Payer Coverage for New Treatments

The ophthalmologist can play a role in introducing coverage policy.

STEPHEN A. KAMENETZKY, MD

The rapid development of new medications and technologies for the treatment of retinal diseases has presented ophthalmologists with a mixed blessing. The ability to preserve vision in situations previously thought hopeless has encouraged patients and physicians alike. However, trying to sort out the efficacy of the various new treatment modalities and obtain payment for them has proved in many instances to be frustrating. As a medical reviewer for a large national commercial payer, I have experienced the process from the payer's perspective and gained a better understanding of how decisions are made. I hope sharing this information will encourage other ophthalmologists to participate in the process and help speed access to new treatments for their patients.

FROM THE PAYER'S POINT OF VIEW

New technology and pharmaceuticals are often expensive and their benefits sometimes unclear or exaggerated. Although Food and Drug Administration (FDA) approval of a particular device or pharmaceutical is critical to their acceptance and indicates that the treatments have been carefully evaluated, it is insufficient on its own to allow automatic approval by all payers. It also does not facilitate approval of the off-label use of a drug.

In an era of rapidly escalating health spending, payers (who function in a zero-sum game environment) are understandably reluctant to spend scarce resources on treatments unless they are sure that the value obtained is worth the cost. Most have developed carefully controlled processes that are used to evaluate both technology and pharmaceuticals. Approval is rarely given unless there is evidence-based research that demonstrates patient outcomes better than (or at least equal to) those that currently approved technology and pharmaceuticals can deliver.

This is important because the cost of ineffective treatments is significant both in terms of dollars spent and opportunities lost. The potential of injury to patients from treatments that seem like a good idea (like bone marrow transplants for patients with metastatic breast cancer), but prove not to be, is significant. When payers initially refused to allow bone marrow transplants for patients because it had never really been shown to be effective, they were pilloried by the press, politicians, and patient advocacy groups and forced to reverse their decision. The academic community bypassed clinical trials because it was considered unethical to withhold this "life-saving" treatment. Unfortunately, when the trials were eventually performed, the evidence was clear: those who received bone marrow transplants died "quicker and sicker" than those treated with conventional methods — money wasted and opportunity lost.

"THE PLURAL OF ANECDOTES IS NOT EVIDENCE"

The decision-making process used by payers to determine whether to cover certain new drugs and technology has become quite rigorous. Information derived from randomized clinical trials forms the basis for the decision-making process. Medicare and most private payers have created teams of scientists, clinicians, statisticians, epidemiologists, and ethicists who are charged with dispassionately reviewing the available data and assessing both their statistical validity and clinical significance. Although the precise process varies from payer to payer, almost all decisions are based on a definition of "medically necessary."

Transparency is much improved over past decision-making processes, and cost is rarely if ever the primary driver for either commercial or government payers. Medicare and most commercial payers make their final policy available on their Web sites, complete with references and documentation of the process that led to the decision. My experience with the process is that it is clearly evidence driven, remarkably fair in approach, and not designed to limit treatment based solely on cost. However, this is not a conclusion that I would have reached before I became a part of the process.

BALANCING CONCERNS

As a member of the American Academy of Ophthalmology (AAO) Health Policy Committee (HPC), I often heard other members state that they had a good relationship with a local carrier medical director and that they would contact the individual to pass along some recommendations from the committee. I never gave it much thought until I took a part-time position as a medical reviewer for a large national commercial payer. Word spread quickly that I was the only ophthalmologist employed by the company and I began to get calls from medical directors all across the country asking for help in resolving issues involving coding, bundles, new technology, and assistants-at-surgery. I answered the questions as a fair broker, not a specialty advocate, using the guidance of the AAO Coding Modules (I am the chair), the HPC recommendations, and curbstone consults from my friends in various subspecialties. All my sources provided candid advice on best practices, allowing me, in turn, to give evidence-based advice that is fair to physicians, their patients, and the payer.

After a year or so, I was asked to help review and revise many of the company clinical guidelines for ophthalmology. I incorporated many of the recommendations of the HPC (acting as an honest broker) and put the company decision makers in direct contact with our Health Policy team in Washington, DC, if they had any questions. There is no question in my mind that the ability to assist a payer in this fashion has helped ophthalmologists expedite access to current best practices for our patients. You will see how as you consider the process in more detail.

