[Congressional Bills 110th Congress]
[From the U.S. Government Publishing Office]
[S. 3046 Introduced in Senate (IS)]







110th CONGRESS
  2d Session
                                S. 3046

   To amend the Federal Food, Drug, and Cosmetic Act to create a new 
conditional approval system for drugs, biological products, and devices 
  that is responsive to the needs of seriously ill patients, and for 
                            other purposes.


_______________________________________________________________________


                   IN THE SENATE OF THE UNITED STATES

                              May 21, 2008

 Mr. Brownback (for himself, Mr. Casey, Mr. Coleman, Mr. Specter, and 
  Mr. Inhofe) introduced the following bill; which was read twice and 
  referred to the Committee on Health, Education, Labor, and Pensions

_______________________________________________________________________

                                 A BILL


 
   To amend the Federal Food, Drug, and Cosmetic Act to create a new 
conditional approval system for drugs, biological products, and devices 
  that is responsive to the needs of seriously ill patients, and for 
                            other purposes.

    Be it enacted by the Senate and House of Representatives of the 
United States of America in Congress assembled,

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Access, Compassion, Care, and Ethics 
for Seriously Ill Patients Act'' or the ``ACCESS Act''.

SEC. 2. FINDINGS.

    Congress finds the following:
            (1) As of 2008, the standards of the Food and Drug 
        Administration for approval of drugs, biological products, and 
        devices may deny the benefits of medical progress to seriously 
        ill patients who face morbidity or death from their disease.
            (2) Seriously ill patients have a right to take actions to 
        preserve their life by accessing available investigational 
        drugs, biological products, and devices.
            (3) The emphasis on statistical analysis of clinical 
        information needs to be balanced by a reliance on clinical 
        evaluation and patient-reported outcomes and considered with an 
        understanding of the risks to patients from their disease, with 
        the goal of providing additional treatment options for patients 
        and their physicians to consider.
            (4) Food and Drug Administration advisory committees should 
        have greater representation of medical clinicians and patient 
        advocates who represent the interests of seriously ill patients 
        in early access to promising investigational therapies.
            (5) The use of available investigational products for 
        treatment is the responsibility of the physician and the 
        seriously ill patient.
            (6) The use of combinations of available investigational 
        and approved products for treatment is the responsibility of 
        the physician and the seriously ill patient.
            (7) The Food and Drug Administration should have the 
        expertise and flexibility to address the growing needs of 
        seriously ill patients for individualized or personalized 
        therapies.

SEC. 3. COMPASSIONATE INVESTIGATIONAL ACCESS APPROVAL SYSTEM FOR DRUGS, 
              BIOLOGICAL PRODUCTS, AND DEVICES.

