House Committee Reveals Drug Development Incentives, FDA Regulatory Overhaul

The House Energy & Commerce Committee that oversees the FDA is planning an overhaul of agency procedures to increase orphan drug development, streamline clinical trials oversight, give industry incentives to find new uses for old drugs and expand U.S. drug manufacturing.

The widely anticipated proposal was released last week by Rep. Fred Upton (R-Mich.), the committee chair, and committee member Diana DeGette (D-Colo). It grew out of hearings the committee held last year under its 21st Century Cures initiative, which sought suggestions for improving the process of developing and approving new medicines.

The 400-page draft bill touches on several federal agencies, including the NIH and Centers for Medicare and Medicaid Services, as well as the FDA. The committee is seeking comment on the draft to work in to a final bill that will address five broad areas: patient-focused medicines development, promoting drug development for unmet needs, modernizing clinical trials, lifting regulations that hinder U.S. drug manufacturing and improving the science of medicine.

Patient-focused drug development. To bring patients into the drug development process, the bill would direct the FDA to require more direct input from patients about the risks and benefits of a medicine when evaluating it.

In addition, drugmakers would have to establish and publicize details of their compassionate use policies and expanded access programs for drugs still under development.

The proposal would also allow the FDA to approve new breakthrough drugs using only promising early-stage trial data.

Promoting drug development for unmet needs. In language aimed at promoting development of drugs that are often ignored or overlooked, the bill would authorize collaboration between the FDA and industry to develop clinical trial protocols for needed antibiotics.

In addition, it would encourage drugmakers to look for new uses for existing drugs by establishing new exclusivity periods for companies that develop a new use for an old drug, provided the new indication addresses an unmet medical need, such as Alzheimer’s or a rare disease.

Modernizing clinical trials. To lift some of the costs of conducting clinical trials, the bill would streamline the IRB approval process for new protocols, especially in multisite trials.

The proposal would also direct the agency to be more open with drugmakers in establishing standards for surrogate endpoints for clinical trials, and encourage greater use of adaptive trial designs.

In addition, the bill calls for periodic reviews of postapproval studies so that they could be terminated when not needed.

The plan also calls for the FDA to develop a management succession plan to minimize the impact of personnel changes at the agency.

Improving the U.S. drug manufacturing climate. To encourage more U.S.-based drug manufacturing, the draft contemplates extending the exclusivity periods for first-filer generics and biosimilars that are made in the U.S.

In addition, it calls for updates to the FDA guidance on novel manufacturing techniques to encourage manufacturing innovation. It also proposes allowing third-party accreditors to approve minor manufacturing changes rather than requiring FDA approval.

Improving medicinal science. The proposal also includes efforts to improve the field of medicinal science, calling on the NIH to develop a strategic funding master plan.

The bill hints at regulatory relief in other areas as well, such as a loosening of some of the prohibitions on off-label marketing and new, less restrictive rules for promotion of drugs via social media.

The committee has not produced a timeline for when it wants to file the bill, much of which is still loosely shaped. However, Upton has said he would like to put the bill on track to reach the president by the end of the year. The draft draws on a list of proposals from 40 representatives.

See the discussion draft here: www.fdanews.com/01-27-15-21st-Century-Cures.pdf. A separate white paper on the initiative can be found at www.fdanews.com/01-27-15-21st-Century-White-Paper.pdf. — Bryan Koenig