WHAT IS MEDICALLY NECESSARY?

As noted earlier, new treatments are not generally approved for payment unless they are felt to be "medically necessary." Although the precise definition of this term may vary from payer to payer, in general they all include language similar to the following:

"Medically necessary" involves using procedures, treatments, and devices that a medical practitioner exercising prudent clinical judgment would provide for the purpose of preventing, evaluating, diagnosing, and/or treating an illness, injury, or disease or its symptoms that are:

► In accordance with generally accepted standards of medical practice

► Clinically appropriate in terms of type, frequency, extent, and duration

► Not primarily for the convenience of the patient, physician, or other provider

► No more costly than alternative services, which are at least as likely to produce equivalent therapeutic or diagnostic results.

Generally accepted standards of medical practice are usually further defined to include:

► Criteria based on credible scientific evidence published in peer-reviewed literature

► Supported by physician specialty and subspecialty society recommendations

► Supported by practitioners in the relevant medical community

THE OPHTHALMOLOGY ADVANTAGE

Although the standards used are not specialty-specific, ophthalmology nonetheless has several potential advantages that may help speed consideration of requests for approval of new drugs and technology. The first is that loss of vision is universally regarded as a devastating event that not only is a personal tragedy but also adds costs down the line as patients utilize both additional medical and other social resources. New treatments that hold the promise of preventing or delaying vision loss are therefore given very careful consideration, especially when currently approved treatments for the condition are either nonexistent or relatively ineffective.

Another important issue is that, unlike many medical conditions, a delay in obtaining treatment may have an especially detrimental effect in many eye diseases, especially those involving retinal pathology. Whereas the eventual clinical result may be the same if a patient with arthritis of the knee is required to try physical therapy, injections, multiple analgesics, and other "conservative" treatment modalities before total knee replacement is approved, the same thing is not true for a patient with "wet" age-related macular degeneration (AMD). With AMD, time to treatment is critical and inordinate delay can result in scarring and permanent destruction of tissue, precluding any improvement of visual function. This encourages payers to speed consideration of new treatment modalities.

Patients threatened with vision loss are also not inclined to be passive in making their treatment preferences known. They want immediate access to all of the new drugs and surgical techniques they have heard about on the news or from the medical specialists, at the beauty parlor, or on the Internet. Several years ago, these groups, assisted behind the scenes by industry, filed lawsuits against the Centers for Medicare and Medicaid Services (CMS) when many believed that access to a new treatment for AMD was being unfairly withheld. Their victory resulted in a change in CMS policy.

NATIONAL VS LOCAL DECISIONS

The "reach" of coverage decisions is important to the approach. Most payment decisions are actually made at the local level by individual carrier medical directors. For Medicare, often several states may be covered by these "local" carrier intermediary determinations. Sometimes, however, decisions are made nationally that set policy for every entity in the system. For Medicare, that national decision would bind all intermediaries in every state. For commercial carriers, it would cover all of the local entities operating under the national umbrella (eg, WellPoint or United HealthCare).

The question of whether to approach coverage issues on a national or local level is a complex one and there is really no right answer. The advantage of obtaining a national coverage decision from CMS or the large commercial payers is that it sets policy for all of the local entities that pay for services under their name. It is not necessary to contact and convince 50 or more carrier medical directors to grant approval. This universal setting of policy simplifies the effort and makes it less costly for those lobbying for a particular result.

Unfortunately, the disadvantage of pursuing a national approach is also that if coverage is not approved, the decision will likely be impossible to overturn at the local level. So what to do? It is a bit of a crap-shoot, but in general, if the evidence is very strong in favor of the proposed treatment and there is strong support from the national medical specialty societies, it is reasonable to try for a national coverage policy. Otherwise, local efforts might turn out to be more rewarding, at least in the short term, until more evidence can be developed to satisfy the more stringent requirements of the national decision makers.

OPHTHALMOLOGISTS AS ADVOCATES

With such a complex process required to obtain payment for new drugs and technology, you might wonder whether ophthalmologists, individually or collectively, can effectively influence the process. The answer, fortunately, is yes. The process by which the off-label use of bevacizumab (Avastin, Genentech) received national approval for treatment of neovascular AMD by CMS and other payers is a prime example of how the ophthalmic community can influence big payers. This case illustrates how important the input from ophthalmologists working together can be in achieving an outcome that allows patients to receive the treatment they need.

