Sponsors would be able to reach preliminary agreements with FDA for product development through accelerated approval in the House 21^st Century Cures discussion draft.

The idea appears to be similar to the non-binding special protocol assessment FDA already employs for some products and could help create more certainty for sponsors.

The 21^st Century Cures Act discussion draft released April 29 is the latest iteration in the House Energy and Commerce Committee’s effort to streamline drug development and get new drugs to market faster.

It was dramatically scaled down compared to the discussion document released in January (199 v. 393 pages). Among the dropped provisions were exclusivity extensions for some products ([A#14150127007]).

The Biotechnology Industry Organization joined a number of other groups in praising the latest version.

President and CEO Jim Greenwood said in a written statement that it strongly supports “provisions intended to further incentivize biomedical innovation and help expedite the development of new treatments and cures, particularly in areas of unmet medical need where existing incentives may be inadequate to drive sufficient investment.”

But the group also said it would work to add more incentives to the next draft or final bill.

Officials from FDA and the National Institutes of Health are expected to discuss the bill with committee members during an April 30 hearing.

A similar bill is under development, albeit at a slower pace, in the Senate Also see "Senator Tells FDA To Create ‘Red Team’ For Regulatory Streamlining" - Pink Sheet, 28 April, 2015..

Speed And Confidence

Accelerated approval development plans are intended to encourage early interaction between FDA and sponsors, which ideally can help increase the likelihood of product clearance.

The plans would be voluntary and not required for an accelerated approval.

Agreements could be reached any time after “submission of an application for the investigation” of a drug or biologic, according to the draft.

The plans would include a determination that an unmet medical need exists, as well as an agreement on the study design, including planned interim analyses, if applicable, that will use a surrogate endpoint.

FDA and the sponsor also would agree to the “minimum magnitude of the effect of the drug on the surrogate endpoint that would be reasonably likely to predict clinical benefit,” according to the draft.

In addition, plans could include post-marketing commitments and the surrogate endpoint to be assessed in the study.

Accelerated approval development plans could be changed or terminated if found to be insufficient to demonstrate safety and efficacy or the product is no longer eligible for accelerated approval, according to the draft.

Setting the reasons for ending an agreement could prove important. FDA has been criticized for diminishing the value of SPAs because information about rescissions has not always been made available ([A#14140612001]).

A Communication Issue

Allowing FDA-sponsor agreements in the early development stages could be a solution that may encourage investments, as well as improving communication to ensure development remains on track.

FDA embraced the concept when it was creating its biosimilar program. The agency designed a formal meetings process funded by user fees to allow pre-submission interaction for sponsors to seek scientific and other advice on their development programs Also see "How To Get A Meeting With FDA: Guidance Describes Formal Meetings For Biosimilars" - Pink Sheet, 29 March, 2013..

In PDUFA V, FDA and industry also agreed to extend the length of NDA and original BLA reviews to allow for more interaction between agency officials and sponsors, in the hopes of increasing the rate of first-cycle approvals.

An interim assessment of the program indicated that sponsors on the whole thought the enhanced communication was positive ([A#00150406021]).

NIH Gains Funding, But Will It Stick?

The latest draft Cures bill also would create additional money for NIH.

NIH would receive $2 billion for a new Innovation Fund in fiscal years 2016 through 2020, but its use remains somewhat of a mystery. The draft states it would support precision medicine, young emerging scientists and other items, but also indicated more language was to be supplied.

The fund’s creation also may be uncertain going forward, if for no other reason than the Energy and Commerce Committee does not appropriate money.

In the existing budget environment, it may be difficult to find such a boost for NIH. FDA already is facing little or no increase in budget authority in FY 2016 ([A#14150304004]).

Draft ‘Cures’ Bill Allows Advance Accelerated Approval Agreements

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