[Federal Register Volume 78, Number 191 (Wednesday, October 2, 2013)]
[Proposed Rules]
[Pages 60810-60813]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-24094]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

42 CFR Part 121

RIN 0906-AB02


Change to the Definition of ``Human Organ'' Under Section 301 of 
the National Organ Transplant Act of 1984

AGENCY: Health Resources and Services Administration, HHS.

ACTION: Notice of proposed rulemaking.

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SUMMARY: This notice seeks public comment on the proposed change in the 
definition of ``human organ'' in section

[[Page 60811]]

301 of the National Organ and Transplant Act of 1984, as amended, 
(NOTA) to explicitly incorporate hematopoietic stem cells (HSCs) within 
peripheral blood in the definition of ``bone marrow.'' This would 
clarify that the prohibition on transfers of human organs for valuable 
consideration applies to HSCs regardless of whether they were recovered 
directly from bone marrow (by aspiration) or from peripheral blood (by 
apheresis). This amendment will also conform section 301 to the 
provisions of the Stem Cell Research and Therapeutic Act of 2005, as 
amended.

DATES: To be considered, comments should be submitted by December 2, 
2013. Subject to consideration of the comments submitted, the 
Department intends to publish final regulations.

ADDRESSES: You may submit comments, identified by Regulatory 
Information Number RIN 0906-AB02, by any of the following methods, but 
the first option is preferred:
     Federal eRulemaking Portal: http://www.regulations.gov. 
Follow the instructions for submitting comments.
     Agency Web site: http://www.hrsa.gov/. Follow the 
instructions for submitting comments on the Agency Web site.
     Email: [email protected]. Include RIN 0906-AB02 in the 
subject line of the message.
     Fax: (301) 594-6095.
     Mail: Shelley Grant, MHSA, Branch Chief, Blood Stem Cell 
Transplantation Program, Division of Transplantation, Healthcare 
Systems Bureau, Health Resources and Services Administration, 5600 
Fishers Lane, Room 12C-06, Rockville, Maryland 20857.
    Instructions: All submissions must include the agency name and RIN 
for this rulemaking. All comments received will be posted without 
change to http://www.hrsa.gov/, including any personal information 
provided. Additional information concerning the submission of comments 
and/or the rulemaking process can be obtained from the Regulations 
Officer, Division of Policy Information and Coordination, Health 
Resources and Services Administration, 5600 Fishers Lane, Room 14-101, 
Rockville, Maryland 20857.
    Docket: For access to the docket to read background documents or 
comments received, go to the Division of Transplantation, Healthcare 
Systems Bureau, Health Resources and Services Administration, 5600 
Fishers Lane, Room 12C-06, Rockville, Maryland 20857, weekdays (Federal 
holidays excepted) between the hours of 8:30 a.m. and 5 p.m. To 
schedule an appointment to view public comments, phone (301) 443-7757.

FOR FURTHER INFORMATION CONTACT: Shelley Grant, MHSA, at the above 
address; telephone number (301) 443-8036.

SUPPLEMENTARY INFORMATION: 

I. Background

A. Statutory Background

    Congress enacted the National Organ Transplant Act of 1984 (NOTA), 
Public Law 98-507, to develop a national comprehensive policy regarding 
organ transplantation. Within NOTA, section 301 criminalizes the 
transfer of organs for use in human transplantation for ``valuable 
consideration.'' ``Human organ'' is defined to include ``bone marrow * 
* * or any subpart thereof'' or any organ specified by the Secretary in 
regulation. NOTA section 301(c)(1) [codified at 42 U.S.C. 274e(c)(1)]. 
The law criminalizes the transfer of any human organ for valuable 
consideration with a fine of up to $50,000 and imprisonment up to five 
years. Though the general prohibition has been in place since 1984, 
Congress has made numerous amendments to NOTA and otherwise has focused 
recurring attention on organ and bone marrow donation and 
transplantation. In 1988, Congress specifically amended section 301 to 
broaden the definition of ``human organ'' to include ``any subpart 
thereof.'' Organ Transplant Amendments of 1988, Pub. L. 100-607, 
section 407, 102 Stat. 3048, 3116 (Nov 4, 1988). Congress again amended 
section 301 in 2007 to exclude paired donation from the definition of 
``valuable consideration.'' Charlie Norwood Living Organ Donation Act. 
Sec. 102, Public Law 110-144, section 102, 121 Stat. 1813 (2007).
    When enacted, NOTA provided for a demonstration study on the 
feasibility of a ``national registry of voluntary bone marrow donors.'' 
Sec. 401, Public Law 98-507, 98 Stat. 2347 (1984). In 1988, in the same 
law which broadened the definition of organ to ``any subpart thereof,'' 
Congress directed the Secretary to establish a national bone marrow 
registry. Sec. 404, Public Law 100-607, 102 Stat. 3116 (1988). 
Subsequently, Congress established the National Bone Marrow Donor 
Registry. Transplant Amendments Act of 1990. Sec.101, Public Law 101-
616, 104 Stat. 3279 (1990). The National Bone Marrow Donor Registry is 
now called the C.W. Bill Young Cell Transplantation Program, Public Law 
109-129, 119 Stat. 2250 (2005) [42 U.S.C. 274k, et seq.]. Enacted in 
2005, and reauthorized in 2010, the statute defines ``bone marrow'' as 
``the cells found in adult bone marrow and peripheral blood.'' 42 
U.S.C. 274l-1 (emphasis added).