At the time payers were considering various proposals for using bevacizumab to treat wet AMD, photodynamic therapy (PDT) using verteporfin (Visudyne, QLT/Novartis) and intravitreal injections of the anti-vascular endothelial growth factor (VEGF) agent pegaptanib sodium (Macugen, OSI/Pfizer) were the only FDA-approved drug-related treatments for the disease. Both were expensive but represented an improvement over placebo in terms of stabilization of acuity. However, neither did much to improve vision. Ranibizumab injection (Lucentis, Genentech), another promising anti-VEGF agent, was still in trials and not yet available.

There was evidence to support the use of bevacizumab for treatment of wet AMD, although not in the form of the randomized clinical trial that was usually demanded for new therapy approval. However, it was being widely used by retina specialists to treat AMD. There was also good "list-serve" evidence to support both its safety and efficacy when injected intravitreally in humans. Results reported from the many small series available were very encouraging: Many patients showed an improvement of acuity with treatment, something not seen in patients treated with either PDT or pegaptanib sodium injection. A retinal specialist who was a member of the AAO Health Policy Committee told me that he could usually tell whether patients were receiving bevacizumab or the approved agent for treatment of AMD by simply looking at their faces (a smile was a good marker for bevacizumab) and checking the visual acuity. The AAO, in concert with the American Society of Retinal Surgeons (ASRS), collected the references and clinical data available and created a concise "white paper" supporting the use of bevacizumab if physicians felt it was in the patient"s best interest to do so.

In my capacity as the medical reviewer for my employer, the payer, I was available to answer technical questions about some of the material and also to offer support for the joint request of the AAO and ASRS that physicians be allowed to use their best clinical judgment to make treatment decisions. This was important because ophthalmology can be difficult for nonophthalmologists to understand. Although I had no direct role in the final policy decision, I was pleased that it was approved with language that allowed physicians to choose the treatment they felt would be most appropriate for the patient. This unusual decision to grant off-label approval to use a low dose of a chemotherapeutic agent for another purpose was helped by the fact that we made it clear that ophthalmology was not using the drug as a chemotherapeutic agent but rather for its anti-VEGF properties.

The subsequent FDA approval of ranibizumab for treatment of wet AMD complicated the process even further. Ranibizumab injection, very similar in chemical makeup to bevacizumab, had been proven effective in a well-designed randomized clinical trial. Bevacizumab was thought to be effective through many clinical observations on multiple patients, but the evidence was not as rigorous. Further complicating the process was the $2000 difference in price per treatment for a drug that requires multiple doses over a 2-year period. What should payers do? What should physicians do? Should the policy for bevacizumab be revised to reflect the fact that another agent was now available that had FDA approval? Once again, the AAO and ASRS's recommendation (which I passed along) was to keep the policies unchanged and continue to allow physicians to use their best judgment for each clinical situation. In the end, the policy makers, after carefully considering the various options, decided to take that route.

There are many remaining issues to be addressed going forward. These include approval of new anti-VEGF agents as they become available, combination of various approved agents to treat AMD and use of these and other agents for treatment of diabetic retinopathy and diabetic macular edema. How technology can be better used to affect clinical outcomes is a pressing issue as well. Reliable data to answer all of these questions need to be made available, and often, ophthalmologists are the only ones who can provide it.

WE CAN MAKE A DIFFERENCE

I hope after reading this article you will agree that ophthalmologists have an important role in helping to guide payers toward making coverage decisions that are fair and evidence-supported. On the national level, it is critical that the AAO and the subspecialty societies, such as the ASRS, continue to take the lead by providing assistance and information to the policy makers and make their voices heard when best practices are not followed. It is also important to publicly say "thank you" when they are.

Locally, try getting involved before you need a "favor" regarding a coverage issue. Introduce yourself to the medical directors of the major carriers in your area. Volunteer to help with coding and coverage issues, as well as grievances and appeals. Be a fair arbiter, remembering that if you are perceived as an advocate rather than an umpire, your advice is likely to go unheeded. If a rule is unfair, try to get it changed, but follow it in your decision making. No pouting allowed, no being a sore loser if the decision goes "the wrong way" — you will not win every argument, but you are likely to win your fair share once you gain the trust of those who depend on you to help them make the right decision. In doing so, you will help yourself and your profession — but even more importantly, your patients. RP