To amend the Federal Food, Drug, and Cosmetic Act with respect to fast track approval of certain orphan drugs.

Summary: Unlocking Lifesaving Treatments for Rare-Diseases Act or ULTRA - Amends the Federal Food, Drug, and Cosmetic Act to authorize the Secretary of Health and Human Services (HHS) to approve an application for a drug as a fast track product using a surrogate endpoint, based on the existence of reasonable scientific data that support and qualify the relevance of such endpoint to the disease state and treatment, if the Secretary: (1) makes an initial determination that the drug is eligible for approval as a drug designated for a rare disease or condition (orphan drug) and as a fast track product, and (2) determines that the drug is a treatment for a disease or condition that affects a small number of patients in the United States. Prohibits the Secretary from requiring clinical treatment or other historical clinical data on such endpoint as a prerequisite to assessment of that endpoint if such scientific data is not available.

Directs the Secretary to issue guidance providing details and options for qualifying surrogate endpoints without clinical data, taking into account and balancing: (1) the unmet need served by the drug and the adverse effects of the rare disease or condition on quality and length of life, (2) the very low likelihood that clinical data would exist or that clinical studies would be completed to support a surrogate endpoint due to the small size of the U.S. patient population and other significant barriers inherent in performing such studies due to the prevalence of the disease or related factors, and (3) the full scope of available basic scientific data and information that the Secretary deems reasonably predictive of a clinical benefit in the absence of clinical data.

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Unlocking Lifesaving Treatments for 
Rare-Diseases Act'' or ``ULTRA''.

SEC. 2. IMPROVING THE ACCELERATED APPROVAL PATHWAY FOR FAST TRACK 
              PRODUCTS TO SERVE THE UNMET NEEDS OF INDIVIDUALS WITH 
              ULTRA RARE DISEASES.

    Section 506 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
356) is amended by adding at the end the following:
    ``(e) Scientific Standards for Approval of Certain Orphan Drugs as 
Fast Track Products.--
            ``(1) In general.--The Secretary may approve an application 
        for a drug designated under section 526 for a rare disease or 
        condition as a fast track product using a surrogate endpoint as 
        described under paragraph (2) if--
                    ``(A) the Secretary makes an initial determination 
                that the drug is eligible for approval--
                            ``(i) as a drug designated for a rare 
                        disease or condition under section 526; and
                            ``(ii) as a fast track product under this 
                        section; and
                    ``(B) the drug is a treatment for a disease or 
                condition that affects a small number of patients in 
                the United States, as determined by the Secretary in 
                designating the drug for a rare disease or condition 
                under section 526.
            ``(2) Surrogate endpoint definition for certain fast track 
        products.--
                    ``(A) In general.--If a drug meets the criteria 
                established in paragraph (1), the Secretary--
                            ``(i) may use a surrogate endpoint for the 
                        approval of the drug as a fast track product 
                        based on the existence of reasonable scientific 
                        data that support and qualify the relevance of 
                        the surrogate endpoint to the disease state and 
                        treatment; and
                            ``(ii) shall not require clinical treatment 
                        data or other historical clinical data on the 
                        surrogate endpoint as a prerequisite to 
                        assessment of the surrogate endpoint under this 
                        subsection if such data are not available.
                    ``(B) Use of clinical data.--
                            ``(i) Subject to subparagraph (A)(ii), in a 
                        surrogate endpoint assessment under this 
                        subsection, the Secretary may take into 
                        consideration any reliable clinical data that 
                        are readily available and published.
                            ``(ii) For a surrogate endpoint which the 
                        Secretary decides to use in accordance with 
                        subparagraph (A), nothing in this subsection 
                        shall preclude the Secretary from requiring 
                        clinical data that makes use of the surrogate 
                        endpoint as a condition of approval for the 
                        fast track product.
                    ``(C) Guidance and considerations.--Not later than 
                1 year after the date of enactment of the Unlocking 
                Lifesaving Treatments for Rare-Diseases Act, the 
                Secretary shall issue guidance providing details and 
                options for qualifying surrogate endpoints without 
                clinical data pursuant to this subsection. In 
                qualifying a surrogate endpoint under this subsection, 
                the Secretary shall take into account and balance the 
                following considerations:
                            ``(i) The unmet need served by the drug and 
                        the adverse effects of the rare disease or 
                        condition on quality of life and length of 
                        life.
                            ``(ii) The very low likelihood that 
                        clinical data would exist or that clinical 
                        studies would be completed to support a 
                        surrogate endpoint due to the small size of the 
                        patient population in the United States and 
                        other significant barriers inherent in 
                        performing such clinical studies due to the 
                        prevalence of the disease or related factors.
                            ``(iii) The full scope of available basic 
                        scientific data and information describing the 
                        pathophysiology of the disease, mechanism of 
                        action of the drug, biology of the relevant 
                        disease pathway, information regarding the 
                        quality of the biomarker assay, model treatment 
                        data, or other supportive scientific 
                        information that the Secretary deems reasonably 
                        predictive of a clinical benefit in the absence 
                        of clinical data.''.