‘‘(C) SMALL POPULATION STUDIES.—The 
external experts on the list maintained pursuant 
to subparagraph (A) may include experts on the 
science of small population studies.’’. 
(e) STUDY
ON SUFFICIENCY
AND USE
OF FDA 
MECHANISMS FOR INCORPORATING THE PATIENT AND 
CLINICIAN PERSPECTIVE IN FDA PROCESSES RELATED 
TO APPLICATIONS CONCERNING DRUGS FOR RARE DIS-
EASES OR CONDITIONS.— 
(1) IN GENERAL.—The Comptroller General of 
the United States shall conduct a study on the use 
of Food and Drug Administration mechanisms and 
tools to ensure that patient and physician perspec-
tives are considered and incorporated throughout the 
processes of the Food and Drug Administration— 
(A) for approving or licensing under sec-
tion 505 of the Federal Food, Drug, or Cos-
metic Act (21 U.S.C. 355) or section 351 of the 
Public Health Service Act (42 U.S.C. 262) a 
drug designated as a drug for a rare disease or 
condition under section 526 of the Federal 
Food, Drug, and Cosmetic Act (21 U.S.C. 
360bb); and 
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(B) in making any determination related 
to such a drug’s approval, including assessment 
of the drug’s— 
(i) safety or effectiveness; or 
(ii) postapproval safety monitoring. 
(2) TOPICS.—The study under paragraph (1) 
shall— 
(A) identify and compare the processes 
that the Food and Drug Administration has 
formally put in place and utilized to gather ex-
ternal expertise (including patients, patient 
groups, and physicians) on specific applications 
for diseases or conditions affecting 20,000 or 
fewer patients in the United States and specific 
applications for diseases or conditions affecting 
200,000 or fewer patients in the United States; 
(B) examine tools or mechanisms to im-
prove efforts and initiatives of the Food and 
Drug Administration to collect and consider 
such external expertise with respect to applica-
tions for diseases or conditions affecting 20,000 
or fewer patients in the United States com-
pared to applications for diseases or conditions 
affecting 200,000 or fewer patients in the 
United States throughout the application review 
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and approval or licensure processes, including 
within internal benefit-risk assessments, advi-
sory committee processes, and postapproval 
safety monitoring; and 
(C) examine processes or alternatives to 
address or resolve conflicts of interest that im-
pede the Food and Drug Administration in 
gaining external expert input on rare diseases 
or conditions with a limited set of clinical and 
research experts. 
(3) REPORT.—Not later than 2 years after the 
date of enactment of this Act, the Comptroller Gen-
eral of the United States shall— 
(A) complete the study under paragraph 
(1); 
(B) submit a report on the results of such 
study to the Congress; and 
(C) include in such report recommenda-
tions, if appropriate, for changes to the proc-
esses and authorities of the Food and Drug Ad-
ministration to improve the collection and con-
sideration of external expert opinions of pa-
tients, patient groups, and physicians with ex-
pertise in rare diseases or conditions, including 
any specific recommendations for diseases or 
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conditions affecting 20,000 or fewer patients in 
the United States. 
(f) DEFINITION.—In this section, the term ‘‘rare dis-
ease or condition’’ means a disease or condition affecting 
fewer than 200,000 persons in the United States. 
Æ 
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