The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187

One Hundred Twelfth Congress of the United States of America

An Act  To amend the Federal Food, Drug, and Cosmetic Act to revise and extend the user-fee programs for prescription drugs and medical devices, to establish user-fee programs for generic drugs and biosimilars, and for other purposes.

Signed into law on July, 9, 2012

FDASIA included:


Sec. 901. Enhancement of accelerated patient access to new medical treatments.

From the ULTRA/FAST ACT:   Unlocking Lifesaving Treatments for Rare Diseases Act (ULTRA),HR 3737 /Faster Access to Specialized Treatments (FAST), HR 4132

Considerations. – In developing the guidance . . . . the Secretary shall consider . . . . for drugs designated for a rare disease or condition under section 526 of the Federal, Food, Drug, and Cosmetic Act; and
(2)how to incorporate novel approaches to the review of surrogate endpoints based on pathophysiologic and pharmacologic evidence in such guidance, especially in instances where the low prevalence of a disease renders the existence or collection of other types of data unlikely or impractical.”

Sec. 902. Breakthrough therapies.

Advancing Breakthrough Therapies for Patients Act of 2012, S.2236/HR 5334

Sec. 903. Consultation with external experts on rare diseases, targeted therapies,and genetic targeting of treatments.

Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act of 2012 H.R.4156S.2281

Sec. 905. Risk-benefit framework.

Sec. 906. Grants and Contracts for the Development of Orphan Drugs.

Sec. 908. Rare pediatric disease priority review voucher incentive program.

The Creating Hope Act of 2011 (S 606, HR 3059): Market Incentives to Develop Pediatric Rare Disease Drugs


TITLE XI—OTHER PROVISIONS:  Subtitle C—Miscellaneous Provisions 


Subchapter E of chapter V (21 U.S.C. 360bbb et seq.), as amended by section 1123 of this Act, is further amended by adding at the end the following:

‘‘(a) IN GENERAL.—The Secretary shall develop and implement strategies to solicit the views of patients during the medical product
development process and consider the perspectives of patients during regulatory discussions, including by—

‘‘(1) fostering participation of a patient representative who may serve as a special government employee in appropriate agency meetings with medical product sponsors and investigators; and

‘‘(2) exploring means to provide for identification of patient representatives who do not have any, or have minimal, financial
interests in the medical products industry.

‘‘(b) PROTECTION OF PROPRIETARY INFORMATION.—Nothing in this section shall be construed to alter the protections offered by
laws, regulations, or policies governing disclosure of confidential commercial or trade secret information and any other information
exempt from disclosure pursuant to section 552(b) of title 5, United States Code, as such laws, regulations, or policies would apply
to consultation with individuals and organizations prior to the date of enactment of this section.

‘‘(c) OTHER CONSULTATION.—Nothing in this section shall be construed to limit the ability of the Secretary to consult with
individuals and organizations as authorized prior to the date of enactment of this section.

‘‘(d) NO RIGHT OR OBLIGATION.—Nothing in this section shall be construed to create a legal right for a consultation on any
matter or require the Secretary to meet with any particular expert S. 3187—133 or stakeholder. Nothing in this section shall be construed to alter agreed upon goals and procedures identified in the letters described in section 101(b) of the Prescription Drug User Fee Amendments of 2012. Nothing in this section is intended to increase the number of review cycles as in effect before the date of enactment of this section.

‘‘(e) FINANCIAL INTEREST.—In this section, the term ‘financial interest’ means a financial interest under section 208(a) of title
18, United States Code.’’.