingency 
                response capabilities; in coordination with 
                Federal programs, to prepare for and respond 
                effectively to biological, chemical, or 
                radiological attacks, and other public health 
                emergencies that can damage marrow, so that the 
                capability of supporting patients with marrow 
                damage from disease can be used to support 
                causalities with marrow damage; and
                  (H) with respect to the system under 
                subparagraph (A) collect, analyze, and publish 
                data in a standardized electronic format, as 
                required by the Secretary, on the number and 
                percentage of patients at each of the various 
                stages of the search process, including data 
                regarding the furthest stage reached, the 
                number and percentage of patients who are 
                unable to complete the search process, and the 
                reasons underlying such circumstances.
          (3) Single point of access; standard data.--
                  (A) Single point of access.--The Secretary 
                shall ensure that health care professionals and 
                patients are able to search electronically for 
                and facilitate access to, in the manner and to 
                the extent defined by the Secretary and 
                consistent with the functions described in 
                paragraphs (1)(A) and (2)(A), cells from bone 
                marrow donors and cord blood units through a 
                single point of access.
                  (B) Standard data.--The Secretary shall 
                require all recipients of contracts under this 
                section to make available a standard dataset 
                for purposes of subparagraph (A) in a 
                standardized electronic format that enables 
                transplant physicians to compare among and 
                between bone marrow donors and cord blood units 
                to ensure the best possible match for the 
                patient.
          (4) Definition.--The term ``qualified cord blood 
        bank'' means a cord blood bank that--
                  (A) has obtained all applicable Federal and 
                State licenses, certifications, registrations 
                (including pursuant to the regulations of the 
                Food and Drug Administration), and other 
                authorizations required to operate and maintain 
                a cord blood bank;
                  (B) has implemented donor screening, cord 
                blood collection practices, and processing 
                methods intended to protect the health and 
                safety of donors and transplant recipients to 
                improve transplant outcomes, including with 
                respect to the transmission of potentially 
                harmful infections and other diseases;
                  (C) is accredited by an accreditation entity 
                recognized by the Secretary under subsection 
                (b);
                  (D) has established a system of strict 
                confidentiality to protect the identity and 
                privacy of patients and donors in accordance 
                with existing Federal and State law;
                  (E) has established a system for encouraging 
                donation by a genetically diverse group of 
                donors; and
                  (F) has established a system to 
                confidentially maintain linkage between a cord 
                blood unit and a maternal donor.
    (e) Bone Marrow Recruitment; Priorities; Information and 
Education.--
          (1) Recruitment; priorities.--The Program shall carry 
        out activities for the recruitment of bone marrow 
        donors. Such recruitment program shall identify 
        populations that are underrepresented among potential 
        donors enrolled with the Program. In the case of 
        populations that are identified under the preceding 
        sentence:
                  (A) The Program shall give priority to 
                carrying out activities under this part to 
                increase representation for such populations in 
                order to enable a member of such a population, 
                to the extent practicable, to have a 
                probability of finding a suitable unrelated 
                donor that is comparable to the probability 
                that an individual who is not a member of an 
                underrepresented population would have.
                  (B) The Program shall consider racial and 
                ethnic minority groups (including persons of 
                mixed ancestry) to be populations that have 
                been identified for purposes of this paragraph, 
                and shall carry out subparagraph (A) with 
                respect to such populations.
          (2) Information and education regarding recruitment; 
        testing and enrollment.--
                  (A) In general.--The Program shall carry out 
                informational and educational activities, in 
                coordination with organ donation public 
                awareness campaigns operated through the 
                Department of Health and Human Services, for 
                purposes of recruiting individuals to serve as 
                donors of bone marrow, and shall test and 
                enroll with the Program potential bone marrow 
                donors. Such information and educational 
                activities shall included the following:
                          (i) Making information available to 
                        the general public, including 
                        information describing the needs of 
                        patients with respect to donors of bone 
                        marrow.
                          (ii) Educating and providing 
                        information to individuals who are 
                        willing to serve as potential bone 
                        marrow donors.
                          (iii) Training individuals in 
                        requesting individuals to serve as 
                        potential bone marrow donors.
                  (B) Priorities.--In carrying out 
                informational and educational activities under 
                subparagraph (A), the Program shall give 
                priority to recruiting individuals to serve as 
                donors of bone marrow for populations that are 
                identified under paragraph (1).
          (3) Transplantation as treatment option.--In addition 
        to activities regarding recruitment, the recruitment 
        program under paragraph (1) shall provide information 
        to physicians, other health care professionals, and the 
        public regarding bone marrow transplants from unrelated 
        donors as a treatment option.
          (4) Implementation of subsection.--The requirements 
        of this subsection shall be carried out by the entity 
        that has been awarded a contract by the Secretary under 
        subsection (a) to carry out the functions described in 
        subsection (d)(1).
    (f) Bone Marrow Criteria, Standards, and Procedures.--The 
Secretary shall enforce, for participating entities, including 
the Program, individual marrow donor centers, marrow donor 
registries, marrow collection centers, and marrow transplant 
centers--
          (1) quality standards and standards for tissue 
        typing, obtaining the informed consent of donors, and 
        providing patient advocacy;
          (2) donor selection criteria, based on established 
        medical criteria, to protect both the donor and the 
        recipient and to prevent the transmission of 
        potentially harmful infectious diseases such as the 
        viruses that cause hepatitis and the etiologic agent 
        for Acquired Immune Deficiency Syndrome;
          (3) procedures to ensure the proper collection and 
        transportation of the marrow;
          (4) standards for the system for patient advocacy 
        operated under subsection (h), including standards 
        requiring the provision of appropriate information (at 
        the start of the search process and throughout the 
        process) to patients and their families and physicians;
          (5) standards that--
                  (A) require the established of a system of 
                strict confidentiality of records relating to 
                the identity, address, HLA type, and managing 
                marrow donor center for marrow donors and 
                potential marrow donors; and
                  (B) prescribe the purposes for which the 
                records described in subparagraph (A) may be 
                disclosed, and the circumstances and extent of 
                the disclosure; and
          (6) in the case of a marrow donor center or marrow 
        donor registry participating in the program, procedures 
        to ensure the establishment of a method for integrating 
        donor files, searches, and general procedures of the 
        center or registry with the Program.
    (g) Cord Blood Recruitment; Priorities; Information and 
Education.--
          (1) Recruitment; priorities.--The Program shall 
        support activities, in cooperation with qualified cord 
        blood banks, for the recruitment of cord blood donors. 
        Such recruitment program shall identify populations 
        that are underrepresented among cord blood donors. In 
        the case of populations that are identified under the 
        preceding sentence:
                  (A) The Program shall give priority to 
                supporting activities under this part to 
                increase representation for such populations in 
                order to enable a member of such a population, 
                to the extent practicable, to have a 
                probability of finding a suitable cord blood 
                unit that is comparable to the probability that 
                an individual who is not a member of an 
                underrepresented population would have.
                  (B) The Program shall consider racial and 
                ethnic minority groups (including persons of 
                mixed ancestry) to be populations that have 
                been identified for purposes of this paragraph, 
                and shall support activities under subparagraph 
                (A) with respect to such populations.
          (2) Information and education regarding recruitment; 
        testing and donation.--
                  (A) In general.--In carrying out the 
                recruitment program under paragraph (1), the 
                Program shall support informational and 
                educational activities in coordination with 
                qualified cord blood banks and organ donation 
                public awareness campaigns operated through the 
                Department of Health and Human Services, for 
                purposes of recruiting pregnant women to serve 
                as donors of cord blood. Such information and 
                educational activities shall include the 
                following:
                          (i) Making information available to 
                        the general public, including 
                        information describing the needs of 
                        patients with respect to cord blood 
                        units.
                          (ii) Educating and providing 
                        information to pregnant women who are 
                        willing to donate cord blood units.
                          (iii) Training individuals in 
                        requesting pregnant women to serve as 
                        cord blood donors.
                  (B) Priorities.--In carrying out 
                informational and educational activities under 
                subparagraph (A), the Program shall give 
                priority to supporting the recruitment of 
                pregnant women to serve as donors of cord blood 
                for populations that are identified under 
                paragraph (1).
          (3) Transplantation as treatment option.--In addition 
        to activities regarding recruitment, the recruitment 
        program under paragraph (1) shall provide information 
        to physicians, other health care professionals, and the 
        public regarding cord blood transplants from donors as 
        a treatment option.
          (4) Implementation of subsection.--The requirements 
        of this subsection shall be carried out by the entity 
        that has been awarded a contract by the Secretary under 
        subsection (a) to carry out the functions described in 
        subsection (d)(2).
    (h) Patient Advocacy and Case Management for Bone Marrow 
and Cord Blood.--
          (1) In general.--The Secretary shall establish and 
        maintain, through a contract or other means determined 
        appropriate by the Secretary, an office of patient 
        advocacy (in this subsection referred to as the 
        ``Office'').
          (2) General functions.--The Office shall meet the 
        following requirements:
                  (A) The Office shall be headed by a director.
                  (B) The Office shall be staffed by 
                individuals with expertise in bone marrow and 
                cord blood therapy covered under the Program.
                  (C) The Office shall operate a system for 
                patient advocacy, which shall be separate from 
                mechanisms for donor advocacy, and which shall 
                serve patients for whom the Program is 
                conducting, or has been requested to conduct a 
                search for a bone marrow donor or cord blood 
