patient reported outcomes

The ability to capture real-world patient reported outcomes (PROs) has become a priority for developers in the clinical research space. Both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have published guidance and reflection papers on the use of PROs in the drug development and approval process..

But regulatory interest isn’t the only reason biopharma companies should be paying more attention to this data. PROs are a valuable resource across the drug development lifecycle, providing a baseline for current treatments, informing trial design and helping researchers deliver outcomes that will be meaningful to patients and physicians -- as well as regulators.

 These patient important outcomes can be used to more clearly link the value proposition of a treatment to the personal needs of the patient. For example, the Crohn’s and Colitis Foundation recently gathered patient reported outcomes from its members that shows patients with irritable bowel syndrome have more anxiety, depression, fatigue and sleep disturbance, and less social satisfaction than the general population, and that using corticosteroids made all of these outcomes worse. This kind of insight could directly impact clinical decision-making as well as influence researchers to include endpoints in trials that will measure outcomes that can have the greatest impact on patients’ lives.

Patient reported data can also be used to demonstrate the benefit and add contextual meaning to the impact of treatments on patient lives in support of approval and pricing efforts. A report published in 2013 shows studies with PROs as primary endpoints are far more likely to facilitate positive regulatory review and acceptance of PROs in support of labeling claims. The report also shows that inclusion of PROs as non-primary endpoints can also be used to provide support for primary endpoints. For example, a study may use a well-accepted clinician-reported outcome (such as in depression) and then include a PRO to measure patients’ perceptions of severity of the condition (such as the Beck Depression Inventory).

In related research, my colleagues at Quintiles conducted a study into the types of PRO claims that are most beneficial to clinical research, which they shared in a poster at the 2015 ISPOR European Congress in November. Their study shows the PROs that have the greatest impact on prescribing and reimbursement align directly with the competitive advantage a drug brings to the market, which will vary according to that value proposition – i.e. for one drug it might be significant impact on cost-effectiveness, whereas for others it might be tied to the reduction of adverse events, greater tolerance, or less pain.

All of these examples underscore the value that PRO data can bring to the clinical research process. The challenge now is figuring out the best way to collect and apply this data.

The right PRO platform for your study

Technology-based systems and online data gathering tools are making it faster, easier and cheaper for biopharma companies to engage with patient communities and capture patient reported data within their clinical research and commercialization programs. But these tools can still be improved upon.

At the 2016 BIO International conference this week, I will be participating on a panel discussing how we can better use technology to capture real world patient experience for comparative effectiveness. In that session we will explore how organizations can harness registries, social media and other technologies to capture PRO data, how to best integrate these resources with other clinical and claims databases, who should bear the cost of new technology for PRO collection, and how regulators’ statements on this topic may affect clinical research decision making.

There are still many questions to be answered about the most effective way to capture and use this data throughout the research process. I’m excited to gain insights from my colleagues on this panel, and to participate in a broader discussion with stakeholders from across the industry on how we can make the best use of this valuable information.