The Patient Matters: Thanks FDA

Many of us involved in the biopharmaceutical and medical device industry do not often consider regulators the cutting edge folks—the people who lead the charge. Times have changed. FDA has led the way in pushing us to focus on the patient.

Jean Paty, PhD

How? By releasing the final "Guidance for Industry, Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims" (or PRO guidance) in December 2009. FDA had previously released the draft version of this guidance in February 2006.

So, what's the big deal? FDA releases Guidance for Industry documents all the time. This PRO guidance is different: it has almost single-handedly driven our industry to take note of the patient—to listen, understand, and document the patient's perspective during medical product development. The momentum started building following the release of the draft guidance in February 2006. In the almost three years between draft and final guidance, FDA regularly and actively communicated its key message: listen to the patient.

In the past year, following the release of the final version of the guidance, significant strides have been made toward focusing on the patient during product development. Concretely, what has happened? The answer lies in how the specific recommendations in the guidance have had an impact on product development strategy. Here is how the story unfolded...

The guidance delineates FDA's current expectations for PRO instruments (that capture data directly from the patient with no interpretation by a clinical professional) that will be used to support labeling language in medical products. The recommendations from FDA are based upon the discipline of developing PRO instruments in psychology and other areas—psychometrics. There is nothing new or surprising in terms of what should be done to develop and evaluate PRO instruments.

Pleasantly surprising is that the PRO guidance has legitimized PROs, elevating their importance in product development planning. The mere fact that the FDA finalized this guidance has given a clear message to the industry that the patient perspective is important enough to be a formal part of the regulatory-based communication of a product: the label. This has led many pharmaceutical companies to re-evaluate their hierarchy of endpoints. In the past year, we have seen over a dozen pharmaceutical companies that have elevated PROs to be alongside other primary or key secondary biomarkers or physician-assessment endpoints. Such companies are seeking PRO-based labels for their new products. They have worked closely and collaboratively with FDA on the PRO instruments that will support these labels. These companies are taking advantage of the opportunity to clearly communicate the value of their product to the people who matter most—patients.

The elevation of PROs in clinical programs to endpoints that will form the basis of labeling claims has had a follow-on effect within pharmaceuticals. When an endpoint, or class of endpoints, is regularly part of the label plan, or target product profile, it starts to become part of the strategic planning for pipelines within a pharmaceutical company. Multiple pharmaceutical companies have adopted PROs as part of their product strategy within and across portfolios. In fact, some of the largest pharmaceutical companies have now made consideration of PRO endpoints as part of their documented process for developing a product strategy. PROs are strategic. The patient benefits.

Thank you, FDA.

Jean Paty, PhD Senior Vice President invivodata, inc. www.invivodata.com