[Congressional Bills 109th Congress]
[From the U.S. Government Publishing Office]
[H.R. 6303 Introduced in House (IH)]








109th CONGRESS
  2d Session
                                H. R. 6303

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


_______________________________________________________________________


                    IN THE HOUSE OF REPRESENTATIVES

                           September 29, 2006

  Mr. Shays introduced the following bill; which was referred to the 
                    Committee on Energy and Commerce

_______________________________________________________________________

                                 A BILL


 
   To amend the Federal Food, Drug, and Cosmetic Act to create a new 
   three-tiered 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) The necessity of placebo controlled studies has been 
        questioned on both scientific and ethical grounds for seriously 
        ill patients.
            (2) The current standards of the Food and Drug 
        Administration for approval of drugs, biological products, and 
        devices deny the benefits of medical progress to seriously ill 
        patients who face morbidity or death from their disease.
            (3) Promising therapies intended to treat serious or life 
        threatening conditions or diseases and which address unmet 
        medical needs have received unjustified delays and denials of 
        approval.
            (4) Seriously ill patients have a right to access available 
        investigational drugs, biological products, and devices.
            (5) The current Food and Drug Administration and National 
        Cancer Institute case-by-case exception for compassionate 
        access must be required to permit all seriously ill patients 
        access to available experimental therapies as a treatment 
        option.
            (6) The current emphasis on statistical analysis of 
        clinical information needs to be balanced by a greater reliance 
        on clinical evaluation of this information.
            (7) Food and Drug Administration advisory committees should 
        have greater representation of medical clinicians who represent 
        the interests of seriously ill patients in early access to 
        promising investigational therapies.
            (8) The use of available investigational products for 
        treatment is the responsibility of the physician and the 
        patient.
            (9) The use of combinations of available investigational 
        and approved products for treatment is the responsibility of 
        the physician and the patient.
            (10) The development and approval of drugs, biological 
        products, and devices intended to address serious or life-
        threatening conditions or diseases is often delayed by the 
        inability of sponsors to obtain prompt meetings with the Food 
        and Drug Administration and to obtain prompt resolution of 
        scientific and regulatory issues related to the investigation 
        and review of new technologies.

SEC. 3. TIERED APPROVAL SYSTEM FOR DRUGS, BIOLOGICAL PRODUCTS, AND 
              DEVICES.

    Section 506 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
356) is amended to read as follows:

``SEC. 506. TIERED APPROVAL SYSTEM.

