Regulators, Sponsors Seek to Streamline Research

The key to accelerating drug development, according to numerous experts, is to revise requirements that generate lengthy, complex, and costly clinical trials. Several provisions of the FDA Safety & Innovation Act (FDASIA) aim to smooth the process for designing appropriate studies to accelerate new drug approvals. And a coalition of leading pharmaceutical companies have mapped out specific strategies for modernizing clinical research practices.

These efforts may build on a report from a White House expert panel that urges more efficient clinical research as one way to double the number of innovative new medicines coming to market over the next decade. The September report from the President's Council of Advisors on Science and Technology (PCAST) calls on sponsors and regulators to "incorporate new efficiencies into clinical trials of candidate medicines," and to form a public-private "Partnership to Accelerate Therapeutics" to achieve this and other goals.

One prominent proposal is for FDA to approve new drugs under a "special medical use" designation, which offers a rapid "initial approval" of a medicine for a narrow, high-risk population that stands to benefit. More broadly, PCAST proposes to improve FDA management and regulatory processes, to enhance monitoring of approved drugs, and to improve public understanding of the benefits and risks of medicines. Most of these ideas are not particularly new, and the report is short on specifics for realizing its goals. Yet, the panel's proposals may be a first step in developing a national strategy for addressing drug development challenges, noted FDA senior advisor Vicki Seyfert-Margolis at the 2012 Mid-Atlantic Bio conference in September.

FDASIA initiatives

The PCAST deliberations were complicated by FDA negotiations at the same time with industry and Congress on goals and policies for user fee renewal legislation. The resulting FDASIA includes some provisions for streamlining clinical research and for bringing new therapies to market that parallel the PCAST report. These include measures to reduce the number of patients needed to test orphan and "breakthrough" drugs and to provide fast track review of critical treatments by FDA. Additional policies aim to advance meta-analysis methodologies and the use of biomarkers, pharmacogenomics, and patient reported outcomes measures. Sponsors and FDA also may gain access to rare disease experts, moreover, under a program designed to encourage the use of novel clinical trial designs and statistical modeling approaches for complex drug development projects.

The infectious disease community is optimistic that extended exclusivity for "qualified infectious disease products" under the Generating Antibiotic Incentives Now (GAIN) provision will spur development of sorely-needed antibacterials and antifungals. And more treatments for children may result from legislative language that makes permanent extended exclusivity for drugs that develop pediatric labeling, along with added rewards for developing orphan drugs for children. A change in timing for submitting pediatric drug development plans to FDA will match up with European regulatory requirements and thus support global pediatric drug development efforts.

To smooth the drug approval process, PDUFA V establishes new "liaisons" in the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research. These review staffers will help sponsors gain answers to questions about a development program and the status of an application during review, but not respond to technical or scientific questions, noted CDER Office of New Drugs Director John Jenkins. He pointed to the need for better education of both reviewers and sponsors on "best communication practices," noting at a conference on user fee specifics sponsored by the Food and Drug Law Institute and Drug Information Association in September that FDA is not industry's "on-call consultant for every question that comes to mind."

More collaboration

While FDA strives to implement FDASIA, pharma companies are taking steps themselves to tackle R&D costs and delays through the TransCelerate BioPharma project. Ten leading firms announced the initiative in September, outlining an initial focus on developing standards to streamline clinical research. The project will be headed by former Johnson & Johnson VP Garry Neil and seeks fast action in several pre-competitive projects: a shared user interface for investigator site portals; mutual recognition of study site qualification and training; risk-based standards for site monitoring and clinical data; and a comparator drug supply model. The member firms will provide financial support and personnel to work on these initiatives, and the board of member R&D directors aims to achieve visible progress by next year.

—Jill Wechsler