B. Scientific Development

    Hematopoietic stem cells (HSCs) originate in the spongy tissue 
within bones commonly referred to as ``bone marrow'' and give rise to 
mature blood cells, namely red blood cells, white blood cells, and 
platelets. HSCs are found in the highest concentration in bone marrow 
and in lower concentrations in circulating (peripheral) blood. What are 
commonly referred to as ``bone marrow'' transplants are actually 
transplants of hematopoietic stem cells, regardless of source. ``Bone 
marrow'' transplantation (i.e., HSC transplantation) is commonly used 
to treat certain blood cancers like leukemia, other blood diseases like 
aplastic anemia, and immune-deficiency diseases.
    Until recently, available techniques required that HSCs be obtained 
from the marrow by inserting a needle into the marrow to extract liquid 
containing the HSCs. The extracted material is then put through a 
filtration process to separate HSCs from other marrow components and 
concentrate them, before the HSCs are then transplanted into the 
transplant recipient. This type of HSC collection is known as the 
``aspiration method.''
    Under a newer process, known as peripheral blood stem cell 
apheresis, donors receive five daily injections of an HSC stimulating 
drug that causes increased production and mobilization of HSCs from the 
bone marrow into the circulating blood stream (peripheral blood). Once 
these drug doses have been administered, a sufficient quantity and 
concentration of HSCs become available for retrieval in a donor's 
peripheral blood. At this point, a needle is inserted into one of the 
donor's peripheral or central veins, and his or her blood then passes 
through an apheresis machine that isolates and collects the 
hematopoietic stem cells. The remaining blood components are then 
returned to the donor through the intravenous catheter. The apheresis 
collection procedure can take up to eight hours. Most apheresis 
donations occur in one daylong session, although some are completed 
over the course of two days. A donor's total blood volume is run 
through the process three to five times to collect a sufficient number 
of hematopoietic stem cells necessary for successful transplantation. 
The C.W. Bill Young Cell Transplantation Program and its predecessor 
program, the National Bone Marrow Donor Registry, have coordinated 
apheresis donations since 1999. U.S. General

[[Page 60812]]

Accounting Office, Bone Marrow Transplants: Despite Recruitment 
Success, National Program may be Underutilized 6 (2002). Hematopoietic 
stem cells themselves have always been recognized as the critical 
component of bone marrow donation. Thomas' Hematopoietic Cell 
Transplantation 36-37, 72-7, 618 (Frederick Appelbaum, et al., eds. 4th 
ed. 2009).
    Though safer and less invasive than aspiration, apheresis still 
carries risks to the donor. Side-effects of the HSC stimulating drug 
may include rupture of the spleen or a low platelet count 
(thrombocytopenia). There may also be serious risks related to the 
placement of a central venous line in larger veins (jugular, 
subclavian, or femoral) in donors without adequate peripheral vein 
access. More importantly, aspiration is the medically indicated method 
of donation for a substantial number of transplants. American Society 
of Hematology, ``Increased Incidence of Chronic Graft-Versus-Host 
Disease (GVHD) and No Survival Advantage with Filgrastim-Mobilized 
Peripheral Blood Stem Cells (PBSC) Compared to Bone Marrow (BM) 
Transplants From Unrelated Donors: Results of Blood and Marrow 
Transplant Clinical Trials Network (BMT CTN) Protocol 0201, a Phase 
III, Prospective, Randomized Trial,'' Anasetti, Claudio, Confer, 
Dennis, et al., 2011; Biology of Blood and Marrow Transplantation, 
``Peripheral Blood Grafts from Unrelated Donors Are Associated with 
Increased Acute and Chronic Graft-Versus-Host Disease without Improved 
Survival,'' Eapen, Mary, Anasetti, Claudio, et al., 2007. It is 
important to note that, even assuming the relative safety of apheresis, 
a substantial number of potential transplant recipients will continue 
to require HSCs obtained by aspiration.
    Congress has consistently updated the law as advances in organ 
donation technology have been made. As noted above, Congress expanded 
the scope of NOTA's definition of organ in 1988 to include ``any 
subpart thereof.'' In the 2005 Act, Congress defined ``bone marrow'' to 
include HSCs in the ``peripheral blood.'' And, as previously stated, 
Congress expressly granted the Secretary authority to define organs 
through regulation as the field of transplantation evolves.

