CBO Projects 21st Century Cures Act’s FDA Initiatives Cost $872M

The Congressional Budget Office is estimating that provisions in the proposed drug overhaul bill, if implemented by the FDA, would cost $872 million from 2016-2020, according to a report issued last week.

The measure, the 21st Century Cures Act, or H.R. 6, was unanimously approved by the House Energy & Commerce Committee. It has not yet gone to the House floor.

Provisions in the massive overhaul bill would extend the rare pediatric disease priority review voucher program, encourage broader use of observational studies, patient registries and actual therapeutic use to support approvals of new drugs and enhance postmarket surveillance, and allow for greater use of innovative statistical methods in clinical trial designs. The bill also directs the FDA to issue guidance on acquiring clinical evidence on different categories of drugs outside randomized clinical trials.

CBO estimates that the FDA would spend $270 million through 2020 implementing provisions related to drugs and biologics, including:

• $33 million to establish a process to qualify or validate certain drug development tools such as biomarkers;
• $33 million to identify and publish a list of interpretive criteria for tests that characterize the susceptibility of particular bacteria, fungi or other microorganisms to drugs;
• $31 million to facilitate approval of certain antibacterial and antifungal drugs used by a limited population of patients;
• $21 million to implement a program aimed at providing incentives to drug companies to develop new indications for drugs and biologics that target rare diseases and conditions and extending the voucher program for rare pediatric diseases through Dec. 31, 2018;
• $20 million to develop a regulatory structure to allow the use of new protocols for statistical modeling and trial designs to support marketing applications for drug and biological products;
• $18 million to devise a plan with sponsors of drugs and biologicals eligible for accelerated approval to agree on details of the clinical study design so that approvals can be expedited; and
• $14 million to establish a streamlined data review program to allow sponsors to submit qualified summaries of clinical data to support approval or licensure of new indications under certain circumstances.

The bill also includes $327 million to establish a Cures Innovation Fund, which would encourage public-private partnerships and award grants to foster the collection, analysis and availability of data on the natural history of disease; support initiatives of the Council for 21st Century Cures; create a regulatory framework at the FDA that incorporates patient information including risks and benefits of new drugs; and administer a new FDA grant program to study continuous drug manufacturing and other production-related technologies.

No comparable measure exists in the Senate. 

The CBO report can be read here www.fdanews.com/06-24-15-cboreport.pdf. — John Bechtel