Author + information
- Spencer B. King III, MD, MACC, Editor-in-Chief, JACC: Cardiovascular Interventions∗ ()
- ↵∗Address correspondence to:
Spencer B. King III, MD, MACC, Saint Joseph's Heart and Vascular Institute, 5665 Peachtree Dunwoody Road NE, Atlanta, Georgia 30342
- David E. Kandzari, MD, Director, Interventional Cardiology and Chief Scientific Officer
It is the goal of all practicing physicians to provide our patients with optimal medical and surgical care for their condition. Part of providing this care is the ability to participate in clinical research. While participating in research is fundamentally positive for the physician, the hospital, and the patient, the requirements to successfully secure Medicare reimbursement for procedures used in medical device trials under current regulations and processes has become exceedingly difficult in some regions of the United States.
As background, on September 8, 1995, the U.S. Food and Drug Administration (FDA) entered into a memorandum of understanding with the administrator of the Medicare program, the Health Care Finance Administration (HCFA) (now identified as the Centers for Medicare and Medicaid Services [CMS]), to provide information about medical devices under an Investigational Device Exemption (IDE) to aid in the agency's reimbursement decisions regarding these new technologies. Under this agreement, certain devices could be viewed as “reasonable and necessary” by Medicare, and treatments could be covered by Medicare if all other applicable coverage requirements are met. The intent of this memorandum of understanding was to provide Medicare beneficiaries access to novel or next-generation medical devices, depending on the FDA classification provided to HCFA (CMS). Under federal regulation, Medicare is required to cover the use of devices that are “reasonable and necessary for the diagnosis and treatment of an injury or illness or to improve the functioning of a malformed body member.” To assist CMS with the determination of “reasonable and necessary,” the FDA created categories of devices depending on their novelty or similarity to existing technologies. The idea was that devices similar to existing devices or procedures should be covered while the product was still under investigation in an FDA-approved IDE study. The regulations also allow CMS to delegate responsibility for determining what is or is not “reasonable and necessary” to its regional Medicare Administrative Contractors (MACs) for individual trial coverage. Herein lays the opportunity for variable interpretation of the “reasonable and necessary” threshold for CMS coverage and in many instances, inability for Medicare beneficiaries to have access to new therapies that are available only through participation in clinical research studies.
The inconsistency with which the different MAC medical directors review and approve (or do not approve) coverage has caused widespread dissatisfaction among clinical trial sponsors, investigators, and clinical sites. The MAC medical directors make decisions independently and have no centralized reporting structure that ensures uniform policy making. The frequency in MAC changes across the regions, MAC staffing turnover (both medical directors and IDE coordinators), and the variable depth and quality of clinical protocol reviews has greatly hindered efficient and timely patient enrollment. There are numerous examples that have hindered conduct and access to clinical research. In one instance, a MAC (medical director and team) reviewed and approved a clinical site within 3 days while other sites in another MAC region waited more than 30 days for the same study to be reviewed and approved. Another medical director decided to approve only 10% of a site's potential population, considerably limiting enrollment. Still other regional medical directors have refused approval unless specific revisions were made to an already FDA-approved protocol—an impractical demand that is disruptive to the scientific validity of a study by imposing an inconsistent protocol implemented across trial sites. Finally, a medical director may refuse to review studies to which the FDA has granted approval “with conditions,” even when the FDA has agreed that there are no safety issues, instead insisting on “approval without conditions” from the FDA. Such approval may not be received until clinical studies are already underway or are completed. Despite earlier conditional approval, for example, final FDA approval of the first transcatheter aortic valve application occurred just 5 months prior to completion of patient enrollment. Among centers whose Medicare reimbursement was delayed, many otherwise eligible patients could not be enrolled in the study and thus could not receive treatment. Furthermore, the FDA itself has stated that approval “with conditions” is often sufficient to initiate trials, because outstanding issues do not pose safety concerns to patients that would preclude enrollment.
These types of inconsistencies have created an atmosphere of uncertainty among many physician investigators, hospital research programs, and medical device manufacturers that have adverse consequences on clinical research in the United States. To complicate matters further, many hospitals now refuse to issue an institutional review board (ethics review) approval until Medicare approval is granted. Likewise, some hospitals even refuse to screen or enroll commercially insured patients until Medicare approves the study, a decision predicated on concern regarding accusations of discrimination against Medicare patients. Based on these obstacles, one can see that the entire clinical trial process in the United States could grind to a halt, thwarting the development and evaluation of new potentially beneficial or life-saving medical device innovations, therefore driving these activities out of the United States. Attention is being focused on the longer time required to bring new medical device technology to patients in the United States compared with those in other parts of the world. Although much of the concern has been directed toward FDA review and approval, an under-appreciated obstacle is that even after the FDA has deemed that a clinical study of an investigational device can commence, this Medicare reimbursement process can delay or disrupt clinical research.
At present, no formal solution has been proposed to develop a more efficient and predictable process. One possibility would be designation of a central MAC to approve or deny coverage for all Medicare beneficiaries for all MACs. Enhanced communication with the FDA prior to the agency's approval of a protocol may also preempt misunderstandings related to protocol interpretation among MACs. Given that there is no formal method of determining variability and time delays in approval across MACs, such a policy would establish a record of accountability. Such measures would advance the consistency and reliability within the MAC review process. Participation in clinical research is a privilege for investigators, practitioners, and patients. A fundamental tenet of research should be equal and have timely access to such opportunities. While there is no single, rate-limiting step that delays the evaluation and availability of innovative medical treatments to patients in the United States, addressing the inconsistent Medicare reimbursement process will be an important step forward.
The authors acknowledge Geri Cramer and Mitchell Sugarman for their editorial review.
- American College of Cardiology Foundation