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New drug approvals in oncology


The traditional regulatory drug approval paradigm comprising discrete phases of clinical testing that culminate in a large randomized superiority trial has historically been predominant in oncology. However, this approach has evolved in the current era of drug development, with multiple other development pathways now being utilized. Indeed, treatment approaches designed on the basis of an improved understanding of cancer biology have led to unprecedented responses in early phase trials, sometimes resulting in drug approvals in the absence of large-scale trials. At the same time, improved molecular diagnostic technologies have led to the identification of ever-smaller patient subgroups for molecularly targeted therapy. Moreover, new FDA regulatory paradigms have enabled the rapid review and accelerated approval of certain drugs in the absence of survival data. Regulatory approvals based on large-cohort trials with surrogate or intermediate clinical end points or on non-inferiority trials, as well as new tumour-agnostic indications, also set important precedents in the field. In this Viewpoint, we asked two leading oncologists involved in clinical drug development, an expert in regulatory science and prescription drug policy and a prominent patient advocate, to provide their opinions on the implications of these changes in regulatory practices for patient care.

The contributors

R.K. is the Director of the Center for Personalized Cancer Therapy and founder/director of the Rare Tumor Clinic (as well as Associate Director of the Clinical Science division and Leader of the Experimental Therapeutics Program) at the University of California San Diego Moores Cancer Center. Previously, at MD Anderson Cancer Center, she founded, built up and chaired one of the world’s largest clinical trials departments. Her expertise and interests are in patient-focused precision medicine.

H.M.K. is the Chair of the Leukaemia Department at MD Anderson Cancer Center. He joined the institution in 1981 as a fellow and over the past 38 years has continued his clinical-translational research in leukaemia. He is also a Special Fellow on Healthcare Policies at the Rice Baker Institute and advocates for health-care issues pertinent to patients with cancer and leukaemia.

A.S.K. is a Professor of Medicine at Harvard Medical School and Director of the Program On Regulation, Therapeutics, And Law (PORTAL) in the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital. PORTAL is an interdisciplinary research centre focusing on intersections among prescription drugs and medical devices, patient health outcomes, and regulatory practices and the law.

E.V.S. is the Chairperson and Founder of Friends of Cancer Research, and in addition is the Chair of the Board of Directors at the Reagan–Udall Foundation for the FDA, board member of the Foundation for the National Institute of Health, where she chairs the Portfolio Oversight Committee, and is on the Board of Governors of the Patient-Centered Outcomes Research Institute (PCORI). She also holds leadership positions with a broad range of cancer advocacy, public policy organizations and academic health centres including: MD Anderson Cancer Center External Advisory Board, the Duke University Cancer Center Board of Overseers, and The Sidney Kimmel Comprehensive Cancer Center Advisory Council.

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The work of R.K. is funded in part by the Joan and Irwin Jacobs Fund and by National Cancer Institute grant P30 CA023100. The work of A.S.K. is funded by Arnold Ventures, the Harvard-MIT Center for Regulatory Science, and the Engelberg Foundation.

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Correspondence to Razelle Kurzrock, Hagop M. Kantarjian, Aaron S. Kesselheim or Ellen V. Sigal.

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Competing interests

R.K. receives research funding from Foundation Medicine, Genentech, Grifols, Guardant Health, Incyte, Konica Minolta, Merck Serono, Pfizer and Sequenom, and consultant fees from Actuate Therapeutics, Genentech, LOXO, NeoMed and X-Biotech. She receives speaker fees from Roche and has an equity interest in Curematch and IDbyDNA. H.M.K. has received research funding from Abbvie, Agios, Amgen, Ariad, Astex, BMS, Cyclacel, Daiichi–Sankyo, Immunogen, Jazz pharma, Novartis and Pfizer, and declares honoraria from Abbvie, Actinium, Agios, Amgen, Pfizer and Takeda. The other contributors declare no competing interests.

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All comments by Hagop Kantarjian are his personal opinions and do not reflect the opinions of his institutional affiliation.

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Kurzrock, R., Kantarjian, H.M., Kesselheim, A.S. et al. New drug approvals in oncology. Nat Rev Clin Oncol 17, 140–146 (2020).

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