House "Cures" Proposal Faces Tough Road Ahead

The “discussion draft” for legislation to speed “21^st Century Cures” to patients emerged very quietly on Capitol Hill this week, muted by an absence of bipartisan support which had generated considerable enthusiasm for this effort to promote biomedical research and streamline regulation. The massive document (nearly 400 pages) offered by House Energy & Commerce Committee chairman Fred Upton (R-Mich) includes just about every Republican reform proposal offered in recent years, including a number of changes in the FDA approval process likely to dismay agency leaders. Rep. Frank Pallone (D-NJ), E&C ranking Democrat, issued a statement expressing disappointment with the proposal, and Rep. Diana DeGette (D-Col), titular co-chair of the Cures initiative, withheld her endorsement, but left the door open to reaching bipartisan consensus.

While analysts continue to examine the specifics of the draft plan [http://energycommerce.house.gov/sites/republicans.energycommerce.house.gov/files/114/Analysis/Cures/20150127-Cures-Discussion-Document-Section-by-Section.pdf ], a general objection is that it offers no new funding to support the multiple programs and mandates added to FDA’s already overloaded agenda. Similarly, numerous changes in National Institutes of Health (NIH) operations without expanded resources are generating protests from the research community. Health and biopharma organizations issued statements voicing the usual support for the effort, but clearly lacked enthusiasm.

More pointed protest came from the Generic Pharmaceutical Association, which said it was “deeply disappointed” with proposals in the draft plan to boost market exclusivity for certain new therapies. GPhA predicted that these changes would erode generic drug utilization and savings by upsetting “the important balance between creating competition and encouraging innovation in the pharmaceutical marketplace.”

GPhA president Ralph Neas is referring to a number of  “exclusivity stacking” provisions, as described by attorney Kurt Karst in the Hyman, Phelps & McNamara FDA Law Blog [http://www.fdalawblog.net, Jan. 28, 2015]. Karst notes exclusivity “opportunities” for certain new chemical entities, added indications, antibiotics, and a new one, “dormant therapies.” One area where there may be agreement is to use incentives to spur development of antibiotics, a goal championed by all sides and recently backed by President Obama. And some generics makers could support a provision offering extended exclusivity for “American-manufactured” generics and biosimilars.

The something-for-everyone document contains several provisions to revise clinical trial operations (revise human subject protections) and research methods (encourage adaptive trials, Bayesian methods). There’s a “placeholder” to add something like Obama’s “Precision Medicine” initiative, plus a long list of reforms to streamline oversight of medical devices, which have considerable support from device manufacturers.

Compassionate access to not-yet-approved therapies for severely ill patients also gets a nod. Here the legislators want to require pharma companies to be more “transparent” regarding expanded access programs and look to a new task force to recommend further reforms of FDA’s expanded access process.   

There will be more hearings and discussion of the proposal. But without some fast agreement on a few specific measures that generate support on both sides of the aisle, this much-ballyhooed “Cures” initiative will fall by the wayside, as do many grand policy reform efforts in Washington.