ICER Publishes White Paper Evaluating Policy Options to Strengthen FDA’s Accelerated Approval Pathway for Prescription Drugs

— Informed by expert input from patient groups, former regulators, payers, and life science companies, white paper analyzes potential impact of a variety of reform options that address the balance between uncertainty, access, innovation, and costs

BOSTON, April 26, 2021 - The Institute for Clinical and Economic Review (ICER), in collaboration with researchers from the Drug Pricing Lab at Memorial Sloan Kettering Cancer Center, has published a new white paper: "Strengthening the Accelerated Approval Pathway: An Analysis of Potential Policy Reforms and Their Impact on Uncertainty, Access, Innovation, and Costs." This paper aims to create a clearer understanding of both the opportunities and challenges inherent in the accelerated approval pathway (AAP), and to present an analysis of the potential risks and benefits of a range of reform options that policymakers may consider in efforts to strengthen the AAP moving forward.

"Since 1992, the FDA's Accelerated Approval Program has brought important new treatments to patients faster than would have been possible through the traditional approval process," said Steven D. Pearson, MD, MSc, President of ICER. "The successes are many, and the benefits for patients overall have been substantial. But criticisms have emerged as well, including concerns that the FDA has lacked consistency in its application of evidentiary standards, and that the incentives and procedures in place today have proven inadequate to get high quality confirmatory trials completed in a reasonable time frame. From various points on the spectrum, concerns are also raised that patients may not be getting fair access to these drugs, while some question whether the high prices for most of the drugs approved through this pathway are merited given the level of uncertainty about their effectiveness at launch. 

"So today, after nearly three decades of experience, it is a good time to take stock of where the AAP is, and what might be done to strengthen it. No policy reform options come without potential downsides, and the goal of our white paper is to outline the potential benefits and risks of major policy options."

The white paper explores multiple policy reforms that could be accomplished through FDA action:

  • Strengthening the selection of surrogate endpoints
  • Developing standardized AAP templates to increase consistency and evidence standards
  • Requiring greater use of randomized controlled trials
  • Creating a new "alert" on the label of AAP therapies
  • Tying economic incentives to the completion of confirmatory trials
  • Changing the conditions for continued marketing approval, if confirmatory evidence is not provided by a pre-determined date
  • Offering a "safety-only" approval, without requirement of public or private insurance coverage, for treatments that have demonstrated safety but not an ability to improve patients' lives

In addition, the paper explores options that require a mix of actions across US payers, drug manufacturers, and federal policymakers:

  • Increasing mandatory federal rebate levels until time of full approval
  • Pricing at marginal cost until confirmatory evidence reviewed, or allowing full market pricing at launch that reverts to marginal cost pricing if confirmatory trials are not completed in a reasonable timeframe
  • Requiring payment for AAP therapies be tied to outcomes-based contracts

The lead author of the paper is Anna Kaltenboeck, a Senior Health Economist at the Center for Health Policy and Outcomes at Memorial Sloan Kettering Cancer Center.  The paper went through several iterations following ICER's Policy Summit in March 2021, where ICER's Policy Leadership Forum — comprising senior policy leaders from 30 payer and life science companies — discussed a preliminary draft, debated the reform proposals, and provided suggestions for revisions to the paper. None of these individuals, nor their organizations, is responsible for the final contents of this report or should be assumed to support any part of this report, which is solely the work of the ICER team and its affiliated researchers.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER's reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER's reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER's reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER's website.

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