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Ann Oncol. 2017 May 1;28(5):1111-1116. doi: 10.1093/annonc/mdx053.

Clinical benefit, price and approval characteristics of FDA-approved new drugs for treating advanced solid cancer, 2000-2015.

Vivot A1,2, Jacot J1,2, Zeitoun JD1,3,4, Ravaud P1,2,5,6, Crequit P1,2, Porcher R1,2,6.

Author information

1
METHODS Team, UMR1153 Epidemiology and Statistics Sorbonne Paris Cité Research Center (CRESS), INSERM, Paris, France.
2
Clinical Epidemiology Unit, Hôtel Dieu Hospital, Greater Paris University Hospitals (AP-HP), Paris, France.
3
Proctology Department, Croix Saint-Simon Hospital, Paris, France.
4
Gastroenterology and Nutrition Department, Saint-Antoine Hospital, Greater Paris University Hospitals (AP-HP), Paris, France.
5
Department of Epidemiology, Columbia University Mailman School of Public Health, New York, USA.
6
Department of Public Health, Paris Descartes University Medical School, Paris, France.

Abstract

Background:

Prices of anti-cancer drugs are skyrocking. We aimed to assess the clinical benefit of new drugs for treating advanced solid tumors at the time of their approval by the US Food and Drug Administration (FDA) and to search for a relation between price and clinical benefit of drugs.

Materials and methods:

We included all new molecular entities and new biologics for treating advanced solid cancer that were approved by the FDA between 2000 and 2015. The clinical benefit of drugs was graded based on FDA medical review of pivotal clinical trials using the 2016-updated of the American Society of Clinical Oncology Value Framework (ASCO-VF) and the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Characteristics of drugs and approvals were obtained from publicly available FDA documents and price was evaluated according to US Medicare, US Veterans Health Administration and United Kingdom market systems.

Results:

The FDA approved 51 new drugs for advanced solid cancer from 2000 to 2015; we could evaluate the value of 37 drugs (73%). By the ESMO-MCBS, five drugs (14%) were grade one (the lowest), nine (24%) grade two, 10 (27%) grade three, 11 (30%) grade four and two (5%) grade five (the highest). Thus, 13 drugs (35%) showed a meaningful clinical benefit (scale levels 4 and 5). By the ASCO-VF which had a range of 3.4-67, the median drug value was 37 (interquartile range 20-52). We found no relationship between clinical benefit and drug price (P = 0.9). No characteristic of drugs and of approval was significantly associated with clinical benefit.

Conclusion:

Many recently FDA-approved new cancer drugs did not have high clinical benefit as measured by current scales. We found no relation between the price of drugs and benefit to society and patients.

KEYWORDS:

costs and cost analysis; neoplasms/drug therapy; prescription drugs; relative value scales; value-based purchasing

PMID:
28453694
DOI:
10.1093/annonc/mdx053
[Indexed for MEDLINE]

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