Focus Area: Capacity Building

[CAMBRIDGE, Mass., June 25, 2019]– OptumLabs and the MRCT Center announced that two academic institutions were awarded grants to support replication of established clinical trials through the use of electronic health record (EHR) and claims data, which could help improve clinical trial design and drug development.

The U.S. Food and Drug Administration (FDA) and other regulatory agencies are evaluating the potential role of real-world data to support approval of new indications and to satisfy post-approval requirements, as directed by the Prescription Drug User Fee Act VI and the 21st Century Cures Act.

The grantees, Brown University and Harvard Pilgrim Health Care Institute, will work to replicate two trials – ROCKET for Atrial Fibrillation and LEAD-2 for Type 2 diabetes control – using de-identified health care claims from commercial and Medicare Advantage plan participants and electronic health record (EHR)–derived data from a nationwide collection of care provider groups within the OptumLabs Data Warehouse. They will also examine the utility and possible benefit of combining claims data with EHR data to generate real-world evidence.

“There are significant opportunities for using real-world evidence and observational data to contribute to reducing costs while still fostering innovation,” said Paul Bleicher, M.D., Ph.D., CEO of OptumLabs. “These opportunities are even greater when we can bring together stakeholders from across industry, academia and government working together to examine new approaches for evidence generation.”

“While randomized clinical trials have been the gold standard of evidence generation, particularly for new drugs, we must learn how to use previously collected, real-world data to learn about the safety and effectiveness of existing medicines,” said Barbara Bierer, M.D., Professor of Medicine, Harvard Medical School, and faculty director of the MRCT Center. “This novel approach may not only bring new drug indications to market faster, but also reduce the need to involve people in trials for this purpose.”

An expert panel made up of members of ISPOR, National Pharmaceutical Council, Duke-Margolis Center for Health Policy and 11 pharmaceutical companies chosethe two winning teams to receive the $150,000 awards.

The grants were awarded as part of the Observational Patient Evidence for Regulatory Approval and uNderstanding Disease (OPERAND) initiative, which includes representatives from academia, industry and regulatory agencies, and will examine principles, methodologies, and appropriate utilization of real-world data in regulatory review and approvals of medical treatments.

OPERAND is being sponsored by Amgen, AstraZeneca, Merck, Optum, Pfizer, Sanofi and UCB BioSciences, Inc.

About OptumLabs

OptumLabs is a collaborative research and innovation center, dedicated to improving patient care and patient value through data-driven health care research, leading-edge data science and strong partner collaboration. OptumLabs was founded through a partnership between Optum, a leading health services and innovation company, and Mayo Clinic, a nonprofit worldwide leader in medical care, research and innovation.  AARP, a nonprofit, nonpartisan organization with membership of more than 37 million individuals, is its Founding Consumer Advocate Organization. Today OptumLabs has more than 25 collaborating organizations, representing stakeholders from across the health system.

About the Multi Regional Clinical Trials Center of BWH and Harvard (MRCT Center) is a research and policy center that addresses the ethics, conduct, oversight, and regulatory environment of international, multi-site clinical trials.  Founded in 2009, it functions as a neutral convener to engage diverse stakeholders from industry, academia, patients and patient advocacy groups, non-profit organizations, and global regulatory agencies. The MRCT Center focuses on pre-competitive issues, to identify challenges and to deliver ethical, actionable, and practical solutions for the global clinical trial enterprise. In addition, the Center is involved in several in-country engagements and has a long-standing commitment to training global regulators, investigators, and others.

On March 19, 2019, the Ministry of Health and Family Welfare of India released the “New Drugs and Clinical Trials Rules, 2019.”[1]  These rules included significant definitions, clarifications, opportunities, and expectations that, together, improved the clinical trial regulatory environment in India.

Several provisions are particularly notable: compensation provisions for death, permanent disability, and injury related to participation in a clinical trial; the DCGI through expert committee review, retaining the responsibility for determination of the quantum of compensation if any; publication by schedule for the amount of compensation; clarification of requirements for medical management of trial-related injury; procedure for post-trial access to investigational products determined to be beneficial to the participant; clarification of the requirement for audio- versus audiovisual recording of the informed consent process; definitions of academic trials and of vulnerable populations; certain provisions for research ethics committees; expedited review of the application for new clinical trials, including a 30 day review for any new drug that was discovered in India or will be manufactured and marketed in India; definition of special situations wherein the regulations for a new drug can be relaxed, abbreviated, or modified; introduction of pre- and post- submission meetings; penalties for non-compliance and the right of the aggrieved party to appeal; among other provisions.

Taken together, the rules constitute a significant advance for the country and encompass welcome clarifications for sponsors and investigators seeking to site clinical trials in India.

[1]New Drugs and Clinical Trials Rules, 2019, Ministry of Health & Fam. Welfare, Notification, G.S.R. 227(E), (March 19, 2019)

Initially created in 2006 by the World Health Organization, the African Vaccine Regulatory Forum (AVAREF) was created as an informal capacity building platform dedicated to improving the regulatory and ethical oversight of interventional clinical trials in Africa. In 2016, a revised governance structure was agreed upon and the remit of AVAREF was expanded to include all interventional clinical trials in Africa.

AVAREF seeks to enhance the efficiency and quality of regulatory and ethical reviews and inspections,  develop and disseminate harmonized guidelines and approaches, protect participant and patient safety, and improve the timeliness of regulatory decisions for all trials. As a  pan-African network, AVAREF is an important contributor to the African Medicines Regulatory Harmonization (AMRH) initiative and the future African Medicines Agency (AMA).  Dr. Bierer was invited to describe the work of the MRCT Center and to participate in discussions of capacity building and regulatory systems strengthening.