Outcomes # 3

Way to Patient-Centered Outcomes Research

Original article published on Assay Depot Blog, on 8 Oct 2013 here.

Patient perspective is a crucial element in addressing health and illness issues for individuals on therapy. This final article in the series follows up on the discussion about patient-reported outcomes and cancer by talking about the approach to patient-centered outcomes research.

Patient-centered outcomes research (PCOR) was highlighted in the 2010 Patient Protection and Affordable Care Act by the creation of an independent patient-centered outcomes research institute (PCORI). Such a patient-centered approach to medical research involves actively addressing the preferences, decision-making needs and characteristics of different patient populations.

A Methodological Committee that is part of the PCORI provides recommendations for standards that are necessary to carry out PCOR. For effective PCOR, it is important to address four distinct areas for development:

  1. Prioritize research questions – Identify and address crucial knowledge gap
  2. Use appropriate study design and analysis – Careful consideration of question at hand
  3. Incorporate patient perspectives – Engage patients throughout the research continuum
  4. Efficiently implement results – Design research with actionable results

Patient-reported outcomes (PRO) are important to further the idea of any PCOR. Hence reliable recording and analyses of PRO data is of utmost importance. Researchers can use as study endpoints findings that are recorded using well-defined and validated PRO instruments. More importantly, these findings can be placed on product labels and help guide both, the prescribing physicians and patients.

Specifically, efforts are being made to move drug development in the pharmaceutical and biotechnology world towards a more patient-centered approach. For this purpose, drug development research must use a design that considers patient perspectives. Further, the reporting of research needs to incorporate the patient perspective as well. Overall, a six-step approach has been put forth to achieve a patient-oriented drug development effort (see Table).

Drug Development Cancer
Approach to Patient-Oriented Drug Development; Table adapted from: Basch E. NEJM 2013; DOI: 10.1056/NEJMp1114649

In addition to implementing the steps outlined above, it is critical to change the mentality of drug development entities towards a patient-oriented approach. Equally important is the change in attitude of regulatory agencies. This dual change would help bring the patient perspective to the center of any discussion related to drug development and clinical management.

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