To amend the Federal Food, Drug, and Cosmetic Act to mandate early access by desperately ill patients to treatment use of new drugs under clinical investigation for a serious or immediately life-threatening disease condition for whom no comparable or satisfactory drug or other therapy is available.

Summary: Early Access to Treatment Act - Amends the Federal Food, Drug, and Cosmetic Act to require a drug that is not approved for marketing, but that is under clinical investigation for a serious or immediately life-threatening disease condition in patients for whom no comparable or satisfactory alternative drug or other therapy is available, to be made available for treatment use by an individual patient if: (1) the drug is intended to treat such condition; (2) there is no comparable or satisfactory alternative available to treat that stage of the disease; (3) the drug is under investigation in a controlled clinical trial as an investigational new drug or all clinical trials have been completed; and (4) an application for treatment use has been filed with the Food and Drug Administration (FDA) by a licensed practitioner which sets forth the intended use of the drug, an explanation of the rationale for its use, the treatment protocol, statements of the practitioner's qualifications to use the drug and familiarity with its safety and effectiveness, and a notarized statement of the patient's informed consent.

 Requires the Secretary of Health and Human Services, within 30 days after an application is filed, to grant permission to the sponsor to furnish such drug to the practitioner under such terms as the sponsor, practitioner, and patient determine to be appropriate, including any agreement to waive liability. Provides for application transmission by telephone or other means of rapid communication and for rapid action on it in an emergency situation.

Requires (current law authorizes) the Secretary to approve an application under the Public Health Service Act for a fast track product determined to have an effect on a clinical endpoint or on a surrogate endpoint that is reasonably likely to predict clinical benefit.

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Early Access to Treatment Act''.

SEC. 2. FINDINGS.

    Congress finds the following:
            (1) Section 506(a)(1) of the Federal Food, Drug, and 
        Cosmetic Act (21 U.S.C. 356(a)(1)) requires the Secretary of 
        Health and Human Services, at the request of the sponsor of a 
        new drug, to ``facilitate the development and expedite the 
        review of such drug if it is intended for the treatment of a 
        serious or life-threatening condition and it demonstrates the 
        potential to address unmet medical needs for such a 
        condition'', designating such a drug to be a ``fast track 
        product''.
            (2) Section 506(b)(1) of such Act, however, provides that 
        the Secretary need not approve an application for approval of a 
        fast track product, even though the Secretary has determined 
        ``that the product has an effect on a clinical endpoint or on a 
        surrogate endpoint that is reasonably likely to predict 
        clinical benefit''.
            (3) Section 312.34(a) of title 21, of the Code of Federal 
        Regulations, provides for treatment use of a new drug, although 
        such ``drug . . . is not yet approved for marketing,'' but is 
        ``under clinical investigation for a serious or immediately 
        life-threatening disease condition in patients for whom no 
        comparable or satisfactory alternative drug or other therapy is 
        available''.
            (4) Although the stated purpose of both current law and 
        regulations, as stated under section 312.34(a) of such title, 
        is ``to facilitate the availability of promising new drugs to 
        desperately ill patients as early in the drug development 
        process as possible, before general marketing begins'', in 
        practice applications for approval of promising therapies 
        intended to treat such desperately ill patients suffering from 
        serious and immediately life-threatening diseases for whom no 
        comparable or alternative drug or other therapy is available 
        have encountered unjustified delays and denials.
            (5) As a consequence of such delays and denials of 
        applications for such treatment use, desperately ill patients 
        suffering from serious and immediately life-threatening disease 
        conditions have been denied the last, and perhaps the only, 
        best chance for treatment protocols that might have saved their 
        lives.
            (6) Applications for such treatment use ought not be 
        subject to the discretion of the Secretary, but should be 
        granted, in all cases, if such applications meet the statutory 
        criteria governing such applications.
            (7) Applications for fast track product review ought not be 
        subject to the discretion of the Secretary, but should be 
        granted, in all cases, if such applications meet the statutory 
        criteria governing such applications as set forth in section 
        506(b)(1) of the Federal Food, Drug, and Cosmetic Act (21 
        U.S.C. 356(b)(1)).