    ``(a) In General.--Notwithstanding any other provision of law, the 
sponsor of an investigational drug, biological product, or device may 
submit an application to the Secretary for Tier I or Tier II approval 
in accordance with this section.
    ``(b) Tier I Approval.--
            ``(1) In general.--
                    ``(A) Application content.--A sponsor of an 
                investigational drug, biological product, or device 
                applying for Tier I approval of the product shall 
                submit to the Secretary an application as described 
                under section 505(b)(1) or 505(b)(2), section 351(a) of 
                the Public Health Service Act, or section 510(k) or 
                515(c)(1), as applicable, 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 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; and
                            ``(iii) an assurance that the sponsor will 
                        continue clinical investigation to obtain Tier 
                        III approval.
                    ``(B) Limitation.--Tier I approval shall be 
                primarily based upon clinical evaluation, not 
                statistical analysis.
            ``(2) Determination by secretary.--
                    ``(A) In general.--Not later than 30 days after the 
                receipt of an application for Tier I approval, the 
                Secretary shall either--
                            ``(i) approve the application; or
                            ``(ii) refer the application to the 
                        Accelerated Approval Advisory Committee.
                    ``(B) Recommendation.--Within 90 days after receipt 
                of an application for approval, the Accelerated 
                Approval Advisory Committee shall issue a 
                recommendation to the Secretary on whether the 
                Secretary should approve the application.
                    ``(C) Final decision.--Within 30 days after receipt 
                of the recommendation from the Accelerated Approval 
                Advisory Committee, the Secretary shall either approve 
                the application or shall issue an order setting forth a 
                detailed explanation of the reasons why the application 
                was not approved and the specific data that the sponsor 
                must provide so that the application may be approved.
            ``(3) Appeal.--If the Secretary does not approve an 
        application for which the Accelerated Approval Advisory 
        Committee recommended approval, the sponsor of the application 
        shall have the right to appeal the decision to the Commissioner 
        of Food and Drugs. The Commissioner shall provide the sponsor 
        with a hearing within 30 days following the nonapproval of the 
        application and shall issue an order within 30 days following 
        the hearing either concurring in the nonapproval or approving 
        the application. The Commissioner shall not delegate the 
        responsibility described in this paragraph to any other person.
            ``(4) Criteria.--In making a determination under paragraph 
        (2), 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 approve the application.
            ``(5) Product labeling.--The labeling approved by the 
        Secretary for the drug, biological product, or device--
                    ``(A) shall state 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 may include 
                        consideration of the lack of a source of supply 
                        or geographic factors); and
                    ``(B) shall state 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;
                            ``(ii) a written waiver of the right to sue 
                        the manufacturer or sponsor of the drug, 
                        biological product, or device, or the 
                        physicians who prescribed the product or the 
                        institution where it was administered, for an 
                        adverse event caused by the product, which 
                        shall be binding in every State and Federal 
                        court; and
                            ``(iii) 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 Tier II or Tier III approval.
            ``(6) Limitation on conditions.--Tier I approval may be 
        subject to the requirement that the sponsor conduct appropriate 
        post-approval studies.
    ``(c) Tier II Approval.--
            ``(1) In general.--A sponsor of an investigational drug, 
        biological product, or device applying for Tier II approval 
        shall submit to the Secretary an application as described under 
        section 505(b)(1) or 505(b)(2), section 351(a) of the Public 
        Health Service Act, or section 510(k) or 515(c)(1), 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) an assurance that the sponsor will continue 
                clinical investigation to obtain Tier III approval.
            ``(2) Determination by secretary.--
                    ``(A) In general.--Not later than 30 days after the 
                receipt of an application for Tier II approval, the 
                Secretary shall either--
                            ``(i) approve the application; or
                            ``(ii) refer the application to the 
                        Accelerated Approval Advisory Committee.
                    ``(B) Recommendation.--Within 90 days after receipt 
                of an application for approval, the Accelerated 
                Approval Advisory Committee shall issue a 
                recommendation to the Secretary on whether the 
                Secretary should approve the application.
                    ``(C) Final decision.--Within 30 days after receipt 
                of the recommendation from the Accelerated Approval 
                Advisory Committee, the Secretary shall either approve 
                the application or issue an order setting forth a 
                detailed explanation of the reasons why the application 
                was not approved and the specific data that the sponsor 
                must provide so that the application may be approved.
            ``(3) Appeal.--If the Secretary does not approve an 
        application for which the Accelerated Approval Advisory 
        Committee recommended approval, the sponsor of the application 
        shall have the right to appeal the decision to the Commissioner 
        of Food and Drugs. The Commissioner shall provide the sponsor 
        with a hearing within 30 days following the nonapproval of the 
        application and shall issue an order within 30 days following 
        the hearing either concurring in the nonapproval or approving 
        the application. The Commissioner shall not delegate the 
        responsibility described in this paragraph to any other person.
            ``(4) Limitation on conditions.--
                    ``(A) Post-approval studies.--Tier II approval may 
                be subject to the requirement that the sponsor conduct 
                appropriate post-approval studies to validate the 
                surrogate endpoint or biomarker or otherwise confirm 
                the effect on the clinical endpoint.
                    ``(B) Rule of construction.--Nothing in this 
                subsection shall be construed to permit the Secretary 
                to condition Tier II approval on compliance with any 
                other standards, including any standard necessary to 
                meet Tier III approval.
    ``(d) Tier III Approval.--For purposes of this Act, the term `Tier 
III 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).
    ``(e) Promotional Materials.--Approval of a product under either 
Tier I or II may be subject to the requirements that--
            ``(1) the sponsor submit copies of all advertising and 
        promotional materials related to the product during the 
        preapproval review period and, following approval and for such 
        period thereafter as the Secretary determines to be 
        appropriate, and at least 30 days prior to the dissemination of 
        the materials;
            ``(2) all advertising and promotional materials prominently 
        disclose the limited approval for the product and data 
        available supporting the safety and effectiveness of the 
        product; and
            ``(3) the sponsor shall not disseminate advertising or 
        promotional material prior to obtaining written notification 
        from the Secretary that the advertising or promotional material 
        complies with this subchapter.
    ``(f) Expedited Withdrawal of Approval.--The Secretary may withdraw 
Tier I or Tier II approval using expedited procedures (as prescribed by 
the Secretary in regulations which shall include an opportunity for a 
hearing) if--
            ``(1) the sponsor fails to conduct post-approval studies 
        with due diligence, considering all of the circumstances 
        involved;
            ``(2) a post-approval study fails to verify clinical 
        benefit of the product for even a small patient subpopulation;
            ``(3) other evidence demonstrates that the product is not 
        safe or effective under the conditions of use for even a small 
        patient subpopulation; or
            ``(4) the sponsor disseminates false or misleading 
        promotional materials with respect to the product and fails to 
        correct the material promptly after written notice from the 
        Secretary.
    ``(g) 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.
            ``(2) Delegation.--The Secretary may delegate authority for 
        the Accelerated Approval Advisory Committee to the Commissioner 
        of Food and Drugs. The Accelerated Approval Advisory Committee 
        shall be staffed and administered in the Office of the 
        Commissioner.
            ``(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; and
                            ``(iii) the same opportunity as the 
                        Secretary to participate in meetings of the 
                        Committee.
                    ``(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, and 
                shall encourage free and open participation by all 
                interested persons.
                    ``(C) Summaries.--At all meetings of the Committee, 
                the Secretary shall provide a summary to the Committee 
                of all Tier I and Tier II applications that the 
                Committee did not consider that were approved by the 
                Secretary since the last meeting of the Committee.
    ``(h) 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 for Tier I or Tier II approval 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.
    ``(i) Inapplicability of Provisions.--The following provisions 
shall not apply to Tier I or Tier II applications and approvals:
            ``(1) Chapter VII, subchapter C, parts 2 and 3 relating to 
        fees for drugs, biological products, and devices.
            ``(2) The provisions of the Drug Price Competition and 
        Patent Term Restoration Act of 1984 that authorize approval of 
        abbreviated new drug applications and applications submitted 
        under section 505(b)(2). Market exclusivity and patent term 
        restoration of Tier I and Tier II approved drugs, biological 
        products, and devices shall be determined solely at the time of 
        Tier III approval without regard to prior Tier I or Tier II 
        approval. Prior to Tier III approval, the Secretary shall not 
        approve any application submitted under section 505(b)(2) or 
        section 505(j) that references a drug approved under 
        subsections (b) or (c) of this section.''.

SEC. 4. ETHICS IN HUMAN TESTING.

    Chapter V of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
351 et seq.) is amended by adding at the end of section 505(i) the 
following:
            ``(5) Notwithstanding any other provision of law, the 
        Secretary shall prohibit placebo-only or no-treatment-only 
        concurrent controls in any clinical investigation conducted 
        under this chapter or, in the use of the last-observation-
        carried-forward convention, in any clinical investigation 
        conducted under this chapter or section 351 of the Public 
        Health Service Act with respect to any life-threatening 
        condition or disease where reasonably effective approved 
        alternative therapies exist for the specific indication.''.

SEC. 5. EXPANDED ACCESS TO INVESTIGATIONAL DRUGS AND DEVICES.

    (a) In General.--Chapter V of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 351 et seq.) is amended by adding at the end of section 
561 the following:
    ``(f) Expanded Access Program.--The Food and Drug Administration 
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 Tier I and 
        Tier II applications and approvals; and
            ``(3) facilitates the provision of investigational drugs 
        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.
    ``(g) Implementation of Expanded Access Programs.--
            ``(1) Training of personnel.--Not later than 90 days after 
        the date of enactment of this subsection, 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.
    ``(h) Development of Surrogate Endpoints and Biomarkers.--The 
Secretary shall--
            ``(1) establish a 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 medical 
        needs;
            ``(2) 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
            ``(3) 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 Tier I or Tier II approval for 
        marketing.''.
    (b) Conforming Amendment.--Section 561(c) of the Federal Food, 
Drug, and Cosmetic Act is amended by striking the heading and inserting 
``Expanded Access to Investigational Drugs and Devices for Seriously 
Ill Patients''.

SEC. 6. 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. 565. POLICIES RELATED TO STUDY EVALUATION INFORMATION.

    ``(a) In General.--
            ``(1) Nonstatistical measures.--The Secretary shall give 
        equal weight to clinical judgment and statistical analysis in 
        the evaluation of the safety and effectiveness of drugs, 
        biological products, and devices, and shall not disapprove a 
        product application solely on the basis of a statistical 
        analysis or the rigid use of the 95 percent confidence level 
        convention. This policy shall apply--
                    ``(A) in evaluating clinical study designs and 
                endpoints; and
                    ``(B) in making decisions with respect to product 
                applications.
            ``(2) Types of nonstatistical measures.--The policy 
        established under paragraph (1), for the purposes described in 
        such paragraph--
                    ``(A) shall include but not be limited to 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 within 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 chapter V and 
section 351 of the Public Health Service Act shall be construed to 
incorporate the policy established in this section.''.

SEC. 7. MEMBERSHIP OF ONCOLOGY DRUGS ADVISORY COMMITTEE.

    Membership of the Oncology Drugs Advisory Committee of the Food and 
Drug Administration shall consist of no less than 2 patient 
representatives who are voting members of the committee.
                                 <all>