CRITICAL PATH INSTITUTE

CPAD works on drug development tools to advance new therapy development for Alzheimer's and Parkinson's diseases.

These tools include data standards, clinical data databases, disease progression models, prognostic biomarkers, and clinical outcome assessment instruments.

The Patient-Reported Outcome (PRO) Consortium is based upon a collaborative framework of appropriate stakeholders that develops qualified and publicly available PRO instruments for use in clinical trials in order to support labeling claims. The PRO Consortium was formed in December of 2008 and formally launched in March of 2009. The PRO Consortium’s membership is comprised of pharmaceutical companies along with representatives from the FDA, EMA and NIH who provide advice to the Coordinating Committee.

PRO instruments involve measurement of one or more aspects of a patient’s health status based on information gathered directly from the patient, without interpretation by physicians or others. Patients provide information concerning the impact of an intervention or therapy from their perspective. PRO instruments offer a means for capturing how a patient feels or functions with respect to her/his health, condition, or disease.

There are many disease areas for which adequate PRO instruments are not available. PRO instrument development is expensive, resource-intensive, and time consuming due to the extensive research and testing process. Under the direction of Stephen Joel Coons, PhD, the PRO Consortium provides a critical mass of experts with different skill sets, experiences, and perspectives. The PRO Consortium consists of scientists from C-Path, pharmaceutical, biotechnology, device, and diagnostic companies, FDA and National Institutes of Health (NIH).

Critical Path Institute has established the Electronic Patient-Reported Outcome (ePRO) Consortium, in cooperation with firms that provide electronic data collection technologies/services to the medical products industry for capturing patient-reported outcome (PRO) endpoints in clinical trials.

The mission of the ePRO Consortium is to advance the quality, practicality, and acceptability of electronic data capture (EDC) methods used in clinical trials for PRO endpoint assessment. To accomplish this mission, they provide a non-competitive, neutral environment to test the measurement equivalence of PRO measures migrated to or among alternative administration methods. The ePRO Consortium will work with the PRO Consortium to migrate the PRO instruments developed within the PRO Consortium to all relevant EDC platforms. Further, specification documents will be developed for the adaptation/migration of existing PRO instruments to the relevant EDC platforms. The Consortium also will provide guidance on methodological considerations for PRO instrument migration and adaptation.

The ePRO Consortium provides a pre-competitive space where a critical mass of experts can collaborate to generate measurement equivalence data, develop specification documents and data standards, and produce methodological guidance on measurement issues related to ePRO applications. The ePRO Consortium will work closely with C-Path’s PRO Consortium, a group of pharmaceutical companies working to develop novel PRO measures, to make the new measures available in multiple data collection formats. The overarching aim is to enhance public health by optimizing the value of PRO data in medical product evaluation and clinical decision making.

The Polycystic Kidney Disease (PKD) Outcomes Consortium is a successful collaboration between Critical Path Institute (C-Path), the PKD Foundation, Clinical Data Interchange Standards Consortium (CDISC), and four leading academic medical centers (Tufts University, University of Colorado – Denver, Emory University, and Mayo Clinic). Its mission is to promote research that will lead to the discovery of treatments for PKD and improve the lives of all it affects. The Consortium is led by C-Path and funded through a grant from the PKD Foundation and philanthropic donations. Additionally, a representative from the U.S. Food and Drug Administration (FDA) serves as an active advisor to the Consortium.

Autosomal Dominant PKD (ADPKD) is a debilitating genetic disease affecting more than 600,000 Americans and 12 million people worldwide and for which there is currently no known cure or effective treatment. Traditional endpoints of renal function only show changes very late in the course of the disease, making it difficult to assess the effectiveness of new medications. There is critical need for a biomarker that will assess disease progression at an earlier stage when patients may be more likely to respond to new therapies.

The primary goals of the PKD Consortium are to develop CDISC research data standards for PKD and to use clinical data from ADPKD patients collected over many years through patient registries and clinical trials to support the FDA-qualification of an imaging biomarker, Total Kidney Volume (TKV), as a biomarker in drug development trials.

Scientists will use the data collected to develop a disease progression model that will evaluate the relationship between TKV and the known complications of ADPKD, including rate of loss of kidney function, hypertension, gross hematuria, kidney stones, urinary tract infections, development of end-stage renal disease, and mortality. These analyses will be used to support the regulatory qualification of TKV as an accepted measure for assessing the progression of ADPKD in clinical trials in which new therapies are tested.

The CPTR Initiative is a public‐private partnership initiated in March 2010 by Critical Path Institute, the Bill & Melinda Gates Foundation (BMGF) and the TB Alliance. With participation from the pharmaceutical industry, academia and national and global government agencies, a major portion of this effort is called the CPTR Regulatory Science Consortium, led by C‐Path, which includes:

Developing and integrating data standards, Qualifying biomarkers through the FDA/EMA, Developing quantitative disease progression (natural history) models,
Creating disease response metrics and assays, and Developing new pharmacokinetic/dynamic measures of drug interactions.

The purpose of the CPTR consortium is to deliver a safer, more efficacious and faster‐acting TB regimen by developing and promoting innovative regulatory science essential for supporting new combination drug development.

Since the launch of CPTR in 2010, the consortium has made significant progress. It has actively engaged the FDA, which has released updated regulatory guide-lines for developing new drug regimens. The FDA continues to work to create a more favorable environment for regimen development. In 2011, CDER head Dr. Janet Woodcock wrote an opinion piece in the New England Journal of Medicine expressing her support for “co-development” of therapies for life-threatening diseases such as TB. Additionally, in November 2010, the TB Alliance launched the first-ever clinical trial of a novel combination drug regimen for TB. The trial tested three promising TB drug candidates together, before two of them have been approved and registered individually for use against TB. The study met its milestones, validating the approach to regimen development set forth by CPTR and highlighting the promise of a novel regimen.

Launched in December, 2012, MSOAC is C-Path’s newest consortium – another dynamic partnership formed to promote consensus science. Created jointly with the National Multiple Sclerosis Society, MSOAC will collect, standardize, and analyze data about MS with the goal of qualifying a new measure of disability as a primary or secondary endpoint for future trials of MS therapies.

The National Multiple Sclerosis Society recognized the gap in the MS treatment pipeline and decided to employ a method that has steadily gained support within the research community — consortia science. MSOAC brings stakeholders from industry, academia, patient representatives, and regulatory agencies together to spur development of drug development tools to assess the effectiveness of potential treatments for all forms of MS.