C. Litigation

    On March 27, 2012, a panel of the United States Ninth Circuit Court 
of Appeals issued an opinion holding that bone marrow donors may be 
compensated if the apheresis method of donation is used. Flynn v. 
Holder, 684 F.3d 852 (9 Cir. 2012). The plaintiffs in the case alleged 
that the ban on sale of ``bone marrow'' under NOTA lacked a ``rational 
basis'' under the equal protection clause of the Fifth Amendment. 
Plaintiffs sought to operate a program offering $3,000 awards, in the 
form of scholarships, housing allowances, or gifts to charity, to bone 
marrow donors. The district court found multiple rational bases for the 
prohibition. However, the Ninth Circuit panel held there was no 
constitutional question since the apheresis method of marrow harvesting 
was not covered by the statutory prohibition on the transfer of organs 
for ``valuable consideration.''
    In rejecting the government's arguments that bone marrow included 
HSCs in the peripheral blood, the Ninth Circuit panel instead focused 
on the recent development of apheresis technology as foreclosing the 
possibility that Congress intended the NOTA, when enacted, to cover 
HSCs in the blood stream. Since apheresis was not used to procure HSCs 
in 1984, the Court held that Congress could not have intended HSCs 
obtained through this method to fall under the ban in section 301. 
Therefore, the Ninth Circuit panel believed that the non-
commodification principle and other negative consequences Congress 
sought to avoid were not relevant to HSCs in the peripheral blood. 
Importantly, however, the Ninth Circuit panel did recognize in its 
written opinion that the Secretary had regulatory authority to define 
peripheral blood stem cells as organs. The effect of exercising this 
authority through this proposed amendment is to clarify that HSCs are 
covered by the prohibition on transfers of human organs for valuable 
consideration found in NOTA section 301(c)(1) [codified at 42 U.S.C. 
274e(c)(1)].
    While focused on the proposal of the plaintiffs before it, the 
Court's holding does not limit the compensation donors can demand to 
scholarships, housing allowances, or charitable gifts. Particularly in 
light of the much more stringent matching required between donor and 
recipient for HSC transplants to be successful, the opportunities for 
exploitation of those in medical need of HSC transplantation are much 
greater than for solid organ transplantation.

II. Proposed Rule

    In light of the Congressional, Departmental, and scientific 
community's long understanding of bone marrow as encompassing HSCs in 
peripheral blood, the Department is proposing to amend the definition 
of ``human organ'' in section 301 to explicitly include HSCs in 
peripheral blood as part of the definition of ``bone marrow'' for the 
purposes for section 301. Notwithstanding the Ninth Circuit's decision 
in the Flynn case, the statute expresses a Congressional intent to ban 
the commodification of HSCs that are used in human transplants, curb 
opportunities for coercion and exploitation, encourage altruistic 
donations, and decrease the likelihood of disease transmission 
resulting from paid donations. Furthermore, the Department has clear 
regulatory authority to clarify the regulatory definition of ``human 
organ'' to make explicit that the prohibition applies to both types of 
collection methods (apheresis and aspiration)--authority that the Ninth 
Circuit expressly recognized.
    For these reasons, the Department is proposing to amend 42 CFR 
121.13 to read: ``Human organ'' as covered by section 301 of the 
National Organ Transplant Act, as amended, means the human (including 
fetal) kidney, liver, heart, lung, pancreas, bone marrow and other 
hematopoietic stem/progenitor cells without regard to the method of 
their collection, cornea, eye, bone, skin, and intestine, including the 
esophagus, stomach, small and/or large intestine, or any portion of the 
gastrointestinal tract.'' The Department has amended, and proposed to 
amend, the definition of ``human organ'' on several occasions, as 
medical knowledge has progressed. See 72 FR 10616 (March 9, 2007) 
(defining prohibition in section 301 to include intestines), and 76 FR 
78216 (December 16, 2011) (proposing to include vascularized composite 
allografts in the definition of ``human organ''). The proposed change 
will clarify that the meaning of ``bone-marrow,'' for purpose of the 
prohibition, does not hinge on the collection method used to obtain the 
cells. The proposed change to the definition of ``human organ'' in 
section 301 does not affect the Food and Drug Administration's 
regulation of whole blood and blood components, and of human cells, 
tissues, and cellular-and tissue-based products (HCT/Ps).

