I got a message just this morning telling me that the evolving Patient Centered Outcomes Research Institute (PCORI) is still looking for a wide variety of “non-scientist stakeholders,” including patients, caregivers, and advocacy group representatives, to act as reviewers of research grant applications submitted to PCORI in response to their recent funding announcements.

This is an important opportunity for the rare disease community to give detailed input on specific grant applications being submitted to PCORI and thus to help to shape the type of research that PCORI will support now and into the future. The ability to accurately monitor and assess the implications of patient outcomes in response to new therapies is of particular relevance to all members of the rare disease community. For many patients with rare diseases, the skills needed to manipulate a computer “mouse” or to use an iPad may be of much greater relevance than how far they can walk during a 6-minute walk test, but we have yet to validate this type of clinical trial endpoint as a measure of the effectiveness of drug therapy.

No one knows more about how small improvements in the ability of children with rare disorders to accomplish certain types of tasks can benefit their own lives and the lives of those around them than their caregivers and those young patients themselves. Making sure that PCORI-funded research can benefit from and take account of such knowledge may also be important to others outside the rare disease community too, and so I would encourage anyone who is interested to learn more by clicking here. This link will also take you to a page where you can submit your application to become a reviewer for PCORI.

Just because you have never done anything like this before, don’t think you can’t. PCORI provides a variety of types of training to help new stakeholder reviewers. There are many areas where patients, caregivers, and advocates now work closely with scientists and researchers to review and make recommendations about grant funding applications, and I am personally acquainted with plenty of individuals who have made major contributions to the quality of clinical research by acting as a reviewer of this type — sometimes to their own considerable surprise. When it comes to patient-centered outcomes, the scientific and medical research community really does want patient and caregiver input. And you don’t have to take my word for it either … Just read this blog post on the PCORI web site talking about a recent workshop held by PCORI on how to build a patient-centered research community.