                unit.
                  (D) In the case of such a patient, the Office 
                shall serve as an advocate for the patient by 
                directly providing to the patient (or family 
                members, physicians, or other individuals 
                acting on behalf of the patient) individualized 
                services with respect to efficiently utilizing 
                the system under paragraphs (1) and (2) of 
                subsection (d) to conduct an ongoing search for 
                a bone marrow donor or cord blood unit and 
                assist with information regarding third party 
                payor matters.
                  (E) In carrying out subparagraph (D), the 
                Office shall monitor the system under 
                paragraphs (1) and (2) of subsection (d) to 
                determine whether the search needs of the 
                patient involved are being met, including with 
                respect to the following:
                          (i) Periodically providing to the 
                        patient (or an individual acting on 
                        behalf of the patient) information 
                        regarding bone marrow donors or cord 
                        blood units that are suitably matched 
                        to the patient, and other information 
                        regarding the progress being made in 
                        the search.
                          (ii) Informing the patient (or such 
                        other individual) if the search has 
                        been interrupted or discontinued.
                          (iii) Identifying and resolving 
                        problems in the search, to the extent 
                        practicable.
                  (F) The Office shall ensure that the 
                following data are made available to patients:
                          (i) The resources available through 
                        the Program.
                          (ii) A comparison of transplant 
                        centers regarding search and other 
                        costs that prior to transplantation are 
                        charged to patients by transplant 
                        centers.
                          (iii) The post-transplant outcomes 
                        for individual transplant centers.
                          (iv) Information concerning issues 
                        that patients may face after a 
                        transplant.
                          (v) Such other information as the 
                        Program determines to be appropriate.
                  (G) The Office shall conduct surveys of 
                patients (or family members, physicians, or 
                other individuals acting on behalf of patients) 
                to determine the extent of satisfaction with 
                the system for patient advocacy under this 
                subsection, and to identify ways in which the 
                system can be improved to best meet the needs 
                of patients.
          (3) Case management.--
                  (A) In general.--In serving as an advocate 
                for a patient under paragraph (2), the Office 
                shall provide individualized case management 
                services directly to the patient (or family 
                members, physicians, or other individuals 
                acting on behalf of the patient), including--
                          (i) individualized case assessment; 
                        and
                          (ii) the functions described in 
                        paragraph (2)(D) (relating to progress 
                        in the search process).
                  (B) Postsearch functions.--In addition to the 
                case management services described in paragraph 
                (1) for patients, the Office shall, on behalf 
                of patients who have completed the search for a 
                bone marrow donor or cord blood unit, provide 
                information and education on the process of 
                receiving a transplant, including the post-
                transplant process.
    (i) Comment Procedures.--The Secretary shall establish and 
provide information to the public on procedures under which the 
Secretary shall receive and consider comments from interested 
persons relating to the manner in which the Program is carrying 
out the duties of the Program. The Secretary may promulgate 
regulations under this section.
    (j) Consultation.--In developing policies affecting the 
Program, the Secretary shall consult with the Advisory Council, 
the Department of Defense Marrow Donor Recruitment and Research 
Program operated by the Department of the Navy, and the board 
of directors of each entity awarded a contract under this 
section.
    (k) Contracts.--
          (1) Application.--To be eligible to enter into a 
        contract under this section, an entity shall submit to 
        the Secretary and obtain approval of an application at 
        such time, in such manner, and containing such 
        information as the Secretary shall by regulation 
        prescribe.
          (2) Considerations.--In awarding contracts under this 
        section, the Secretary shall give consideration to the 
        continued safety of donors and patients and other 
        factors deemed appropriate by the Secretary.
    (l) Eligibility.--Entities eligible to receive a contract 
under this section shall include private nonprofit entities.
    (m) Records.--
          (1) Recordkeeping.--Each recipient of a contract or 
        subcontract under subsection (a) shall keep such 
        records as the Secretary shall prescribe, including 
        records that fully disclose the amount and disposition 
        by the recipient of the proceeds of the contract, the 
        total cost of the undertaking in connection with which 
        the contract was made, and the amount of the portion of 
        the cost of the undertaking supplied by other sources, 
        and such other records as will facilitate an effective 
        audit.
          (2) Examination of records.--The Secretary and the 
        Comptroller General of the United States shall have 
        access to any books, documents, papers, and records of 
        the recipient of a contract or subcontract entered into 
        under this section that are pertinent to the contract, 
        for the purpose of conducting audits and examinations.
    (n) Penalties for Disclosure.--Any person who discloses the 
content of any record referred to in subsection (d)(4)(D) or 
(f)(5)(A) without the prior written consent of the donor or 
potential donor with respect to whom the record is maintained, 
or in violation of the standards described in subsection 
(f)(5)(B), shall be imprisoned for not more than 2 years or 
fined in accordance with title 18, United States Code, or both.