III. Impact Analysis

Executive Order 12866 and Paperwork Reduction Act

Economic and Regulatory Impact
    Executive Order 12866 directs agencies to assess all costs and 
benefits of available regulatory alternatives and, when rulemaking is 
necessary, to select regulatory approaches that provide the greatest 
net benefits (including potential economic, environmental, public 
health, safety, distributive, and equity effects).

[[Page 60813]]

In addition, under the Regulatory Flexibility Act, if a rule has a 
significant economic effect on a substantial number of small entities 
the Secretary must specifically consider the economic effect of a rule 
on small entities and analyze regulatory options that could lessen the 
impact of the rule.
    The Secretary has determined that minimal resources are required to 
implement the requirements in this rule because the organizations 
involved (e.g., marrow registries and transplant hospitals) currently 
implement their programs in accordance with the procedures announced in 
this proposed rule. Therefore, in accordance with the Regulatory 
Flexibility Act of 1980 (RFA), and the Small Business Regulatory 
Enforcement Act of 1996, which amended the RFA, the Secretary certifies 
that this rule will not have a significant impact on a substantial 
number of small entities.
    The Secretary also has determined that this proposed rule does not 
meet the criteria for a major rule as defined by Executive Order 12866 
and would not have a major effect on the economy or Federal 
expenditures. We have determined that the proposed rule is not a major 
rule within the meaning of the statute providing for Congressional 
Review of Agency Rulemaking, 5 U.S.C. 801. Similarly, it will not have 
effects on state, local, and tribal governments or on the private 
sector such as to require consultation under the Unfunded Mandates 
Reform Act of 1995.
    The provisions of this rule will not affect the following elements 
of family well-being: Family safety, family stability, marital 
commitment; parental rights in the education, nurture, and supervision 
of their children; family functioning, disposable income, or poverty; 
or the behavior and personal responsibility of youth, as determined 
under section 654(c) of the Treasury and General Government 
Appropriations Act of 1999.
    Section 202 (a) of the Unfunded Mandates Reform Act of 1995 
requires that agencies assess anticipated costs and benefits before 
issuing any rule that includes a federal mandate that could result in 
expenditure in any one year by state, local, or tribal governments, in 
the aggregate, or by the private sector, of $100 million in 1995 
dollars, updated annually for inflation. The current threshold after 
adjustment for inflation using the Implicit Price Deflator for Gross 
Domestic Product is about $141 million. This rule would not meet or 
exceed that threshold.
    This rule is not economically significant under section 3(f) of 
Executive Order 12866 and is not being treated as a ``significant 
regulatory action'' under section 3(f). Accordingly, the rule has not 
been reviewed by the Office of Management and Budget.
    As stated above, this proposed rule would modify the regulations 
governing the nation's Organ Procurement and Transplantation Network 
(OPTN) and section 301 of NOTA based on legal authority.

 Paperwork Reduction Act of 1995

    The amendments proposed in this Rule will not impose any additional 
data collection requirements beyond those already imposed under the 
current information collection requirements, which have been approved 
by the Office of Management and Budget (OMB No. 0915-0310). The 
currently approved data collection includes worksheets and burden for 
all marrow transplants.

List of Subjects in 42 CFR Part 121

    Healthcare, Hospitals, Organ transplantation.


    Dated: September 19, 2013.
Mary K. Wakefield,
Administrator, Health Resources and Services Administration
    Approved: September 25, 2013.
Kathleen Sebelius,
Secretary.

    Therefore, under the authority of section 301 of NOTA, as amended, 
and for the reasons stated in the preamble, the Department proposes to 
amend 42 CFR part 121 as follows:

PART 121--ORGAN PROCUREMENT AND TRANSPLANTATION NETWORK

0
1. The authority citation continues to read as follows:

    Authority: Sections 215, 371-376 of the Public Health Service 
Act (42 U.S.C. 216, 273-274d); sections 1102, 1106, 1138 and 1871 of 
the Social Security Act (42 U.S.C. 1302, 1306, 1320b-8 and 1395hh); 
and section 301 of the National Organ Transplant Act, as amended (42 
U.S.C. 274e).

0
2. Section 121.13 is revised to read as follows:


Sec.  121.13  Definition of human organ under section 301 of the 
National Organ Transplant Act of 1984, as amended.

    ``Human organ,'' as covered by section 301 of the National Organ 
Transplant Act, as amended, means the human (including fetal) kidney, 
liver, heart, lung, pancreas, bone marrow and other hematopoietic stem/
progenitor cells without regard to the method of their collection, 
cornea, eye, bone skin, and intestine, including the esophagus, 
stomach, small and/or large intestine, or any portion of the 
gastrointestinal tract.''

[FR Doc. 2013-24094 Filed 10-1-13; 8:45 am]
BILLING CODE 4165-15-P