Can patients drive advanced evidence generation?

Enhanced evidence in rare diseases is crucial for advancing the development and evaluation of new therapies, strengthening our understanding of disease, and improving clinical decision-making. Danish patient-led nonprofit organization CompCure is making progress toward establishing a new standard in international evidence generation to secure better health outcomes for patients affected by two rare complement-mediated kidney diseases. The platform they have established could eventually benefit patients with other rare diseases.

Over the last three years, we have established a global registry and cohort study to support the development and optimal use of new therapies, while helping to ensure early diagnosis and effective therapeutic interventions targeted to the specific needs of each patient.

A recent milestone achieved by CompCure illustrates the potential of patient-led initiatives, including when it comes to advanced evidence generation. CompCure focuses on two rare complement-mediated kidney diseases, C3 glomerulopathy (C3G) and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN). Over the last three years, we have established a global registry and cohort study to support the development and optimal use of new therapies, while helping to ensure early diagnosis and effective therapeutic interventions targeted to the specific needs of each patient. Recently, our registry surpassed 400 enrolled patients from more than 20 countries, and it is currently being evaluated by the European Medicines Agency (EMA) for regulatory certification, which could allow it to be used as a control arm in clinical trials.

The registry from CompCure shows how the commitment and ambition of patients can contribute to scientific progress and the establishment of real-world evidence in an area defined by complexity, heterogeneity and high unmet medical needs. 

The size of the evidence base matters. However, in rare diseases like C3G and IC-MPGN, where incidence is estimated at 1–2 cases per million, it is difficult to gather the comprehensive evidence needed to advance scientific understanding, support innovation and optimize its application in clinical practice. The registry from CompCure shows how the commitment and ambition of patients can contribute to scientific progress and the establishment of real-world evidence in an area defined by complexity, heterogeneity and high unmet medical needs. 

CompCure was founded by parents whose daughter was diagnosed with IC-MPGN. The association has evolved into an international organization working at the intersection of patient engagement, clinical research, and evidence generation, primarily through close collaboration with the European Rare Kidney Reference Network (ERKNet) and other organizations committed to addressing the unmet needs of patients with rare kidney diseases. 

Data and evidence can help shape a virtuous circle that promotes scientific progress in an underserved disease area. As interest in rare kidney diseases grows, access to data and real-world evidence becomes increasingly important. And as the understanding of the diseases advances, research and development programs can become further de-risked and thus more attractive and more likely to succeed.

Patients no longer wish to just be the recipients of care; they want to be an active part of the development and definition of what optimal and appropriate care looks like.

There are several reasons why patient-led initiatives can make unique contributions to the generation of high-quality real-world evidence that supports researchers, clinicians, regulators, industry and, ultimately, the broader community. Not only can they help drive and promote such initiatives; they can also support the interpretation of the findings and help shape guidelines and relevant recommendations to improve patients’ overall outcomes and quality of life. Patients no longer wish to just be the recipients of care; they want to be an active part of the development and definition of what optimal and appropriate care looks like.

Our multidisciplinary network of experts, physicians, scientists, and patients is in ongoing contact with various stakeholder groups from around the world who seek evidence to support their understanding, definition, and access to optimal care.

When decisions about patients’ lives are being made, patients have the strongest interest in long-term, evidence-based approaches. Our multidisciplinary network of experts, physicians, scientists, and patients is in ongoing contact with various stakeholder groups from around the world who seek evidence to support their understanding, definition, and access to optimal care. That is why evidence generation is a key priority that requires collaboration around shared objectives of driving scientific progress, securing access to care, and ultimately helping patients live the happiest and most fulfilling lives possible. Patient-driven initiatives can, among other things, help establish new standards for worldwide evidence generation for the benefit of all people affected by rare diseases.