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Evolving insight into personalized therapy requires changes in health care delivery, reimbursement
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Editor’s Note: Dr. Chui’s editorial is in response to an article in the Feb. 10 edition, “Iniparib may approve benefits of gemcitabine-carboplatin in triple-negative breast cancer.”
The possibility that a therapeutic such as iniparib will benefit only a small absolute number of patients (ie, BRCA-positive) raises an important issue: Currently, widely employed breast cancer treatment strategies and clinical research have largely been based upon identifying and capitalizing upon the often small relative benefits a therapeutic may provide when applied across a general population of thousands or even tens of thousands of patients. As a society, we have been able to justify the use of treatments in breast cancer that may only provide a small relative benefit (in terms of relapse-free survival, OS, etc.) due to the high incidence of the disease.
For example, if we have a therapeutic that only provides a 2% benefit across a population, but we apply that therapeutic to 100,000 patients, then we have saved 2,000 lives. Of course, the limitation to this approach is that we have also inappropriately treated 98,000 patients in this example.
With the increasing scientific capacity to both identify the many (hundreds? thousands?) subtypes of breast cancer, and rationally develop targeted therapeutics for each of these subtypes, we will have to seriously contemplate the difficult task of re-structuring how we support the development and distribution of novel cancer therapeutics in this emerging era of personalized medicine.
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NCI Cooperative Group research organizations, once set up to demonstrate the small relative benefits of applying treatments across large patient populations, will need to reorganize their collaborative arrangements, financial reimbursement mechanisms, and criteria for conferring academic recognition to facilitate the process of finding examples of the “rare” cancer subtype that might benefit from a uniquely targeted therapy. Only then will sufficient numbers of ‘rare’ patients be identified and accrued to complete an appropriately powered clinical trial of a “personalized therapy.”
Revamping the process and benchmarks for accruing academic/intellectual merit will be particularly important, since no individual investigator or institution will have direct access to sufficient numbers of patients to complete a clinical trial in a “rare patient population” single-handedly.
Furthermore, reimbursement mechanisms for personalized anticancer therapeutics will need to reflect both (a) the significant financial resources necessary to develop these treatments; and (b) the potential limited numbers of patients who will ultimately receive these therapies.
A recent example of this situation has been the important recognition that anti-EGFR therapies appear to only benefit colorectal cancer patients who do not have KRAS mutations. This finding has allowed caregivers to better treat colorectal cancer patients with the appropriate agent. However, this also means that the pharmaceutical companies that invested significant resources into developing these therapies will only be able to recoup their investment through the treatment of a limited number of patients, rather than marketing these agents as a therapy for every colon cancer patient. Increasingly, in an emerging era of personalized medicine, nearly every tumor could become its own “orphan disease.”
Rationally developed therapeutics such as iniparib are exciting and may eventually demonstrate meaningful benefits to a subset of breast cancer patients currently lacking targeted therapy or therapies. The potential for the PARP-inhibitory strategy to only benefit a small subset of the breast cancer patient population (ie, BRCA-positive tumors) is just one example of a circumstance that will likely become increasingly common as the scientific and clinical development of cancer therapeutics progresses in this burgeoning era of personalized medicine. The current individual, institutional and societal incentives that promote superior health care research and delivery will need to quickly evolve to sustainably take advantage of our emerging scientific insights into the individuality of tumors.
Stephen Y. Chui, MD, is an assistant professor of medicine and director of breast cancer clinical research (acting), Knight Cancer Institute, Oregon Health & Science University.
Disclosure: Dr. Chui reported receiving research funding from Bayer BioOncology and GlaxoSmithKline; speaking honoraria from Genentech, Amgen, GlaxoSmithKline and Sanofi-Aventis; and consulting fees from Genentech, Amgen and GlaxoSmithKline.