    (a) Compassionate Investigational Access.--Section 561 of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) is amended--
            (1) by redesignating subsections (d) and (e) as subsections 
        (e) and (f), respectively; and
            (2) by inserting after subsection (c) the following:
    ``(d) Compassionate Investigational Access.--
            ``(1) Purpose.--The purpose of this subsection is to 
        facilitate the availability of promising new drugs to seriously 
        ill patients as early in the drug development process as 
        possible, before general marketing begins.
            ``(2) Access.--Notwithstanding any other provision of law, 
        upon submission by a sponsor of an application intended to 
        provide widespread access to an investigational drug, 
        biological product, or device for eligible patients (referred 
        to in this subsection as `Compassionate Investigational 
        Access'), the Secretary shall permit such investigational drug, 
        biological product, or device, to be made available for 
        expanded access under a treatment investigational new drug 
        application or treatment investigational device exemption if 
        the Secretary determines that the requirements of this section 
        are met with respect to Compassionate Investigational Access.
            ``(3) Compassionate investigational access.--
        Notwithstanding any other provision of law, an investigational 
        drug, biological product, or device that receives approval for 
        Compassionate Investigational Access under this subsection 
        shall be subject to the provisions of section 505(i) or 520(g), 
        as applicable, and regulations promulgated by the Secretary 
        pursuant to this Act. The Secretary and the sponsor may inform 
        national, State, and local medical associations and societies, 
        voluntary health associations, and other appropriate persons 
        about the availability of an investigational drug or 
        investigational device under Compassionate Investigational 
        Access as approved under this subsection. The information 
        submitted by the Secretary, in accordance with the preceding 
        sentence, shall be the same type of information that is 
        required by section 402(i)(3) of the Public Health Service Act.
            ``(4) Submission of application.--
                    ``(A) Application content.--A sponsor of an 
                investigational drug, biological product, or device 
                applying for Compassionate Investigational Access 
                approval of the product shall submit to the Secretary a 
                notice of claimed exemption under section 505(i) or 
                520(g), as applicable, (referred to in this subsection 
                as an `application for Compassionate Investigational 
                Access'), which shall contain--
                            ``(i) data and information from completed 
                        Phase I clinical investigations and any other 
                        nonclinical or clinical investigations;
                            ``(ii) preliminary evidence that the 
                        product may be effective in humans against a 
                        serious or life-threatening condition or 
                        disease, which evidence may be based on 
                        uncontrolled data such as case histories, 
                        information about the pharmacological mechanism 
                        of action, data from animal and computer 
                        models, comparison with historical data, or 
                        other preliminary information, and may be based 
                        on a small number of patients or a subset of 
                        the patient population;
                            ``(iii) evidence that the product is safe 
                        at the dose and duration proposed, considering 
                        whether the potential risk to a patient of the 
                        condition or disease outweighs the potential 
                        risk to a patient of the proposed dose and 
                        duration of treatment with the product, 
                        consistent with the level of information needed 
                        to initiate a Phase II clinical trial; and
                            ``(iv) a statement that the sponsor is 
                        actively pursuing marketing approval with due 
                        diligence.
                    ``(B) Limitation.--Compassionate Investigational 
                Access approval shall be based upon multiple 
                considerations that shall include clinical evaluation 
                and unmet patient needs.
            ``(5) Determination by secretary.--
                    ``(A) In general.--Not later than 30 days after the 
                receipt of an application for Compassionate 
                Investigational Access approval, the Secretary shall 
                either--
                            ``(i) provide Compassionate Investigational 
                        Access approval of the application; or
                            ``(ii) refer the application to the 
                        Accelerated Approval Advisory Committee.
                    ``(B) Recommendation.--Not later than 90 days after 
                receipt of an application for Compassionate 
                Investigational Access approval, the Accelerated 
                Approval Advisory Committee shall issue a 
                recommendation to the Secretary on whether the 
                Secretary shall provide Compassionate Investigational 
                Access approval of the application.
                    ``(C) Final decision.--Not later than 30 days after 
                receipt of the recommendation from the Accelerated 
                Approval Advisory Committee, the Secretary shall either 
                provide Compassionate Investigational Access approval 
                of the application or shall issue an order setting 
                forth a detailed explanation of the reasons why the 
                application was not so approved and the specific data 
                that the sponsor must provide so that the application 
                may be so approved.
            ``(6) Appeal.--If the Secretary does not provide 
        Compassionate Investigational Access approval of an 
        application, the sponsor of the application shall have the 
        right to appeal the decision to the Secretary. The Secretary 
        shall provide the sponsor with a hearing not later than 30 days 
        following the nonapproval under this subsection of the 
        application and shall issue an order not later than 30 days 
        following the hearing either concurring in the nonapproval or 
        so approving the application. The Secretary shall not delegate 
        the responsibility described in this paragraph to any other 
        person.
            ``(7) Criteria.--In making a determination under paragraph 
        (5), the Secretary shall consider whether the totality of the 
