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New medicine demands review of ethics

The power of medical research is rapidly moving from the lab to the patient.

Since the 21st Century Cures Act was passed in 2016, we’ve seen exponential progress in personalized, data-driven medicine and regenerative and gene therapies that will help prevent and treat disease, and even cure patients. Swift advances in science hold great promise for patients in need. At the same time, we must maintain our national standards for safety and ethical responsibility.

Last month, the two of us had the privilege to participate in the Fourth International Vatican Conference, “Unite to Cure — How Science, Technology, and 21st Century Medicine Will Impact Culture and Society,” which was hosted by the Pontifical Council for Culture and the Cura Foundation. The purpose of the conference was to discuss the latest scientific innovations in regenerative medicine to improve health and prevent disease.

The three-day event engaged some of the world’s top scientists, ethicists and physicians; business and religious leaders; patients and families; and current and former policymakers. It culminated with a private audience with His Holiness Pope Francis, who acknowledged the great strides made by scientific research in the area of regenerative medicine and spoke about the importance of collaboration and ethical responsibility in science.

New innovations in cell therapies show great promise for helping patients with rare, autoimmune and neurodegenerative diseases, as well as cancer, diabetes and cardiovascular disease. Thanks to the Cures law, the National Institutes of Health and the Food and Drug Administration now have the tools to accelerate the discovery, development and delivery of safe and effective treatments for patients.

The law provides funding for NIH research on regenerative medicine and authorizes the FDA to expedite its review and approval of these therapies. The results are exciting — to date, the FDA has awarded 16 products a Regenerative Medicine Advanced Therapy designation.

Given the pace of these new therapy developments, we believe the United States must now take steps to evaluate the progress and ensure safety and ethical standards are upheld.

First, Congress and the administration should allocate $3 million in fiscal 2019 appropriations to create a flexible, collaborative 21st-century registry to acquire real-time knowledge and data to understand the effects of these new treatments on patients.

For real-world evidence, the registry should systematically track patients’ conditions and experiences, health outcomes, safety, and efficacy. This will help advance research and development, improve the delivery of treatments, support regulatory and reimbursement processes, and inform clinical and patient decision-making. The registry should also incorporate clinical data from electronic health records now adopted by more than 90 percent of the nation’s hospitals and physician offices, along with patient-generated and claims data.

Registries are common across a host of medical specialties including cardiology, ophthalmology and thoracic surgery. A registry for patients who have received bone marrow and cord blood transplant therapies already exists, and is part of the C.W. Bill Young Cell Transplantation Program administered by the Health Resources and Services Administration and authorized under the Stem Cell Therapeutic and Research Act.

Secondly, the emergence of gene editing and gene therapies as new ways to cure disease has amplified the need for responsible science. Today, scientists are modifying genes to significantly extend human life — a topic that sparks considerable debate on the ethical and societal consequences. We need open dialogue among the scientific, patient, clinical, bioethics and religious communities to ensure that such research is conducted in responsible ways.

Finding new ways to treat andprevent disease must be part of the equation. We must explore other nonclinical determinants of health, including behavioral patterns, genetic predisposition, social circumstances and environmental exposure. Pope Francis at the conference emphasized the need for global collaboration to discover and make available new cures and treatments, as well as the importance of preventing disease through education, physical activity and diet, especially for marginalized populations.

Janet Marchibroda is the director of the Health Innovation Initiative for the Bipartisan Policy Center. Andrew von Eschenbach M.D. is a senior adviser for the Bipartisan Policy Center and a former commissioner of the Food and Drug Administration.

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