21st Century Cures Initiative: HP&M’s Frank Sasinowski Testifies at First Congressional Hearing

May 21, 2014

By Alexander J. Varond & James E. Valentine –

On May 20, 2014, Hyman, Phelps & McNamara, P.C.’s Frank J. Sasinowski appeared on a panel before the House Energy and Commerce’s Subcommittee on Health to present testimony at the first hearing on the 21st Century Cures Initiative.  The Subcommittee sought expert testimony about the proposals put forth in the 2012 “Report to the President on Propelling Innovation in Drug Discovery, Development, and Evaluation” by the President’s Council of Advisors on Science and Technology ("PCAST") (see our previous post here).  The Subcommittee asked the panel to discuss how recommendations presented in the PCAST report could be used by FDA and drug sponsors to advance the mandate of the 21st Century Cures initiative to “ensure that the U.S. owns the discovery, development, and delivery cycle and thus, remains the world leader in innovation.”

On behalf of the National Organization of Rare Disorders and himself, Mr. Sasinowski offered four recommendations:

Proposal #1: FDA should consider the appropriateness of the Accelerated Approval process for every new therapy.

Proposal #2: FDA and sponsors should use intermediate clinical endpoints ("ICE") more often to secure Accelerated Approvals.

Proposal #3: FDA should increase its use of the statutory authority conferred by FDAMA to approve drugs with one adequate and well-controlled study with “confirmatory evidence” and by FDASIA to approve drugs via the Accelerated Approval pathway (Proposal #1) using a simple chart (see below).

Proposal #4:  FDA should issue a guidance on cumulative distribution analyses of clinical study results as a way to help understand the clinical meaningfulness of a new therapy.

Mr. Sasinowski’s written testimony provides greater detail for each of his four proposals, and includes findings from the Subpart H analysis (see our previous post here) he and Alexander J. Varond submitted on August 26, 2013, in response to FDA’s draft guidance entitled “Expedited Programs for Serious Conditions – Drugs and Biologics” (Docket No. FDA-2013-D-0575).  A press release is available online (here), and a video recording of the hearing will also be available online (here).

 Chart for Proposal #3: