RESUMEN
BACKGROUND: The US FDA has developed numerous accelerated pathways (APs) to facilitate faster development and approval of innovative drugs addressing unmet needs. METHODS: To gauge how payers in the United States view APs, PAREXEL and the Network for Excellence in Healthcare Innovation (NEHI) surveyed 20 national, regional, public, and private payers whose coverage decisions impact 228 million patients. RESULTS: The survey shows that APs have created new challenges and concerns for payers, including greater difficulty valuing drugs that have less clinical information available at launch. CONCLUSIONS: Our survey indicates that policies must further the goal of getting needed new medicines to patients expeditiously, while managing the risks AP drugs entail for payers and, most importantly, patients. For developers, a trend toward value-based assessments of both AP and non-AP drugs by payers may require ever-earlier and more data-driven decision making about the value of new products in development.
RESUMEN
A key element required for translating new knowledge into effective therapies is early-stage venture capital that finances the work needed to identify a lead molecule or medical device prototype and to develop it to the proof-of-concept stage. This early investment is distinguished by great uncertainty over whether the molecule or prototype is safe and effective, the stability of the regulatory standards to which clinical trials are designed, and the likelihood that large follow-on investments for commercial development can be secured. Regulatory and reimbursement policies have a profound impact on the amount of capital and the types of life science projects that investors pursue. In this article I analyze several recent trends in early-stage venture capital funding, describe how these trends are influenced by regulatory and reimbursement policies, and discuss the role of policy makers in bringing new treatments to market. Policy makers can foster renewed private investment into critically needed early-stage products by increasing Small Business Innovation Research (SBIR) funding and public support for clinical trials in targeted areas of interest; creating regulatory pathways to enable early testing of experimental compounds in limited populations; and offering economic incentives for investors and developers in designated therapeutic areas.