From Proposal to Patient: How CMS Makes Coverage Decisions

   Some problems are so complex that you have to be highly intelligent and well informed just to be undecided about them. - Laurence J. Peter

   A product takes a long, expensive, and rigorous journey to go from a manufacturer’s think tank to the hands of the end-user. Vague ideas and concepts are molded and moved into the research and development pipeline where, over time, a product is perfected. Once the product has been refined and tested, it is strategically marketed so that it will find its way to the targeted end-user.

   Because pharmaceuticals and medical devices, such as ostomy and wound care supplies, are regulated by different Centers within the Federal Drug Administration (FDA), their paths to the marketplace and the end-user are quite different. Medical devices are less stringently regulated than drugs. Unlike pharmaceuticals, medical devices do not always require proof of safety and effectiveness before they can be marketed – in fact, a number of these products are marketed without any prior filing with the FDA whatsoever. Some manufacturers do conduct time-consuming and costly clinical trials – a process that slows down their ability to move products quickly through their pipeline. Ultimately, however, the investment of time and money is worthwhile as this clinical data is used to obtain payor coverage policies and made available to clinicians and consumers at product launch to support product claims.

   Clinical trials also play a pivotal role in obtaining favorable coverage and payment decisions from insurers, particularly Medicare – the benchmark payer. Many people operate under the mistaken assumption that FDA approval automatically guarantees Medicare coverage. It does not. Once a manufacturer’s new product has been cleared for marketing, the next step is to obtain reimbursement for it from insurers. The Centers for Medicare and Medicaid Services (CMS) evaluate relevant clinical trials to determine whether or not the data is of sufficient quantity and quality to support a finding of clinical effectiveness – the tenet upon which national Medicare coverage and payment policies are based.

   Manufacturers who have not conducted their own clinical trials must rely solely on published evidence in the medical literature (eg, a number of peer-reviewed publications). This evidence will support clinical effectiveness of the benefit they claim their product provides – for example, to the wound type(s) for which coverage is sought. The CMS stated that it has been their “experience that many studies performed to evaluate wound treatments are of poor quality.”¹

   In addition to several generally accepted methodological principles for developing protocol for and conducting a clinical trial, the CMS expects clinical trials for wound care products to include (among other information) the following:
   1. Is the outcome selected for measurement appropriate? For example, the optimal outcome to measure might be the number and proportions of wounds that reach complete closure. Assessing partial healing provides less assurance of clinical effectiveness, because the clinical benefit of partial healing has not been demonstrated in the literature.
   2. Have appropriate measures of endpoints been selected, identified before initiating the trial, and standardized across all study sites? Have clear measurement criteria been provided? Has the process used to measure the selected outcomes and methods in which the study investigators ensured the consistency of this process across different study sites been described?
   3. Was the appropriate patient population studied? For example, was the study performed on patients with the wound type for which coverage is sought?
   4. Has a single reference wound been selected for each patient? Generally, including multiple wounds on a single patient in the analysis provides limited additional data of value because individual wounds are not independent.
   5. Have all subjects, regardless of the protocol arm to which they are assigned (eg, investigational treatment or control) received good standard care and are standard care procedures identical for both arms? Have the standard care procedures been described in the protocol in detail?
   6. Have variables that may affect results been addressed in the analysis, including surface area, depth, and chronicity of wounds, condition of the subject, age of the subject?
   7. Has the effect on the wound of the therapy under investigation been evaluated? Adverse effects on healing can manifest in several ways, including tissue necrosis requiring more debridement, erythema, and drainage.
   8. Have adequate follow-up evaluations been performed? Clinical benefits from wound therapies can be short-lived and, therefore, of limited clinical value.

   Clinical trials published in the literature and conducted by manufacturers with methodologic shortcomings make it difficult for the CMS to draw positive conclusions about a new product. A positive national coverage determination is not supported if the studies conducted by the manufacturer or in the literature contain very small sample sizes with multiple serious study design flaws that make the validity of their results questionable. The CMS also may conclude that the medical literature does not support the conclusion that the new product heals a particular wound type better than a conventional or existing treatment.

   Many innovative medical technologies, including wound care products, have encountered extensive coding, coverage, and payment delays at the CMS. The result is that Medicare patients experience lengthy delays or may even be denied access to new products due to noncoverage policies or low reimbursement levels. Pointing a finger at and blaming the “system” for these delays is easy. However, the ostomy, wound, and continence community has a responsibility to its patients to improve the caliber and quantity of published clinical data.

    Every day, thousands and thousands endure the most terrible sufferings, though medical science could avert them. Will each of my readers think what the last 10 years of his family history would have been if they had been passed without medical or surgical help of any sort? It is time that we should wake from slumber and face our responsibilities. - Albert Schweitzer, 1948

1. Center for Medicare and Medicaid Services. General Methodological Principles of Clinical Study Design. www.hcfa.gov/coverage/8b3-hhh2.htm Cited: February 1, 2002