Annual Meeting: Patient-Centered Outcomes Research Institute (PCORI)

The first annual meeting of PCORI was held in Washington, DC on October 6-8.  1,100 researchers, patients, caregivers, health system and industry representatives, and others were present to hear updates on patient-centered comparative clinical effectiveness research.  PCORnet is a “network of networks” that leverages databases and experience across diseases through Clinical Data Research Networks (CDRN) and Patient Powered Research Networks (PPRN).  


PPMD's DuchenneConnect registry operates as one of the PPRNs within PCORnet. Our aim is to shift health research from researcher-driven to patient-centered. Holly Peay spoke on PPMD's DuchenneConnect Patient Powered Research Network, emphasizing how DuchenneConnect has leveraged patient voice and data to advance understanding of Duchenne and development of new therapeutics.  


As we noted in a blog in August, PPMD received phase 2 PCORI support of DuchenneConnect in August. Taken together, our participation in PCORnet will enable researchers to conduct clinical research more quickly and less expensively than is now possible, and will ensure that research focuses on the questions and outcomes that matter most to patients and those who care for them.  We hope to work with the Duchenne community and PCORnet members to address important, but relatively understudied, questions in Duchenne, including the areas of cardiomyopathy, bone health, and broader quality of life. 


An important symposium at the PCORI annual meeting focused on the topic, "Data Access in Open Science."  While strongly maintaining anonymity of patient data, a central principle of PCORI is to improve quality of life by promoting the sharing of patient data.  Francis Collins, the NIH Director, showed how NIH has promoted data sharing, thereby seeking to prevent siloing and fragmentation of data, and ensuring public access to research results.  


One speaker at the symposium said that: "Good science is how we avoid fooling ourselves, even when we have the incentive to do so, financial and otherwise."  Good science is also shared science—the types of questions now facing us in Duchenne will benefit from data that is not only rigorously collected and evaluated, but data that is pooled, because our broader questions will require information from more than just single studies.  The principles of rigorous science and data sharing are at the core of PPMD’s mission (e.g., our support for replication studies to ensure validity of preclinical data that drives clinical trials and our Duchenne Regulatory Science Consortium design to pool data and develop a disease progression model to improve clinical trials). 


Nearly every speaker in the PCORI symposium emphasized the importance that data, your data, from clinical studies and trials is shared.  There have now been many cases where failure to allow others to independently evaluate data has led to biased interpretations or worse (sometime fatal—see Vioxx). Moreover, years after the end of a study, it "might be hard to find the investigator and they may not be able to access the data later."  It was also noted that: "We don't want the research [data] scattered all over the world."  While single study publications are the currency of academic research, pooling of data from many studies is the only path to answering our broader, more critical questions in Duchenne.


Through data aggregation in DuchenneConnect and in the Duchenne Regulatory Science Consortium, PPMD is working hard to ensure that your privacy is maintained, while the availability and use of clinical data is not a barrier to improved management and treatment of Duchenne. Our participation in PCORnet is advancing that mission.  As an aspirational goal for Duchenne, let's continue to find ways where anonymized patient data can be shared at a level where much more than the original intent of individual studies can be achieved.  It’s the only way forward…


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