Conference urges support for innovations in science and medicine in a collaborative, safe and ethical manner
Posted May 14, 2018
Janet Marchibroda
Andrew von Eschenbach

https://www.rollcall.com/news/opinio...e-c4346bdc22f1

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 and ways to improve human 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.

Finally, finding new innovative ways to not just treat but also prevent disease must be part of the equation. To move the needle, we must explore other nonclinical determinants of health, including behavioral patterns, genetic predisposition, social circumstances and environmental exposure, which together affect approximately 90 percent of an individual’s health.

Pope Francis at the conference emphasized the need for global collaboration in health innovation 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.

We must take on that challenge together. The enormous progress in science and technology holds great promise for patients who suffer from diseases with no known cure. We must embrace and support these dynamic changes in a collaborative, safe and ethical manner, which will vastly improve human health in our society.

Janet Marchibroda is the director of the Health Innovation Initiative for the Bipartisan Policy Center.

Andrew von Eschenbach, MD, is a senior advisor for the Bipartisan Policy Center, a former commissioner of the Food and Drug Administration and a former director of the National Cancer Institute at the National Institutes of Health under President George W. Bush.

The Bipartisan Policy Center is a D.C.-based think tank that actively promotes bipartisanship. BPC works to address the key challenges facing the nation through policy solutions that are the product of informed deliberations by former elected and appointed officials, business and labor leaders, and academics and advocates from both ends of the political spectrum. BPC is currently focused on health, energy, national security, the economy, financial regulatory reform, housing, immigration, infrastructure, and governance. Follow BPC on Twitter or Facebook.