        information available to the Secretary regarding the safety and 
        effectiveness of an investigational drug, biological product, 
        or device, as compared to the risk of morbidity or death from a 
        condition or disease, indicates that a patient (who may be 
        representative of a small patient subpopulation) may obtain 
        more benefit than risk if treated with the drug, biological 
        product, or device. If the potential risk to a patient of the 
        condition or disease outweighs the potential risk of the 
        product, and the product may possibly provide benefit to the 
        patient, the Secretary shall provide Compassionate 
        Investigational Access approval of the application.
            ``(8) Patient eligibility for compassionate access.--In 
        order for a patient to access a product available through 
        Compassionate Investigational Access, the physician must 
        document in writing that the patient--
                    ``(A) is seriously ill;
                    ``(B) has exhausted all treatment options approved 
                by the Secretary for the condition or disease for which 
                the patient is a reasonable candidate; and
                    ``(C) has unsuccessfully sought treatment or 
                obtained treatment that was not effective, with an 
                investigational drug, biological product, or device for 
                which such individual is a reasonable candidate, which 
                shall include consideration of a patient's 
                ineligibility for participation in clinical trials, the 
                lack of source of supply and geographic factors.
            ``(9) Product labeling.--To receive Compassionate 
        Investigational Access approval under this subsection, the 
        sponsor of the product shall provide labeling approved by the 
        Secretary for the drug, biological product, or device that--
                    ``(A) states that the product is intended for use 
                by a patient whose physician has documented in writing 
                that the patient has--
                            ``(i) exhausted all treatment options 
                        approved by Secretary for the condition or 
                        disease for which the patient is a reasonable 
                        candidate; and
                            ``(ii) unsuccessfully sought treatment, or 
                        obtained treatment that was not effective with 
                        an investigational drug, biological product, or 
                        device for which such individual is a 
                        reasonable candidate, which shall include a 
                        patient's ineligibility for participation in 
                        clinical trials, the lack of source of supply 
                        and geographic factors; and
                    ``(B) states that every patient to whom the product 
                is administered shall, as a mandatory condition of 
                receiving the product, provide--
                            ``(i) written informed consent, as 
                        described under part 50 of title 21, Code of 
                        Federal Regulations (or any successor 
                        regulations); and
                            ``(ii) consent for the manufacturer of the 
                        product to obtain data and information about 
                        the patient and the patient's use of the 
                        product that may be used to support an 
                        application for Accelerated Approval or final 
                        approval.
            ``(10) Charging for compassionate investigational access.--
        A sponsor or investigator may charge for a Compassionate 
        Investigational Access drug without notifying the Secretary or 
        seeking or obtaining prior approval of the amount charged, 
        provided the sponsor of the drug is actively pursuing marketing 
        approval with due diligence.
            ``(11) Commencement of review.--If the Secretary 
        determines, after preliminary evaluation of the data and 
        information submitted by the sponsor, that the product may be 
        effective, the Secretary shall evaluate for filing, and may 
        commence review of portions of, an application under this 
        subsection before the sponsor submits a complete application. 
        The Secretary shall commence such review only if the applicant 
        provides a schedule for submission of information necessary to 
        make the application complete.
            ``(12) Immunity.--
                    ``(A) In general.--A manufacturer, distributor, 
                administrator, sponsor, or physician who manufactures, 
                supplies, distributes or prescribes a product approved 
                under an application for Compassionate Investigational 
                Access shall be immune from suit or liability caused 
                by, arising out of, or relating to the design, 
                development, clinical testing and investigation, 
                manufacture, labeling, distribution, sale, purchase, 
                donation, dispensing, prescribing, administration, 
                efficacy, or use of a drug, biological product, or 
                device subject to an approved Compassionate 
                Investigational Access application.
                    ``(B) Claims.--No claim or cause of action against 
                a manufacturer, distributor, administrator, sponsor, or 
                physician who manufactures, supplies, distributes or 
                prescribes a product subject to an approved 
                Compassionate Investigational Access application shall 
                exist in any Federal or State court for claims of 
                property, personal injury, or death caused by, arising 
                out of, or relating to the design, development, 
                clinical testing and investigation, manufacture, 
                labeling, distribution, sale, purchase, donation, 
                dispensing, prescribing, administration, efficacy, or 
                use of a drug, biological product, or device subject to 
                an approved Compassionate Investigational Access 
                application. Any such claim or cause of action that is 
                filed in Federal or State court shall be immediately 
                dismissed.
            ``(13) Final approval.--For purposes of this Act, the term 
        `final approval' means--
                    ``(A) with respect to a new drug or new biological 
                product, approval of such drug or product under section 
                505(b)(1) or 505(b)(2) or section 351 of the Public 
                Health Service Act, as the case may be; and
                    ``(B) with respect to a new device, clearance of 
                such device under section 510(k) or approval of such 
                device under section 515(c)(1).''.
    (b) Accelerated Approval.--Chapter V of the Federal, Food, Drug, 
and Cosmetic Act (21 U.S.C. 351 et seq.) is amended by inserting after 
section 561 the following:

