Critical Path Institute, founded in 2005 in Tucson, Arizona, is an independent, non-profit organization dedicated to bringing scientists from the FDA, industry and academia all together to collaborate and improve the drug development and regulatory process for medical products. Now in it’s tenth year, C-Path President and CEO Martha Brumfield, PhD shares an update on the progress being made.
We have experienced tremendous growth this past year due to increased demand for C-Path’s collaborative model that informs and advances medical innovation and regulatory science. In just five months, we have added four new consortia tackling a wide range of important issues:
- At the request of the FDA in 2013, C-Path succeeded in launching the International Neonatal Consortium in May to meet the challenges involved in developing and testing new therapies for newborn babies.
- A few weeks later, C-Path launched the Pediatric Trials Consortium with the goal of forming a new, independent organization that will develop a network of clinical trial sites with the specific capability to conduct registration quality clinical trials for new medical products for children.
- In August, C-Path partnered with Parent Project Muscular Dystrophy to form the Duchenne Regulatory Science Consortium with the aim of creating drug development tools for the devastating disease of Duchenne Muscular Dystrophy.
- In partnership with Parkinson’s UK in September, C-Path created the Critical Path for Parkinson’s Consortium, which is dedicated to developing tools that will enable greater efficiency in planning and designing clinical trials in early Parkinson’s disease.
C-Path is now comprised of 12 worldwide consortia representing 67 member organizations and over 1300 scientists, all working collaboratively to advance regulatory science across a variety of platforms. In addition, our solid reputation in the critical areas of data standards, curation, and database management has been spreading rapidly and is now recognized by institutions like the World Health Organization.
This increased demand has necessitated a net increase of 16 full and part-time employees since January. I view this growth as a ringing endorsement of our reputation as a trusted and independent organization dedicated to solving tough problems the only way they can be solved: collaboratively.
And, importantly, our consortia have achieved significant regulatory milestones from the U.S. Food and Drug Administration (FDA) including:
- A qualification decision issued to the Polycystic Kidney Disease Outcomes Consortium for using total kidney volume (TKV) as a prognostic biomarker in clinical studies of new therapies for Autosomal Dominant Polycystic Kidney Disease (ADPKD), a debilitating genetic disease affecting 600,000 Americans and 12 million worldwide. The use of TKV will help identify patients likely to experience a progressive decline in renal function. This is great news for both patients and those developing treatments, as there is only one medication developed to treat ADPKD, called tolvaptan, which has been approved in Europe, Japan, and Canada, but not yet in the United States.
- Letters of Support to the Coalition Against Major Diseases (CAMD) for three Alzheimer’s disease biomarkers—two that are measurable proteins in cerebrospinal fluid and one, hippocampal volume, as measured by an MRI imaging test. By using these biomarkers, clinical trial sponsors have more confidence that individuals that lack pathologic hallmarks of the disease will not be enrolled in clinical trials, thereby minimizing exposure for those who do not have the disease and increasing the chance of a successful trial. These important Letters of Support provide biomarker sponsors with an indication that the regulatory agencies support ongoing research into their utility.
- A Letter of Support to CAMD for a Parkinson’s disease (PD) biomarker – Dopamine Transporter Activity as assessed by single-photon emission computed tomography (SPECT) imaging. Low levels of dopamine transporter binding have the potential to indicate that people with motor dysfunction are more likely to show clinical decline of PD-specific outcome measures. Using this molecular imaging biomarker is a way to identify people living with Parkinson’s disease for enrollment in clinical trials that are likely to be at the earliest stages of PD.
- Letter of Support to the Predictive Safety Testing Consortium (PSTC) for four biomarkers to monitor for drug induced skeletal muscle injury in an exploratory context. With further study and data collection, the intent would be to use the biomarkers to enhance predictability when used in conjunction with serum creatinine kinase and aspartate transaminase.
We have also achieved significant regulatory milestones with the European Medicines Agency (EMA) and have expanded our presence with the opening of a European office in London to help advance collaborations with important European-based partners. Regulatory milestones include:
- A positive qualification opinion to C-Path’s Polycystic Kidney Disease Outcomes Consortium for total kidney volume (TKV) as a prognostic biomarker to select patients for clinical trials of new therapies for Autosomal Dominant Polycystic Kidney Disease (ADPKD).
- A positive qualification opinion on the Hollow Fiber System for Tuberculosis (TB). This innovative model aids developers by informing which drugs to combine and at what doses in order to effectively fight TB. This qualification effort, led by the Critical Path to TB Drug Regimens (CPTR), will speed the model’s adoption by drug developers who can now be assured that European regulatory bodies will accept supportive data from research using this method.
- Letter of Support to PSTC for the same four biomarkers mentioned above to monitor for drug induced skeletal muscle injury.
Each of these milestones represents an enormous amount of work on the part of the members of these consortia and I am extremely proud of all they have accomplished. And, there is more to do!
As a not-for-profit organization, we have only been able to make these advances thanks to your financial and moral support. With the demand for C-Path’s work on the rise, more resources will allow us to continue to grow and obtain greater efficiencies as we continue to find more ways to leverage lessons learned across our consortia. As the year’s end approaches, please consider a tax-deductible contribution to help us continue our positive trajectory.