According to the European Medicines Agency, a patient-reported outcome, or PRO “includes any outcome evaluated directly by the patient himself or herself and is based on patient’s perception of a disease and its treatment(s)”. PROs cover:
Today PROs are routinely incorporated in clinical trials and observational studies for regulatory purposes. Regulatory agencies have specific position statements on their preferred endpoint selection, including PROs for clinical trials; however, pharma companies are free to execute their trial design according to their preferences.
Although PROs are more and more frequently addressed by pharma companies in regulatory submissions, historic analysis (2006 – 2010) shows that of those products with PRO data reviewed by the Food and Drug Administration (FDA) in the US, only 14 (19%) had at least one PRO label claim granted. 4
In 2014, the American Society of Clinical Oncology (ASCO) had a workgroup focused on the use of PROs to improve the quality of treatment and care in oncology. However, earlier this year, when ASCO reviewed their value framework, they decided against including PROs. Their rationale was that current clinical trials haven’t adequately measured or reported PROs.5,6
If medical societies and regulators don’t further advocate the use of PROs, then who wants them? If PROs are not valued, then pharmaceutical companies will hardly spend the time, money and increased regulatory risk in gathering such data.
Patients, their families and patient advocate groups like having patient-relevant PROs when evaluating their treatment options. Taking cancer as an example, PROs are helpful for the patient wishing to determine the impact of a prospective treatment on their own quality of life. In the US, where patient co-payment and affordability is an important factor, patient-relevant PROs will inform difficult patient decisions as they attempt to trade off between cost/affordability and value to them.
In fact, for cancer patients, actual clinical benefits have been seen when symptom self-reporting (PROs) is utilized during their care. In a large controlled trial, benefits were demonstrated in outcomes, including quality of life, emergency room utilization, and survival.6
Take, for example, breast cancer physicians. 71% of physicians believe that patients with breast cancer consider keeping their breast as a top priority. Yet the figure from direct patient research (PROs) is only 7%.7 Thus, physicians could benefit from direct real-world evidence of patient views, which might be obtained through patient-reported outcomes.
Payers want outcomes-based evidence, and when they assess competing therapies, they view PROs as a potential way of differentiating products. However, they also recognize that there are few guidelines available on how healthcare decision makers should use and value PRO evidence.
As discussed earlier, products with a PRO-label claim, which manufacturers can leverage in their promotions, are in the minority.4 Current pharma practice shows that if the collection of PRO data doesn’t lead to incorporation of the data within the label (or summary of product characteristics), then PROs are not prioritized by the industry. However, for pharma to pay more than lip service to patient-centricity, PROs should be shifted from “nice to have” to essential.
Indeed, a patient-centered approach to healthcare is boosted by the incorporation of PROs not only in clinical trials but in overall disease management. But are the current PRO instruments adequate for a world of combination therapies, e-devices and integrated care? Probably not. At least not at the moment, if you take the recent example of ASCO not incorporating PROs into their value framework.6
In order to account for a patient’s perspective more systematically, and demonstrate value to all concerned stakeholders (including patients, physicians, payers and regulators), pharma should design and plan PROs early in the clinical development. Manufacturers need to ensure the right PROs are selected, risks are minimized and new PROs are appropriately validated from the inception of the clinical trials program, rather than just adding them as a post-script to the marketed drugs file.
Are you interested in more information? You might also like our blog article “Why pharma must understand the patient as a payer”.
 European Medicines Agency (EMA). Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man. The use of patient-reported outcome (PRO) measures in oncology studies. April 2016.
 Food and Drug Administration (FDA). Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. December 2009.
 EMA. Reflection Paper on the use of patient reported outcome 5 (PRO) measures in oncology studies. June 2014
 DeMuro C, Clark M, Doward L, Evans E, Mordin M, Gnanasakthy A. Assessment of PRO label claims granted by the FDA as compared to the EMA (2006-2010).Value Health. 2013 Dec;16(8):1150-5. doi: 10.1016/j.jval.2013.08.2293. Epub 2013 Oct 17.
 American Society of Clinical Oncology. www.asco.org/about-asco/press-center/news-releases/asco-value-framework-update. Accessed August 2016.
 Basch E, Deal AM, Kris MG, Scher HI, Hudis CA2, et al. Symptom Monitoring With Patient-Reported Outcomes During Routine Cancer Treatment: A Randomized Controlled Trial. J Clin Oncol. 2016 Feb 20;34(6):557-65.
 Lee CN, Dominik R, Levin CA, Barry MJ, Cosenza C, O’Connor AM, Mulley AG Jr, Sepucha KR. Development of instruments to measure the quality of breast cancer treatment decisions. Health Expect. 2010 Sep;13(3):258-72.