``SEC. 561A. ACCELERATED APPROVAL.

    ``(a) In General.--
            ``(1) In general.--As soon as practicable after the date of 
        enactment of the Access, Compassion, Care, and Ethics for 
        Seriously Ill Patients Act, the Secretary shall promulgate 
        regulations to provide for the treatment of an investigational 
        drug, biological product, or device that receives Accelerated 
        Approval under this section. This section shall be carried out 
        in accordance with such regulations (and any successor 
        regulations).
            ``(2) Application.--A sponsor of an investigational drug, 
        biological product, or device applying for Accelerated Approval 
        shall submit to the Secretary an application as described under 
        section 505(b)(1) or 505(b)(2), or section 510(k) or 515(c)(1) 
        of this Act, or section 351(a) of the Public Health Service 
        Act, as applicable, which shall contain--
                    ``(A) data and information that the drug, 
                biological product, or device has an effect on a 
                clinical endpoint or on a surrogate endpoint or 
                biomarker that is reasonably likely to predict clinical 
                benefit to a patient (who may be representative of a 
                small patient subpopulation) suffering from a serious 
                or life-threatening condition or disease; and
                    ``(B) a statement that the sponsor is actively 
                pursuing marketing approval with due diligence.
            ``(3) Determination by secretary.--
                    ``(A) In general.--Not later than 120 days after 
                the receipt of an application for Accelerated Approval, 
                the Secretary shall either--
                            ``(i) provide Accelerated Approval of the 
                        application; or
                            ``(ii) refer the application to the 
                        Accelerated Approval Advisory Committee.
                    ``(B) Recommendation.--Not later than 90 days after 
                receipt of an application for Accelerated Approval, the 
                Accelerated Approval Advisory Committee shall issue a 
                recommendation to the Secretary on whether the 
                Secretary should provide Accelerated Approval of the 
                application.
                    ``(C) Limitation.--The Accelerated Approval 
                Advisory Committee shall not consider off-label uses of 
                drugs, biological products, and devices as existing or 
                available therapies, to the extent that the Accelerated 
                Approval Advisory Committee weighs existing or 
                available therapies in determination of whether an 
                investigational drug provides an improvement over 
                treatments that are already available.
                    ``(D) Final decision.--Not later than 30 days after 
                receipt of the recommendation from the Accelerated 
                Approval Advisory Committee, the Secretary shall either 
                provide Accelerated Approval of the application or 
                issue an order setting forth a detailed explanation of 
                the reasons why the application was not so approved and 
                the specific data that the sponsor must provide so that 
                the application may be so approved.
            ``(4) Appeal.--If the Secretary does not provide 
        Accelerated Approval of an application, the sponsor of the 
        application shall have the right to appeal the decision to the 
        Secretary. The Secretary shall provide the sponsor with a 
        hearing not later than 30 days following the nonapproval under 
        this subsection of the application and shall issue an order not 
        later than 30 days following the hearing either concurring in 
        such nonapproval or so approving the application. The Secretary 
        shall not delegate the responsibility described in this 
        paragraph to any other person.
                    ``(A) with respect to a new drug or new biological 
                product, approval of such drug or product under section 
                505(b)(1) or 505(b)(2) or section 351 of the Public 
                Health Service Act, as the case may be; and
                    ``(B) with respect to a new device, clearance of 
                such device under section 510(k) or approval of such 
                device under section 515(c)(1).
    ``(b) Accelerated Approval Advisory Committee.--
            ``(1) In general.--In order to facilitate the development 
        and expedite the review of drugs, biological products, and 
        devices intended to treat serious or life threatening 
        conditions, the Secretary shall establish the Accelerated 
        Approval Advisory Committee (referred to in this subsection as 
        the `Committee').
            ``(2) Delegation.--The Secretary may delegate 
        decisionmaking authority for the Accelerated Approval Advisory 
        Committee to the Office of the Commissioner of Food and Drugs. 
        Such authority shall not be further delegated.
            ``(3) Composition.--
                    ``(A) In general.--The Committee shall be composed 
                of 11 voting members, including 1 chairperson and 5 
                permanent members each of whom shall serve a term of 3 
                years and may be reappointed for a second 3-year term, 
                and 5 nonpermanent members who shall be appointed to 
                the Committee for a specific meeting, or part of a 
