EXCLUSIVE: Cancer Therapy Benefits Realized Long After FDA Approval, Study Author Tells BioSpace (DHX)
6/26/2015 3:11:43 PM
June 29, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor
A new report last week that shows that the full therapeutic value of a cancer therapy is typically realized long after the drug’s initial FDA approval is a game-changer for treatment, one of the study’s authors told BioSpace (DHX) this week, as the standard of care in oncology continues to evolve.
The report, “The Value of Innovation in Oncology: Recognizing Emerging Benefits Over Time,” was commissioned by the Pharmaceutical Research and Manufacturers of America (PhRMA). It examines the pathways by which additional benefits of cancer therapies are revealed over time through ongoing research after initial U.S. Food and Drug Administration approval and through the use of therapy in real world treatment settings.
Perhaps most interesting? The report underscores that improvement in patient outcomes is generally observed over time, through an incremental process as experience is gained with new treatments and interventions, and longer term survival and other longer-term endpoints are examined.
“This is particularly seen in those therapeutics that the FDA may have granted accelerated approval pathway,” it said. “Biopharmaceuticals approved under these circumstances are granted approval contingent up continued research and clinical investigation of safety and efficacy. In many cases, this continued research and accumulation of evidence may demonstrate even greater benefits.”
BioSpace interviewed study author Thomas F. Goss, Pharm.D, senior vice president of Boston Healthcare Associates, and Nicole Sweeney, manager at Boston Healthcare Associates, about what this report means.
How long a timeline are we looking at for benefits to be realized?
The timeline for benefits to accrue can vary depending on a number of factors, including whether other indications are under evaluation for the drug at the time of initial approval, the availability of companion diagnostics (for some agents companion diagnostics have become available after approval), and what other regimens it may be combined with, to name a few. In general, these benefits are continuously realized over a long period of time. The graphics in the Appendix demonstrate that in some instances, the approval of an additional indication can be within a year or less (for example everolimus, ibrutinib, and ramucirumab)
Please break down the methodology of this study.
Our methodology included a “scoping phase” to validate these hypothesized pathways followed by in-depth research of published literature, clinical trials.gov database and publically available approval information from the FDA website.
Why do you think this is most obvious in therapeutics that the FDA may have granted accelerated approval pathway?
In oncology in particular, new therapies may undergo an accelerated approval pathway where determination of efficacy is based on surrogate or intermediate endpoints. Through ongoing study, after the medicine has been approved, additional survival and disease response benefits are often revealed that were not able to be assessed at the time of that initial approval.
We recognize that this is actually an indication of the success of the FDA’s approach to accelerating approvals in diseases with significant unmet need. This environment incentivizes (requires) additional research following an accelerated approval, with the additional benefits being realized over time. We have identified similar mechanisms of incremental value associated with innovation in other therapeutic areas including inflammation (RA), Diabetes, and HIV/AIDS and have written on these topics as well. Please let me know if you would like to discuss or clarify further.
How can additional value be recognized through a number of different pathways?
The intrinsic value of a drug continues to grow simultaneously through many pathways through both investment by innovators in the systematic evaluation of the drug through rigorous research (often continuous) over very long periods of time and the accumulation of data of the medicine’s use in real-life treatment settings.
Emerging advances in personalized medicine are driving increased utilization of diagnostic tests, for example, which may direct more precise treatment choices. Increasing understanding of the underlying biology of the disease is also revealing that many cancer medicines are useful in other types of cancers besides the one they were originally approved to treat.
As Rumors Swirl About GlaxoSmithKline Bid, Who Could Suitors Be?
Rumors are swirling that Swiss-based Roche (RHHBY) and U.S.-based Johnson & Johnson (JNJ) are eying the U.K. company for approximately $143 billion. But Roche and J&J aren’t the only companies though who have been thought could go after the elephant that is Glaxo.
Last month there was buzz that Pfizer Inc. (PFE) was considering acquiring Glaxo, a year after it failed to acquire AstraZeneca PLC (AZN). Just this month over a third of respondents in a poll conducted by BioSpace (DHX) believe that AstraZeneca PLC could be in the running to acquire struggling GlaxoSmithKline (GSK).
So BioSpace wants to ask our readers again what they predict for this new dealmaking bonanza. Will Glaxo go—and if so, to whom?
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