Coverage With Evidence Development Allows Early Adoption, Better Evaluation

Health plans and providers can provisionally adopt promising therapies before all the evidence is in hand

Mark Perman, MD

With increasing pressure to reduce health care costs, physicians and payers are finding themselves in the midst of a debate over access to the latest, most advanced treatments, tests, and other health care services. While patients and physicians are eager to embrace these innovative treatments, coverage by insurers often lags far behind the pace of innovation. Creative solutions are needed to resolve this catch-22 of no coverage without data, no data without treatment, and no treatment without coverage,

Recently, the Centers for Medicare & Medicaid Services (CMS) signaled that it may have found a model that private payers could adopt. By re-examining its Coverage with Evidence Development (CED) policy, CMS may broaden access to innovative therapies by establishing a transparent and faster pathway to coverage.

CED provides payment for new and/or innovative treatments while simultaneously generating clinical data to demonstrate the treatment’s effect on health outcomes, including comparative effectiveness. The goal of the program is to support innovation and the timely collection of data while helping payers make evidence-based decisions that improve health outcomes for their beneficiaries.

An improved CED process could not only help Medicare figure out which patient populations would be best suited for particular therapies; it could also have implications for commercial payers who could adopt a similar approach for their beneficiaries.

Why CED is changing

CED was developed in 2000 for coverage policies that focused on certain items and services that CMS believed were promising but lacked sufficient data to support coverage or for instances where CMS had additional questions that weren’t answered by the available evidence. Recognizing its value, CMS is now planning CED’s next phase, which will hopefully be better defined so that it can promote innovation and comprehensive patient care.

In May 2012, a meeting was held at the CMS headquarters in Baltimore to examine more ways to use CED. The discussion was lively, with both CMS and commercial payers expressing a desire to explore ways in which data could be collected while at the same time ensuring that the treatments they pay for are beneficial to patients.

Payment with accountability

With health care costs continually on the rise, there is increased consideration of whether many treatments and tests are warranted. CED can provide access to treatment while allowing the payer to monitor associated costs and clinical outcomes. One way is through patient registries, which support data collection on new treatments to evaluate them against existing ones. This allows providers to benchmark how well their patients are doing compared to others, helping to identify better dosing schemes, patient management issues, and other factors that may support better overall outcomes.

Unlike most clinical trials, which are generally limited to academic centers, registries are more accessible to the greater patient population, as they can be implemented by community-based physicians. Registries can help answer questions that are unanswered by narrowly designed clinical trials. They can also be kept open for many years, allowing physicians, researchers, payers, and patients to continually evaluate treatments as the science evolves in a particular field of study (See “Registry Tracks SBRT for Prostate Cancer” below).

Registry tracks SBRT for prostate cancer

Today’s treatment options for prostate cancer are diverse. With such a large portion of the population affected by prostate cancer, payers are eager to determine which therapies work well and which ones may not.

With the age demographic of prostate cancer generally being men of Medicare age, combined with the pressures CMS faces to control costs, it comes as no surprise that CMS held a meeting two years ago on radiation therapies for localized prostate cancer. With no comparative randomized study results available to demonstrate which option is most effective, patients and physicians were then, and remain today, left to choose from a long list of options, each of which present different risks and benefits. In the absence of definitive head-to-head data, several of the 2010 meeting participants and panelists proposed registries as a way to try to answer some of the questions on comparative effectiveness.

Shortly after the 2010 meeting, the first CED prostate registry was developed to help fill in some of the evidence gaps in the area of radiation therapy. In June 2010, the Registry for Prostate Cancer Radiosurgery (RCPR), a Florida-based multi-institutional, internal review board-approved observational trial (ClinicalTrials.gov NCT 01226004), began enrolling prostate cancer patients nationwide. Patients received an advanced treatment called stereotactic body radiation therapy (SBRT), a noninvasive approach to treating prostate cancer that offers patients a combination of benefits that other treatment options, such as conventional radiation therapy and surgery, do not. In the two years since the registry was introduced it has been widely adopted and now has the participation of over 40 hospitals and centers across the United States and has enrolled over 1,000 men. Outcomes data for the RPCR registry were presented at the May 2012 Medicare Evidence Development & Coverage Advisory Committee meeting convened by CMS.

As the first registry of its kind, the RPCR registry serves as a successful example of how a revised CED policy would support medical innovation while benefiting payers, patients, and providers. Through this cost effective approach, evidence is collected through a safe and effective mechanism which promotes access to care. As a complement to randomized clinical trials, registries are a great opportunity for facilities not affiliated with academic institutions to make their real-world experiences count.

With CED, safety is the top priority and for it to be successful, there must be strong collaboration between payers and providers where realistic expectations are set for evidence development and transparency.

Through the CED process, payers and providers could mutually agree on a timetable to check the data for adverse events along the way. From the beginning, the registry would be used to help the payer ensure that each service provides outcomes meeting the standard of care, if not better. After a reasonable period, the payer could make a policy determination as long as the data met the agreed criteria.

Promoting innovation

All too often, the unintended consequences of policies designed to benefit a population end up stifling innovation, hurting the economy, and limiting patients’ access to lifesaving treatments.

To understand this, it’s helpful to look at how innovative services and treatments are developed. After funding years of research, product development, and fulfilling regulatory requirements, companies that manufacture drugs and devices find themselves in a race against the clock to get their products to market and generate revenue before their patents run out. The problem with the current payment system is that by the time a multiyear process of data collection ends and the technologies deemed sufficient for widespread payer coverage, they often run the risk of becoming outdated. The result is a loss of innovation and reduction in competition.

Working hand in hand

Clinical trials and CED are, in fact, complementary. While randomized clinical trials are the gold standard, they are also costly, time-consuming, and often fail to recruit enough patients willing to be randomized against a treatment they view as outdated. If properly implemented, CED can complement clinical trials by collecting “real world” data that can be generalized to the broader patient population. Another key benefit to the CED process is that competing technologies can be compared side by side, which empowers the private payer to revise policies as the evidence develops.

Can CED be applied within the private payer community?

Payers at the May 2012 CMS meeting discussed how CED could be put into practice for the private payer community. Most private insurers voiced support for medical innovation, as it builds both the art and science of medicine. The payers’ concern is to ensure that access to new technologies is provided responsibly. It is critical to have programs that effectively manage the introduction and spread of new technologies and services. Programs like CED can work for payers when they are well-planned and include multiple components and tools.

The author is medical director of South Florida Radiation Oncology.