US Science Advisors Issue Report on Innovation in Drug Discovery and Development

The President’s Council of Advisors on Science and Technology (PCAST) released a

containing recommendations for boosting innovation in drug discovery and development. The report, Propelling Innovation in Drug Discovery, Development, and Evaluation, identified two major areas related to drug discovery and development that should be addressed to advance innovation. The first is a gap in scientific knowledge between basic research and commercial projects. The report notes that advances in basic research are not matched by similar advances in the technologies needed for drug development. The second deficit is inefficiency in clinical trials. According to the report, clinical trials efforts are heavily siloed, with each developer starting from scratch for each trial, and with complex and lengthy requirements for trial approval.

While stakeholders in the study acknowledged that not all the barriers to advancing the development of new drugs were within the purview of FDA, PCAST identified several challenges related to how drugs are evaluated and approved. According to the report, the accelerated approval pathway could be strengthened by stronger tools for and enforcement of postapproval study. The report also called for greater clarity in the approval process and greater consistency, efficiency, and communication between FDA and developers with regards to individual drug applications.

The long-term goal set by the study is “to double the output of innovative, new medicines for patients with important unmet medical needs, while increasing drug efficacy and safety, through industry, academia, and government working together to decrease clinical failure, clinical trial costs, time to market, and regulatory uncertainty.” PCAST believes this goal to be achievable over the next 10–15 years, and set forward specific recommendations aimed at reducing barriers to innovation, including:

• Supporting federal initiatives to accelerate therapeutics to improve drug discovery and development

• Catalyzing the creation of a broad-based partnership to accelerate therapeutics

• Expanding the use in practice of FDA’s existing authorities for accelerated approval and for confirmatory evidence.

• Creating a new pathway for initial approval of drugs shown to be safe and effective in a specific subgroup of patients.

• Exploring approaches for adaptive approval via pilot projects under existing pathways but not creating new adaptive approval pathways through legislation.

• Improving FDA’s tools for monitoring and communication of clinical benefits and risks

• Improving FDA management

• Studying current and potential economic incentives to promote innovation in drug development.

In the accompanying

, Eric Lander, president of the Broad Institute of Harvard and the Massachusetts Institute of Technology and co-chair of the study said, “With improved collaboration among all the participants in the drug development ecosystem and optimization of drug-evaluation pathways, American researchers and companies should be able to accelerate the development of safe and effective drugs while also strengthening the US economy.”