                meeting, in order to provide adequate expertise in the 
                subject being reviewed. The Committee shall include as 
                voting members no less than 2 representatives of 
                patient interests, of which 1 shall be a permanent 
                member of the Committee. The Committee shall include as 
                nonvoting members a representative of interests of the 
                drug, biological product, and device industry.
                    ``(B) Appointments.--The Secretary shall appoint to 
                the Committee persons who are qualified by training and 
                experience to evaluate the safety and effectiveness of 
                the types of products to be referred to the Committee 
                and who, to the extent feasible, possess skill in the 
                use of, or experience in the development, manufacture, 
                or utilization of, such products. The Secretary shall 
                make appointments to the Committee so that the 
                Committee shall consist of members with adequately 
                diversified expertise and practical experience in such 
                fields as clinical medicine, biological and physical 
                sciences, and other related professions. Scientific, 
                industry, and consumer organizations and members of the 
                public shall be afforded an opportunity to nominate 
                individuals for appointment to the Committee. No 
                individual who is in the regular full-time employ of 
                the United States and engaged in the administration of 
                this chapter may be a member of the Committee.
            ``(4) Compensation.--Committee members, while attending 
        meetings or conferences of the Committee or otherwise engaged 
        in its business, shall be entitled to receive compensation at 
        rates to be fixed by the Secretary, but not at rates exceeding 
        the daily equivalent of the rate in effect for grade GS-18 of 
        the General Schedule, for each day so engaged, including 
        traveltime, and while so serving away from their homes or 
        regular places of business each member may be allowed travel 
        expenses (including per diem in lieu of subsistence) as 
        authorized by section 5703 of title 5, for persons in the 
        Government service employed intermittently.
            ``(5) Assistance.--The Secretary shall furnish the 
        Committee with adequate clerical and other necessary 
        assistance.
            ``(6) Annual training.--The Secretary shall employ 
        nongovernmental experts to provide annual training to the 
        Committee on the statutory and regulatory standards for product 
        approval.
            ``(7) Timeline.--The Committee shall be scheduled to meet 
        at such times as may be appropriate for the Secretary to meet 
        applicable statutory deadlines.
            ``(8) Meetings.--
                    ``(A) Opportunities for interested persons.--Any 
                person whose product is specifically the subject of 
                review by the Committee shall have--
                            ``(i) the same access to data and 
                        information submitted to the Committee as the 
                        Secretary;
                            ``(ii) the opportunity to submit, for 
                        review by the Committee, data or information, 
                        which shall be submitted to the Secretary for 
                        prompt transmittal to the Committee;
                            ``(iii) the same opportunity as the 
                        Secretary to participate in meetings of the 
                        Committee; and
                            ``(iv) consent for the manufacturer of the 
                        product to obtain data about adverse events 
                        relating to the patient's use of the product.
                    ``(B) Adequate time; free and open participation.--
                Any meetings of the Committee shall provide adequate 
                time for initial presentations and for response to any 
                differing views by persons whose products are 
                specifically the subject of the Committee review.
                    ``(C) Summaries.--At all meetings of the Committee, 
                the Secretary shall provide a summary to the Committee 
                of all applications submitted under this subsection and 
                section 561(d) that the Committee did not consider that 
                were approved by the Secretary since the last meeting 
                of the Committee.
    ``(c) Final Approval.--For purposes of this Act, the term `final 
approval' means--
            ``(1) with respect to a new drug or new biological product, 
        approval of such drug or product under section 505(b)(1) or 
        505(b)(2) or section 351 of the Public Health Service Act, as 
        the case may be; and
            ``(2) with respect to a new device, clearance of such 
        device under section 510(k) or approval of such device under 
        section 515(c)(1).''.
    (c) Regulations.--The Secretary of Health and Human Services shall 
promulgate regulations that define the terms ``seriously ill'' and 
``serious or life-threatening'' for purposes of the amendments made by 
this Act, considering either--
            (1) the medical prognosis for an individual's life 
        expectancy from a disease or condition; or
            (2) the prospect of irreversible disability from a disease 
        or condition.

