A personalized medicine approach to clinical cancer therapy does not necessarily translate into a patient-centered approach as discussed in part 1 of this series. Currently most treatment strategies use objectives endpoints such as survival instead of patient “opinions” on quality of life. This latter approach to cancer management (of using patient-reported outcomes) is important and is increasingly reflected by the change in the attitude of regulatory bodies the world over. Dr. Sandeep Pingle discusses in this second article of this series how a patient-reported outcomes approach to clinical management begins early, with drug development.

As reported in a recent opinion piece in the New England Journal of Medicine (1), most drug labels and clinical trials, particularly cancer-related, do not include patient perspectives about a particular therapy. In other words, the current cancer drug development platforms overlook patient-reported outcomes (PRO), a key element of clinical management. When a new therapeutic agent is being developed and tested in clinical studies, pharmaceutical companies need to consider inclusion of patient-reported outcomes to get that perspective into research and product development. The US Food and Drug Administration (FDA)  recently issued guidelines on the inclusion of patient-centered outcomes during the clinical development of new cancer therapeutics.

The recent Patient Protection and Affordable Care Act (2010) created a Patient-Centered Outcomes Research Institute (PCORI). This institute comprises a committee that is responsible for developing methodological standards for research involving PROs.

Inclusion of Patient-Reported Outcomes for Drug Labels

The recent development of the drug ruxolitinib (used for management of myelofibrosis) prominently included PRO as a factor during clinical testing. Based on this testing, valuable PRO information obtained for this drug was included on the drug labels that companies typically use for marketing. These labels are an important source of information for the prescribing physicians and oncologists. This drug is the first cancer drug in over a decade that included PRO information on its drug label.

Patient-centered outcomes and drug development

Inclusion of patient-reported outcomes is recommended by US FDA for cancer drug development. (Source: http://dailymed.nlm.nih.gov/)

Though not yet a routine measure, PROs are being recommended by US FDA for cancer drug development. The use of PRO in routine cancer drug development entails development and rigorous evaluation of PRO instruments, tools to capture PRO data. A PRO instrument may be a questionnaire or any documentation that a patient uses to record data about treatment benefits or risks.

PRO-based drug development and clinical management is important for cancer patients as longevity increases due to availability of better therapeutics. This perspective is valuable, as it takes into consideration a patient’s quality-of-life – a patient’s perception of illness and treatment on physical, psychological and social aspects of life. Further, insurance companies consider this valuable information since patients who improve symptomatically and have better HRQL have fewer needs for supportive services.

Including patient perspective in cancer drug development would be a desirable shift from current methods. Hopefully, recommendations from regulatory agencies along with pressure from physicians and patients will hell drive this change.

References Cited:

  1. Basch, E (2013) Toward Patient-Centered Drug Development in Oncology. New England Journal of Medicine 369:397-400. DOI: 10.1056/NEJMp1114649