SEC. 2. IMPROVING THE ACCELERATED APPROVAL PATH-
WAY 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 deter-
mination 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 pa-
tients in the United States, as determined by 
the Secretary in designating the drug for a rare 
disease or condition under section 526. 
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‘‘(2) SURROGATE ENDPOINT DEFINITION FOR 
CERTAIN FAST TRACK PRODUCTS.— 
‘‘(A) IN GENERAL.—If a drug meets the 
criteria established in paragraph (1), the Sec-
retary— 
‘‘(i) may use a surrogate endpoint for 
the approval of the drug as a fast track 
product based on the existence of reason-
able scientific data that support and qual-
ify the relevance of the surrogate endpoint 
to the disease state and treatment; and 
‘‘(ii) shall not require clinical treat-
ment 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 
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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 enact-
ment 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 clin-
ical data would exist or that clinical stud-
ies would be completed to support a surro-
gate endpoint due to the small size of the 
patient population in the United States 
and other significant barriers inherent in 
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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, mecha-
nism of action of the drug, biology of the 
relevant disease pathway, information re-
garding 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.’’. 
Æ 
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