SEC. 4. EXPANDED ACCESS TO INVESTIGATIONAL DRUGS AND DEVICES.

    Chapter V of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
351 et seq.) is amended by inserting after section 561A, as added by 
section 3, the following:

``SEC. 561B. EXPANDED ACCESS TO INVESTIGATIONAL DRUGS AND DEVICES.

    ``(a) Expanded Access Program.--The Secretary shall establish a new 
program to expand access to investigational treatments for individuals 
with serious or life threatening conditions and diseases. In carrying 
out this expanded access program, the Secretary shall publish and 
broadly disseminate written guidance that--
            ``(1) describes such expanded access programs for 
        investigational drugs, biological products, and devices 
        intended to treat serious or life-threatening conditions or 
        diseases;
            ``(2) encourages and facilitates submission of applications 
        and approvals under section 561(d) and 561A; and
            ``(3) facilitates the provision of investigational drugs, 
        biological products, and devices to seriously ill individuals 
        without unreasonable delay by recognizing that the use of 
        available investigational products for treatment is the 
        responsibility of the physician and the patient, and also by 
        recognizing the goal of providing additional treatment options 
        for patients and their physicians to consider.
    ``(b) Implementation of Expanded Access Programs.--
            ``(1) Training of personnel.--Not later than 90 days after 
        the date of enactment of this section, the Secretary shall 
        implement training programs at the Food and Drug Administration 
        with respect to the expanded access programs established under 
        this section.
            ``(2) Policies, regulations, and guidance.--The Secretary 
        shall establish policies, regulations, and guidance designed to 
        most directly benefit seriously ill patients.
    ``(c) Development of Surrogate Endpoints and Biomarkers.--
            ``(1) In general.--The Secretary shall--
                    ``(A) establish a program or expand upon an 
                existing program to encourage the development of 
                surrogate endpoints and biomarkers that are reasonably 
                likely to predict clinical benefit for serious or life-
                threatening conditions for which there exist 
                significant unmet patient needs;
                    ``(B) request the Institute of Medicine to 
                undertake a study to identify validated surrogate 
                endpoints and biomarkers, and recommend research to 
                validate surrogate endpoints and biomarkers, that may 
                support approvals for products intended for the 
                treatment of serious or life-threatening conditions or 
                diseases; and
                    ``(C) make widely available to the public a list of 
                drugs, biological products, and devices that are being 
                investigated for serious or life-threatening conditions 
                or diseases and that have not yet received approval 
                under section 561(d) or 561A for marketing.
            ``(2) Study content.--The study under paragraph (1)(B) 
        shall include endpoints and biomarkers that address the unmet 
        medical needs of subpopulations of patients and that facilitate 
        the development of individualized treatment approaches for 
        patients with serious or life-threatening conditions or 
        diseases.''.