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[SEC. 379A. BONE MARROW SCIENTIFIC REGISTRY.

    [(a) Establishment of Recipient Registry.--The Secretary, 
acting through the Registry under section 379 (in this section 
referred to as the ``Registry''), shall establish and maintain 
a scientific registry of information relating to patients who 
have been recipients of a transplant of bone marrow from a 
biologically unrelated donor.
    [(b) Information.--The scientific registry under subsection 
(a) shall include information with respect to patients 
described in subsection (a), transplant procedures and such 
other information as the Secretary determines to be appropriate 
to conduct an ongoing evaluation of the scientific and clinical 
status of transplantation involving recipients of bone marrow 
from biologically unrelated donors.
    [(c) Annual Report on Patient Outcomes.--The Registry shall 
annually submit to the Secretary a report concerning patient 
outcomes with respect to each transplant center. Each such 
report shall use data collected and maintained by the 
scientific registry under subsection (a). Each such report 
shall in addition include the data required in section 379(l) 
(relating to pretransplant costs).]

SEC. 379A. STEM CELL THERAPEUTIC OUTCOMES DATABASE.

    (a) Establishment.--The Secretary shall by contract 
establish and maintain a scientific database of information 
relating to patients who have been recipients of a stem cell 
therapeutics product (including bone marrow, cord blood, or 
other such product) from a donor.
    (b) Information.--The outcomes database shall include 
information in a standardized electronic format with respect to 
patients described in subsection (a), diagnosis, transplant 
procedures, results, long-term follow-up, and such other 
information as the Secretary determines to be appropriate, to 
conduct an ongoing evaluation of the scientific and clinical 
status of transplantation involving recipients of a stem cell 
therapeutics product from a donor.
    (c) Annual Report on Patient Outcomes.--The Secretary shall 
require the entity awarded a contract under this section to 
submit to the Secretary an annual report concerning patient 
outcomes with respect to each transplant center, based on data 
collected and maintained by the entity pursuant to this 
section.
    (d) Publicly Available Data.--The outcomes database shall 
make relevant scientific information not containing 
individually identifiable information available to the public 
in the form of summaries and data sets to encourage medical 
research and to provide information to transplant programs, 
physicians, patients, and entities awarded a contract under 
section 379 donor registries, and cord blood banks.

           *       *       *       *       *       *       *


SEC. 379A-1. DEFINITIONS.

    In this part:
          (1) The term ``Advisory Council'' means the advisory 
        council established by the Secretary under section 
        379(a)(1).
          (2) The term ``bone marrow'' means the cells found in 
        adult bone marrow and peripheral blood.
          (3) The term ``outcomes database'' means the database 
        established by the Secretary under section 379A.
          (4) The term ``Program'' means the C.W. Bill Young 
        Cell Transplantation Program established under section 
        379.

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[SEC. 379B. AUTHORIZATION OF APPROPRIATIONS.

    [For the purpose of carrying out this part, there are 
authorized to be appropriated $18,000,000 for fiscal year 1999, 
and such sums as may be necessary for each of the fiscal years 
2000 through 2003.]

SEC. 379B. AUTHORIZATION OF APPROPRIATIONS.

    For the purpose of carrying out this part, there are 
authorized to be appropriated $34,000,000 for fiscal year 2006 
and $38,000,000 for each of fiscal years 2007 through 2010.

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