Collaboration Key to Modernizing Clinical Trials

Intense pressure to streamline the drug development process by making clinical research more efficient is prompting a wave of joint initiatives among pharmaceutical companies, government agencies, and academic medical centers. Sponsors are identifying precompetitive areas that can benefit from combining data and resources, while the Food and Drug Administration is encouraging public-private partnerships to develop data standards, biomarkers, and eSubmissison methods.

Support comes from the National Institutes of Health's National Center for Applied Translational Sciences (NCATS), which is collaborating with eight pharma companies on drug repurposing programs and building teams to tackle a range of projects involving rare and neglected diseases. At a November Brookings Institution conference on "New Policy Directions for Biomedical Innovation," NCATS Director Christopher Austin also cited efforts to establish interoperable databases, common IRBs, and specialized research centers to support a shift away from the vertically integrated research model, which "does not work anymore." A squeeze on resources at federal agencies and industry, moreover, is prompting patient and disease groups to play a larger role in funding collaborative research efforts, while healthcare systems are looking to utilize extensive patient data banks to support research that will improve patient care.

Sharing information

In September, 10 big pharma companies unveiled the TransCelerate BioPharma collaboration to speed up new drug R&D by seeking standardization in data collection, study site qualification and monitoring, investigator training, and procurement of marketed drugs for use in comparative trials (see ACT "View From Washington," November 2012). TransCelerate recognizes it will benefit from collaborative efforts already underway by the Clinical Data Interchange Standards Consortium (CDISC); the Critical Path Institute (C-Path); the Clinical Trials Transformation Initiative, headed by Duke University and FDA; the Innovative Medicines Initiative (IMI) in the European Union; and others.

One recent offshoot is the global Investigator Databank, designed to provide information on the training, skills, and experience of investigators and clinical sites so every sponsor doesn't have to start from scratch in forming study networks for new trials. Janssen R&D clinical trial innovation head Andreas Koester announced in November that Merck and Eli Lilly were joining this effort to reduce redundant site assessments and repeat investigator training in good clinical practices. Other biopharma companies and contract research organizations may join the project with an eye to lowering the administrative burden on investigators sufficiently to expand the pool of trained clinical researchers in the United States and overseas.

Separately, several leading pharma companies agreed in October to share clinical trial data from cancer studies in a new database dubbed Project Data Sphere. Launched by the CEO Roundtable on Cancer Life Sciences Consortium headed by Sanofi CEO Chris Viehbacher, participants will provide historic study data to facilitate comparisons of different treatment regimens.

Setting data standards

The Cancer Roundtable and other research streamlining initiatives stand to benefit from data standards for collecting and transmitting study data. To this end, CDISC, C-Path, and FDA officially announced the Coalition for Accelerating Standards and Therapies (CFAST) at the October CDISC International Exchange meeting. Added support comes from IMI, Japan's Translational Research Informatics Institute, the Bill and Melinda Gates Foundation and, other research and disease groups.

CFAST's stated goal is to map standards for 57 therapeutic categories in five years. That ambitious schedule will be hard to meet, as debate continues over data transport standards for submitting files to FDA, audit trails, data integration, and the need to reduce data file size, among other issues. In the past 10 years, CDISC has developed and posted only three therapeutic area (TA) data standards, for Alzheimer's disease, pain, and tuberculosis. Additional standards are under review for cardiovascular disease, Parkinson's, polycystic kidney disease, and virology; diabetes and asthma top the CFAST priority list.

Yet, the FDA Safety and Innovation Act (FDASIA) provides a strong impetus for moving forward. The legislation calls for the development of standardized clinical data terminology through an open process, and for FDA to publish guidance to implement these standards. In addition to accelerating clinical research, TA standards will help FDA provide more consistent reviews, more quantitative approaches to benefit-risk assessment, and more timely post-market safety analysis. At the CDISC exchange meeting, Janet Woodcock, Director of the Center for Drug Evaluation and Research, noted that FDA receives nearly 1,300 datasets a week and needs standards to accomplish a much-needed shift to eSubmissions by 2017 to meet the FDASIA goal.

—Jill Wechsler