By Anupam B. Jena, Rebecca Kee, James R. Baumgardner and Qiufei MaJie Zhang
October 25, 2019

Recent years have brought significant innovation in potentially curative therapies for several diseases. To expedite patient access to these therapies, the U.S. Food and Drug Administration (FDA) has undertaken progressive efforts to accelerate drug approval. For example, the FDA’s Breakthrough Therapy Program, enacted in 2012, has approved nearly 140 breakthrough therapies and shortened novel drug approval times by almost a year. By 2025, the FDA anticipates approving 10 to 20 cell and gene therapy products per year, many of which are intended to be one-time curative treatments.

But despite these efforts to accelerate the evaluation of drug safety and efficacy, uptake of many new therapies has been much slower than expected. Why the lag? The delays in uptake are largely due to the high cost of new treatments and the hurdles in coverage and reimbursement faced by commercial and public payers. Even as new payment methodologies, such as value-based payment models, have arisen, and even as insurers have offered greater reimbursement of some curative therapies, there remain many patients who are either denied coverage of therapies or experience significant delays in getting coverage. There are also challenges in manufacturing these newer therapies.

Addressing these obstacles is critical if we want to ensure patients have access to new life-changing treatments, many of which need to be administered early on to be most effective. Fortunately, there are a number of innovative solutions being tested now that are worth paying attention to.

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