SEC. 5. DEMONSTRATION PROJECT FOR COVERAGE OF CERTAIN DRUGS, BIOLOGICAL 
              PRODUCTS, AND DEVICES UNDER THE MEDICARE PROGRAM.

    (a) In General.--The Secretary of Health and Human Services (in 
this section referred to as the ``Secretary'') shall establish a 
demonstration project under the Medicare program under title XVIII of 
the Social Security Act (42 U.S.C. 1395 et seq.) under which payment is 
made for drugs, biological products, and devices approved for 
Compassionate Investigational Access under section 561(d) of the 
Federal Food, Drug, and Cosmetic Act in the case where the drug, 
biological product, or device is not otherwise covered under the 
Medicare program or by any other organization or entity (including a 
public assistance program or the sponsor of the application for such 
drug, biological product, or device).
    (b) Duration.--The demonstration project under this section shall 
be conducted for a 5-year period.
    (c) Funding.--
            (1) In general.--The Secretary shall provide for the 
        transfer from the Federal Hospital Insurance Trust Fund under 
        section 1817 of the Social Security Act (42 U.S.C. 1395i) and 
        the Federal Supplementary Medical Insurance Trust Fund under 
        section 1841 of such Act (42 U.S.C. 1395t), in such proportion 
        as the Secretary determines to be appropriate, of such sums as 
        are necessary for the costs of carrying out the demonstration 
        project under this section.
            (2) Budget neutrality.--In conducting the demonstration 
        project under this section, the Secretary shall ensure that the 
        aggregate payments made by the Secretary do not exceed the 
        amount which the Secretary estimates would have been paid if 
        the demonstration project under this section was not 
        implemented.
    (d) Waiver Authority.--The Secretary may waive such requirements of 
title XVIII of the Social Security Act (42 U.S.C. 1395 et seq.) as may 
be necessary for the purpose of carrying out the demonstration project 
under this section.
    (e) Report.--Not later than 90 days after the last day of the 5-
year period of the demonstration project under this section, the 
Secretary shall submit to Congress a report describing the rates of 
utilization by Medicare beneficiaries of drugs, biological products, 
and devices approved for Compassionate Investigational Access and the 
total cost of payments made under the Medicare program resulting from 
the demonstration project. The report shall describe recommendations 
for legislation or administrative action as the Secretary deems 
appropriate.
    (f) Termination.--The Secretary shall terminate payments under this 
section on the day after the last day of the 5-year period of the 
demonstration project under this section.

SEC. 6. USE OF PART B DEFINITION OF MEDICALLY ACCEPTED INDICATION FOR 
              PART D DRUGS.

    (a) In General.--Section 1860D-2(e) of the Social Security Act (42 
U.S.C. 1395w-102(e)) is amended--
            (1) in paragraph (1), in the matter following subparagraph 
        (B), by striking ``(as defined in section 1927(k)(6))'' and 
        inserting ``(as defined in paragraph (4))''; and
            (2) by adding at the end the following new paragraph:
            ``(4) Medically accepted indication defined.--
                    ``(A) In general.--Subject to subparagraph (B), for 
                purposes of paragraph (1), the term `medically accepted 
                indication' has the meaning given that term--
                            ``(i) in the case of a covered part D drug 
                        used in an anticancer chemotherapeutic regimen, 
                        in section 1861(t)(2)(B), except that in 
                        applying such section, `PDP sponsor or MA-PD 
                        sponsor' shall be substituted for `carrier' 
                        each place it appears and the compendia 
                        identified in section 1927(g)(1)(B)(i)(III) 
                        shall be deemed to be included in the compendia 
                        described in section 1861(t)(2)(B)(ii)(I); and
                            ``(ii) in the case of any other covered 
                        part D drug, in section 1927(k)(6).
                    ``(B) Consideration of other criteria on a case-by-
                case basis.--Nothing in this subsection shall preclude 
                a PDP sponsor offering a prescription drug plan or an 
                MA organization offering an MA-PD plan from, after a 
                request by an individual enrolled in the plan for a 
                coverage determination under section 1860D-4(g)(1), 
                providing coverage of a covered part D drug for the 
                individual in the case where the sponsor or 
                organization determines, based on guidance provided by 
                the Secretary for determining whether the use of a 
                covered part D drug is for a medically accepted 
                indication and supportive clinical evidence in peer 
                reviewed medical literature, that the use of the 
                covered part D drug is for a medically accepted 
                indication.''.
    (b) Effective Date.--The amendments made by this section shall 
apply to plan years beginning on or after January 1, 2010.

