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CRITical Thinking is a blog written by staff, directors, and friends of the Collaboration for Research Integrity and Transparency (CRIT), a joint program of Yale Law School, Yale School of Public Health, and Yale School of Medicine. CRIT's mission is to promote health by improving the integrity and transparency of biomedical and clinical research.

This blog is published by and reflects the personal views of the individual authors, in their individual capacities. It does not purport to represent Yale University's institutional views, if any. No representation is made about the accuracy of the information, which solely constitutes the authors’ personal views on issues discussed. The information contained in this blog is provided only as general information and personal opinions, and blog topics may be updated after being initially posted.


CRITical Thinking Blog Series—Ensuring Safety, Efficacy, & Access to Medical Products in the Age of Global Deregulation

August 14, 2017

In June 2017, the Yale Collaboration for Research Integrity and Transparency (CRIT) in partnership with the European Public Health Alliance sponsored an international conference titled “Ensuring Safety, Efficacy, and Access to Medical Products in the Age of Global Deregulation.”

Over the next few months—twice a week, on Mondays and Wednesdays—we will be posting comments from the conference participants, which included more than 50 researchers, government officials, patient advocates, clinicians, ethicists, attorneys, and academics from North America, Europe, Africa, and Asia. Our hope is that this blog series will provide a range of perspectives and insight into the complex problems faced by the research and regulatory ecosystem.

For the first post in this series, we share the reasons that led us to convene this global meeting and the issues we hoped to tackle.

Since the passage of the Kefauver-Harris amendments to the U.S. Food, Drug, and Cosmetic Act in the wake of the thalidomide disaster of the early 1960s, drug companies have been required, prior to marketing, to prove that medical products are safe and effective for their intended uses. Though industry resisted these changes at the start, companies have complied with these requirements, and there were few significant modifications to the U.S. Food and Drug Administration’s regulatory mandates for almost three decades.

Beginning with AIDS activists in the 1980s, and with conservative politicians in the 1990s, in the words of Harvard professor Daniel Carpenter, a “rightward shift” began at the FDA, and the post-thalidomide framework began to unravel. The FDA was portrayed as slow and bureaucratic and blamed for stifling innovation in medical research and access to medicines for patients. Even after reforms at the FDA in response to AIDS activists, industry and its allies pushed for greater deregulation of the market for drugs, devices, and other medical products throughout the 1990s and 2000s. In December 2016, the U.S. Congress passed the 21st Century Cures Act, which signaled a shift towards relaxing the regulatory oversight of medical products in the U.S. This has occurred in the face of recent research, which has begun to document an erosion in the evidence base for new drugs and devices over the past several years.

The “rightward shift” in medical product regulation has not been limited to the U.S. In fact, many of the think tanks and industry lobbies that have pushed for reforms like the 21st Century Cures Act, are active in Europe and have urged the European Medicines Agency to take on new ways of speeding drug approval, particularly through specious proposals like Adaptive Pathways. Meanwhile, calls for international harmonization risk enshrining this “rightward shift” on a global scale.

While regulatory agencies in the U.S. and Europe have sped approval of new medical products at notable cost to the evidence base, a new movement to promote research and drug development (R&D) for neglected diseases (or diseases predominantly present in the global South) has emerged over the past few years. Interventions for neglected diseases have faced a special set of regulatory concerns. The lack of market for such interventions has meant that R&D of this sort has relied heavily on governments, foundations, and the cooperation of companies that perceive goodwill rather than profit as the primary aim. As a result, investment in clinical research on drugs for neglected diseases occurs in an exceptionally constrained context, where the ability to generate evidence may be limited by financial concerns, or the size of patient populations available for clinical study. This presents special regulatory challenges, which need to be explored.

How do we ensure the safety and efficacy of new drugs, medical devices, and other medical products as a whole, in a context where regulatory standards are being eroded? How do we reconcile the specific R&D needs of drugs and other products for neglected diseases with those for agents developed for industrialized markets in the global North? This meeting was meant to explore these issues and questions.

Finally, the weakening of evidentiary requirements for medical products comes in the context of rising drug prices in many countries, which means consumers and payors are being required to pay more and more for medicines about which they know less and less. Access to medicines is intricately linked to access to information about them. Patients don’t only need pills to take; they and their health care providers need to know how to best use them. In the quest for open science and data sharing to promote access to information, we must also ensure the rigor of the data obtained on these drugs through the clinical development process—rigor that is achieved through replication of studies and independent analysis of trial data. Transparency and this kind of research integrity go hand-in-hand: patients, health care providers, and payors need access to information, but that information has to be of the highest quality and reliability. While the meeting was about the erosion of the evidence base for medical products across the globe, pricing and transparency issues are implicated in these discussions as well, and were woven into all the panels over the two days of the conference.

Meeting objectives:

  1. To understand the current shifts in regulatory requirements of medical products, particularly drugs and devices, in the U.S. and Europe and in the global South, and the origins of deregulatory efforts.
  2. To understand how patients’ needs for access to new medical products across the globe can be reconciled with the need for evidence on their safety and efficacy.
  3. To understand the specific regulatory needs of drugs and other medical products for neglected diseases.
  4. To develop a policy and research agenda to promote a new pro-patient, pro-evidence, pro-public health regulatory framework for the regulation of medical products.