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Agency proposes new standards in hopes of capitalizing on 2014 surge in new drug approvals.
A surge in review activity at the FDA in December resulted in a near-record approval of 41 new drugs and biologics last year, the most since a record 56 approvals in 1996. A broader tally lists 54 new therapies by adding new drugs launched in Europe and Japan. FDA's total includes more than 15 orphan drugs and 15 first-in-class therapies, reflecting a continued industry shift to specialty drugs that can be developed faster, earn added exclusivity, and command high prices in an increasingly cost-conscious market. These trends support continued interest in developing drugs that qualify for breakthrough drug designation, which should continue to bring to market more new therapies for cancer and many critical rare conditions.
Janet Woodcock, director of FDA's Center for Drug Evaluation and Research (CDER), would like to see more drugs studied in children, more anti-microbials, and more studies supporting additional indications for cancer therapies, she said in presenting a long list of priorities for 2015 at the December FDA/CMS Summit in Washington, D.C. Woodcock highlighted CDER efforts to post more information on participation in trials by sex, race, age, and ethnicity, and to improve drug development and clinical testing through broader use of Bayesian statistics and adaptive clinical trials designs and by further developing patient-reported outcomes measures.
Some of these topics are included in CDER's wish list of 90 new draft and final guidance documents in 2015. In addition to guidelines for approving new sunscreens and for establishing a supply chain drug tracking program, as required by legislation, the agenda proposes standards for developing drugs to treat alcoholism, Duchenne Muscular Dystrophy, head lice infestations, and ulcerative colitis. The agency also seeks more guidance on measuring treatment benefit in pediatric populations and on including pregnant women in clinical trials. And it proposes to expand electronic submissions, including information to support planning for bioresearch monitoring inspections.
FDA's review program benefited from improvements supported by the user-fee supported "program" for making the drug approval process more efficient and effective. John Jenkins, director of CDER's Office of New Drugs (OND), reports that more submissions are being evaluated in only one review cycle, saving time and resources for sponsors and for OND review offices. And two-thirds of novel drugs last year were first approved in the U.S.
At the same time, CDER is devoting considerable resources to handling the unexpected response to the breakthrough drug program and now is evaluating how to make the two-year-old initiative more efficient. The agency has vetted more than 200 requests for designations since January 2013, and has granted 70, generally to products that demonstrate notable clinical effect. The program has led to approval of more than 12 breakthrough drugs for several critical conditions, and dozens more are in the pipeline.
But a lot of work is needed to evaluate the two-thirds of breakthrough designation requests that are denied, which occurs most often for drugs with limited efficacy, tested in very few patients, or with flawed trial designs. To better manage the program, FDA is considering an abbreviated process for writing up rejection reports on "obvious non-starters." Further guidance will aim to clarify the "bar" for breakthrough requests, an issue that will be discussed at a workshop with the Brookings Institution in April.
Such a change could give agency reviewers more time to provide the "focused attention" needed to evaluate streamlined clinical trials and innovative statistical methods key to successful breakthrough drug development. Equally important is assistance in accelerating the manufacturing process and scheduling timely plant inspections for a drug likely to come to market much faster than expected.
FDA's efforts to bring more new drugs to patients has been noted on Capitol Hill, where Congressional leaders are looking to enact legislation this year to further speed the development of drugs and medical devices for unmet medical needs. The House Energy & Commerce Committee is considering a "discussion draft" for a bipartisan bill to promote "21st Century Cures," with a goal of moving it through Congress by summer.
At the same time, Republican leaders in the House and Senate will be airing proposals to "modernize" FDA operations with an eye to reducing what some agency critics describe as bureaucratic hurdles to bringing life-saving therapies to patients. Congressional oversight hearings are expected to press for faster response to compassionate use requests, streamlined approval of new medical devices and diagnostics, and more attention to patient perspectives in designing clinical trials and expanded access programs.
FDA officials hope that Congress also will provide additional funding to support the breakthrough program and other new initiatives that place added demands on staff. CDER currently has some 650 staff vacancies, many in review divisions.