SEC. 3. TREATMENT USE APPROVAL SYSTEM.

    Section 505 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
355) is amended by adding at the end the following new subsection:
    ``(v) Treatment Use of an Investigational Drug.--
            ``(1) In general.--A drug that is not approved for 
        marketing, but is under clinical investigation for a serious or 
        immediately life-threatening disease condition in patients for 
        whom no comparable or satisfactory alternative drug or other 
        therapy is available, shall be made available for treatment use 
        by an individual patient if--
                    ``(A) the drug is intended to treat a serious or 
                immediately life-threatening disease condition;
                    ``(B) there is no comparable or satisfactory 
                alternative drug or other therapy available to treat 
                that stage of the disease in the person for whom the 
                drug is intended;
                    ``(C) the drug is under investigation in a 
                controlled clinical trial as an investigational new 
                drug under subsection (i) in effect for the trial or 
                all clinical trials have been completed; and
                    ``(D) an application for treatment use has been 
                filed in writing with the Food and Drug Administration 
                by a licensed practitioner and such application sets 
                forth--
                            ``(i) the specific intended use of the 
                        drug;
                            ``(ii) an explanation of the rationale for 
                        use of the drug, including an explanation as to 
                        why the use of the investigational drug is 
                        necessary in the treatment of the individual 
                        patient;
                            ``(iii) a treatment protocol, including the 
                        method of the administration of the drug and 
                        dosages;
                            ``(iv) a statement of the qualifications of 
                        the licensed practitioner to use the 
                        investigational drug for the intended treatment 
                        use;
                            ``(v) a statement of the licensed 
                        practitioner's familiarity with information of 
                        the drug's safety and effectiveness derived 
                        from previous clinical and nonclinical 
                        experience with the drug; and
                            ``(vi) a signed and notarized statement of 
                        the individual patient, or guardian, if any, 
                        setting forth in detail--
                                    ``(I) the information about the 
                                drug's safety and effectiveness;
                                    ``(II) risks disclosed to the 
                                patient; and
                                    ``(III) the patient's informed 
                                consent to the treatment protocol to be 
                                administered by the licensed 
                                practitioner.
            ``(2) Permission for treatment use.--
                    ``(A) Deadline.--Within 30 days of the filing of an 
                application for treatment use in compliance with 
                paragraph (1), the Secretary shall grant permission to 
                the sponsor of an investigational drug to furnish such 
                drug to the submitting licensed practitioner under such 
                terms as the sponsor, licensed practitioner, and 
                patient determine among themselves to be appropriate, 
                including any agreement to waive liability.
                    ``(B) Treatment of emergencies.--If the need for an 
                investigational drug arises in an emergency situation 
                that does not allow time for submission of such an 
                application for treatment use in writing, such 
                application may be transmitted to the Secretary by 
                telephone or by other means of rapid communication, and 
                shall be acted on rapidly, but in no case later than 30 
                days after the date of receipt of the application.
            ``(3) Definitions.--In paragraph (1)--
                    ``(A) the term `serious' means, with respect to a 
                disease or condition, a disease or condition that 
                causes major irreversible morbidity; and
                    ``(B) the term `immediately life-threatening' 
                means, with respect to a disease or condition--
                            ``(i) a disease or condition where the 
                        likelihood of death is high unless the course 
                        of the disease or condition is interrupted; or
                            ``(ii) a disease or condition with 
                        potentially fatal outcomes, where the end point 
                        of clinical trial analysis is survival.''.

SEC. 4. REMOVING DISCRETION IN APPROVAL OF APPLICATION FOR FAST TRACK 
              PRODUCTS.

    Section 506(b)(1) of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 356(b)(1)) is amended by striking ``may'' and inserting 
``shall''.