Advisors to President Barack Obama have issued a new report calling for doubling the output of innovative, new medicines for patients with important unmet medical needs. The group also recommends actions to improve drug efficacy and safety through collaboration between industry, academia, and government.
The President’s Council of Advisors on Science and Technology estimates that its ambitious goal is achievable within 10 to 15 years. To get there, it says, America will need to unleash “extraordinary innovation and investment in the service of public health.”
The advisory group is comprised of the nation’s leading scientists and engineers, appointed by the President to augment the science and technology advice available to him from inside the White House and from cabinet departments and other federal agencies.
“The pace of new therapeutic development has not kept up with the explosion in scientific knowledge,” say PCAST chairs John Holdren and Eric Lander in a letter accompanying the report. “The number of novel drugs has remained constant for several decades, even as R&D budgets have substantially increased.”
The U.S. Food and Drug administration approved 30 new molecular entities and biologics during 2011 and has approved 25 so far this year. To double 2011’s performance, drugmakers would need to secure 60 new drug approvals from the FDA, something they have only neared achieving once since 1975, during a banner year in 1996 when a clearing of FDA backlogs made possible by the initiation of user fees led to a remarkable 59 approvals.
To identify and develop solutions to the problems impeding drug innovation, PCAST solicited input from the biopharmaceutical industry, patient advocacy groups, physician societies, and healthcare payers, as well as senior leadership from the FDA and National Institutes of Health.
Based on that feedback, the group has made eight recommendations. It suggests improving drug discovery and development efforts by supporting federal initiatives, such as National Center for Advancing Translational Sciences at NIH, and catalyzing the creation of broad-based partnerships to accelerate therapeutics discovery, development, and evaluation.
To improve drug evaluation, the group says the FDA needs to more frequently pursue accelerated approvals, paired with post-approval studies to evaluate drugs addressing unmet needs; it suggests the agency create a new pathway for approving drugs shown to be safe and effective for a specific subgroup of patients; and, the FDA should explore approaches for adaptive approvals, it says, a framework for iteratively expanding the market for a drug as new evidence about its risks and benefits becomes available.
Post-marketing surveillance of new drugs should also be supported by a Congressionally authorized line item appropriation of $40 million per year to expand the FDA’s Sentinel Initiative, the group says. That initiative is creating a national electronic system to help the agency track the safety of drugs, biologics, and medical devices once they reach the market.
In addition, PCAST calls for the reform of FDA management practices and the creation of a study to determine whether current economic incentives are aligned to promote innovation.
To implement its ideas, the PCAST recommends the creation of a public-private “Partnership to Accelerate Therapeutics” within a year. That partnership would involve representatives from the biopharmaceutical industry, academic biomedical research, ethicists, physician societies, pharmacists, patient-focused research foundations, advocacy groups, healthcare providers, insurers, and the federal government.
By late 2015, PCAST suggests the partnership should be able to build active programs to address filling knowledge gaps in areas such as drug efficacy and toxicity and will have created better-organized and efficient clinical trials networks. It also expects that by then a study of key economic incentives and levers that should be deployed to support biomedical innovation will be complete.
Finally, by late 2017, the group estimates the FDA should have cleared its backlog of guidances—possibly by farming out their creation to the experts assembled in the partnership—and put in place a fully-funded safety monitoring system, such as Sentinel.
The authors of the PCAST report also acknowledge the limitations of their influence, recognizing that “reimbursement policy will have an increasing impact on innovation in drug development,” but it says that the federal government should exercise strong leadership to ensure the United States has “a strong ecosystem for drug discovery and development.”
September 28, 2012