SEC. 7. MODERNIZATION OF THE FOOD AND DRUG ADMINISTRATION.

    Subchapter E of chapter V of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 360bbb et seq.) is amended by adding at the end the 
following:

``SEC. 568. POLICIES RELATED TO STUDY EVALUATION INFORMATION.

    ``(a) In General.--
            ``(1) Nonstatistical measures.--The Secretary shall give 
        consideration to clinical judgment and risks to the patient 
        from the disease or condition involved in the evaluation of the 
        safety and effectiveness of drugs, biological products, and 
        devices that treat serious or life-threatening diseases or 
        conditions. This policy shall apply--
                    ``(A) in evaluating clinical study designs and 
                endpoints; and
                    ``(B) in making decisions with respect to product 
                applications for approval under section 561(d) or 561A.
            ``(2) Types of nonstatistical measures.--The policy 
        established under paragraph (1), for the purposes described in 
        such paragraph--
                    ``(A) shall include such nonstatistical information 
                as--
                            ``(i) clinical evaluation information, such 
                        as case history reports;
                            ``(ii) scientific and clinical studies 
                        designed to measure or define mechanisms of 
                        action or molecular targeting;
                            ``(iii) data from animal and computer 
                        models; and
                            ``(iv) comparison with historical data; and
                    ``(B) shall incorporate the use of--
                            ``(i) evaluations of the adverse effect of 
                        delaying the availability of an investigational 
                        drug to even a small subpopulation of seriously 
                        ill patients; and
                            ``(ii) scientific, observational, or 
                        clinical studies designed and conducted to 
                        collect well-documented information.
    ``(b) Meetings.--A meeting to address any pending scientific, 
medical, regulatory, or other issue relating to the development, 
investigation, review, or other aspect of a drug, biological product, 
or device shall ordinarily be held not later than 15 days of the 
receipt of a written request for the meeting by the sponsor of the 
product, which may be extended to 30 days for good cause. Such meetings 
shall ordinarily be conducted in person, but may be conducted by 
telephone or other form of communication if both parties agree. In 
order to reduce the burden of meetings, only those Food and Drug 
Administration employees who are intended to actively participate in 
the discussion shall attend a meeting. Minutes of a meeting shall be 
promptly prepared and exchanged by both parties immediately following 
the meeting and shall accurately summarize what occurred at the 
meeting.
    ``(c) Rule of Construction.--The provisions of this chapter and 
section 351 of the Public Health Service Act shall be construed to 
incorporate the policy established in this section.''.

SEC. 8. MEMBERSHIP OF ONCOLOGY DRUGS ADVISORY COMMITTEE AND THE 
              CELLULAR, TISSUE, AND GENE THERAPY ADVISORY COMMITTEE.

    Notwithstanding any other provision of law, membership of the 
Oncology Drugs Advisory Committee, the Cellular, Tissue, and Gene 
Therapy Advisory Committee of the Food and Drug Administration, and any 
other committee created by such Administration to evaluate or advise 
with respect to applications submitted under section 561(d) or 561A of 
the Federal Food, Drug, and Cosmetic Act (as added by this Act), shall 
consist of no less than 2 patient representatives who are voting 
members of the committee.
                                 <all>