Regulators and payers may have different priorities in the development of cardiovascular therapeutics, leading to obstacles and delays in patient access to new treatments, according to an article published Nov. 27 in the Journal of the American College of Cardiology.

Faiez Zannad, MD, PhD, et al., describe interactions among regulatory agencies, payers, sponsors and investigators, and propose collaboration among all stakeholders earlier in the development process. They highlight the current complexities in both the U.S. and European health care systems, explaining how in the U.S., there is a substantial difference in reimbursement practices among various payers, and reimbursement decision-making is not transparent. In Europe, the process for reimbursement decisions is based on the predominance of single payer systems and the ability of national organizations to negotiate pricing. Both, however, are actively working to advance the steps toward early alignment.

“Earlier and predictable patient access to effective new cardiovascular therapies is the primary goal underpinning proposals to collaboratively engage regulatory and payer representatives in pre-approval drug development decisions,” the authors write. They also suggest that transparency and accountability can be shown by defining requirements for reimbursement before regulatory approval. They explain that implementing strong scientific standards for cost effectiveness or other analyses used in reimbursement decision-making can help achieve greater transparency and acceptance of reimbursement decisions.

While consistency and quality of payer interactions are questionable, steps are still being taken to promote earlier interaction among regulators and payers in North America, Europe and other parts of the world. For example, although it is limited, the U.S. allows both the Federal Drug Administration and Centers for Medicare and Medicaid Services to concurrently review data about medical devices to incorporate both perspectives into a single clinical trial. Europe has also taken steps to advance progress toward early interaction by engaging a variety of tools to handle uncertainty from lack of information about budget impact, cost effectiveness, use in real life and access.

Additionally, the authors explain that a great resource allocation is needed for updating data on efficacy and safety, and revoking either regulatory or reimbursement approvals should not be underestimated.

“There is no easy solution, but collaborative engagement of regulators, payers, health technology assessment bodies, pharmaceutical companies, academic leaders, investigators, patients, physicians, professional organizations, and other health care providers represents an important step to developing a system that reflects the views and needs of all stakeholders,” the authors write. 

Keywords: United States, Technology Assessment, Biomedical, Single-Payer System, Uncertainty, Cost-Benefit Analysis, Research Personnel, Centers for Medicare and Medicaid Services, U.S., Medicare, Medicaid, Decision Making, Budgets, Social Responsibility, Resource Allocation, Health Personnel, Pharmaceutical Preparations, Europe, North America

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