Only 1 in 5 Cancer Drugs Receiving FDA Accelerated Approval Have Proven Benefits in Confirmatory Trials

MedicalResearch.com Interview with:

Bishal Gyawali  MD PhD Med Onc. Asst. Professor 

Dr. Gyawali

Bishal Gyawali  MD PhD

  • Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women’s Hospital and Harvard Medical School, Boston, Massachusetts
  • Department of Oncology, Department of Public Health Sciences, and Division of Cancer Care and Epidemiology, Queen’s University, Kingston, Ontario, Canada

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Accelerated approval pathway from the FDA allows cancer drugs to come to market sooner by showing improvement in surrogate measures such as change in tumor size. Surrogate measures do not reflect clinical benefit in terms of living longer or feeling better. So, when a drug receives accelerated approval, the drug is required to undergo a confirmatory trial to confirm that true clinical benefit from the drug actually exists.

Last year, a paper from the FDA argued that accelerated approval pathway is working effectively because 55% of such drugs confirmed clinical benefit. However, we saw that most of those drugs were actually improving only a surrogate measure even in confirmatory trials. So the confirmatory trials were not confirming clinical benefit but actually confirming benefit in a surrogate endpoint. We investigate that issue in our study using updated results from the confirmatory trials that were ongoing at the time of FDA review.

Our main finding is that only one-fifth of cancer drugs that received accelerated approval actually improved overall survival later in confirmatory trials. For, 20% of other drugs, the confirmatory trials tested the same surrogate endpoint as did the preapproval trial. For another 21%, the confirmatory trial showed benefit in a surrogate endpoint different from the one used in preapproval trial. Furthermore, when drugs fail to confirm clinical benefits in confirmatory trials, they still continue to remain on market.  Continue reading

Cancer Drug Trials: Does Changing the Endpoint from Overall Survival Hasten the Approval Process?

MedicalResearch.com Interview with:

Emerson Chen, MDChief Fellow, Hematology-Oncology, PGY-6Oregon Health & Science University

Dr. Chen

Emerson Chen, MD
Chief Fellow, Hematology-Oncology, PGY-6
Oregon Health & Science University

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Many cancer drugs are approved annually giving the appearance of innovation; however, some drugs may have been approved because of a lower bar. Use of lesser endpoints like response rate (how tumor shrinks) and progression-free survival (how tumor has delayed growth) have been proposed to speed trials when compared against traditional endpoints like overall survival (how long patients might live).

Using published trials that led to cancer drug approval from 2006 to 2017, we estimated how long it would take to get each of these three endpoints across all cancer drugs and indications to see how much time we could save by using these weaker but faster endpoints.

We see that many trials using overall survival used less time than anticipated, and many trials using response rate or progression-free survival actually took quite a bit of time.  In part that is due to researchers needing to document the duration of the response. But, whatever the reason, the time to get each of the three endpoints is actually more similar than different, and we estimate that our current use of  these faster endpoints are saving us only 11 months compared to using only overall survival.

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Costs of Older Cancer Drugs Have Risen More Than New Medications

MedicalResearch.com Interview with: Dr. Sham Mailankody, MBBS

Dr. Sham Mailankody

MedicalResearch.com Interview with:
Dr. Sham Mailankody, MBBS
Memorial Sloan Kettering Cancer Center

MedicalResearch.com: What is the background for this study?

Response: The high price of older drugs has been increasingly criticized in part because of recent dramatic price hikes. There are some well known examples like pyrimethamine and more recently EpiPen. Whether and to what degree examples like pyrimethamine represent a common problem or exceptional cases remains unknown. Using Medicare data available for Part B, we sought to analyze the change in average sales price of cancer drugs between January 2010 and January 2015, and whether older drugs were more likely to undergo price increases than newer drugs.

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