For patients struggling with rare disease, finding care and navigating the disease is not often easy. Complexities abound, from potentially long time periods between initial symptoms and diagnosis to locating doctors knowledgeable in the disease and starting on therapy (assuming a drug for the condition even exists). Part of this journey has eased somewhat with the explosion of rare disease drugs that have hit the market in recent years. Between 2013 and 2017, the U.S. Food and Drug Administration approved nearly 250 drugs under the orphan drug program that provides incentives to companies investigating drugs for rare diseases. The growing number of treatable rare diseases is a welcomed trend.
Approval of drugs is one thing; accessibility is quite another. Rare disease therapies can be fraught with challenges for patients trying to access them. One of the primary issues is cost. Partly due to needing to recoup research investment in a small group of patients and also due to limited ability of insurers and the U.S. government to dictate price, the cost of rare disease drugs is often very high. Rare disease drugs customarily launch with prices well over $100,000 per year, and some of the new cell-based and gene therapies have price tags in excess of half a million dollars. Availability can be complex, as well. Many rare disease drugs may be accessible at only certain pharmacies or hospitals, which can be hard for some patients, especially in rural areas. Whether or not these specific pharmacies and hospitals are in the insurer’s provider network adds another layer in an already bewildering situation for the average patient. Unfortunately, the environment is not going to get easier.
Insurers and payers, including state and federal governments, have found striking the right balance between accessibility of rare disease drugs and controlling cost to be challenging. To keep benefits affordable, insurers and payers have responded with measures to manage rare disease drugs. The trend in health care of high deductibles and coinsurance means patients on rare disease drugs can face thousands of dollars in bills for each month of treatment until the out-of-pocket maximum is met. Prior authorization programs, designed to determine if a patient is appropriate for therapy, can be confusing and take time to process. There may also be restrictions on where the patient can get the drug.
One insurer program from where rare disease drugs have traditionally been safe is exclusion from coverage. Pharmacy benefit managers, or PBMs, began introducing exclusion lists in 2012. Put simply, an exclusion list is a list of drugs that are not covered at all. A list of covered, or preferred, medications is also shown so the doctor and patient can see the covered alternatives. Excluded drugs were generally in competitive categories where many drugs are very similar, and thus only the least expensive ones would be covered. Drug categories where some therapies are commonly excluded include diabetes, inhalers for chronic obstructive pulmonary disease and pain medications — but rarely rare disease. However, times change. In August 2018, PBM Express Scripts announced their drug exclusion list for 2019, and for the first time, included the rare disease categories of hemophilia, human immunodeficiency virus and hereditary angioedema. Express Scripts may have felt these diseases now had enough similar drugs to warrant excluding the more expensive ones. Other PBMs could follow suit in the future in these and/or other rare disease drug categories.
For patients with rare disease, the addition of these drug classes to exclusion lists adds another layer of complexity. Express Scripts has said that patients already treated with an excluded drug for certain rare diseases will be able to continue. For those patients who will be starting therapy, however, the exclusion means the provider and patient may have fewer options to choose from. Doctors not aware of the exclusions may accidentally prescribe an excluded drug that will be rejected for coverage. The rejection will add additional time before the patient can access therapy. In addition, the exclusion list adds to, rather than replaces, the other measures in place, including prior authorization, mandatory pharmacy or hospital points of access, and high patient cost.
Rare disease patients and their caregivers tend to be an engaged and knowledgeable group, which is useful in a difficult health care environment. Patients can take a number of steps to help them navigate exclusions. For those patients on an excluded drug, be sure to work with your doctor to file for an exception with your plan as soon as possible. Doing so can reduce the risk of therapy interruption. For patients new to treatment, look into what your health plan covers. If your health plan has exclusions, share this information with your doctor. If your doctor plans on prescribing an excluded drug anyway, at least he or she will know ahead of time and can get paperwork started. However, if the doctor feels one of the preferred drugs would work just as well, the choice could mean easier access and potentially less cost for the patient. Finally, patients should look into copay and coverage assistance programs offered by pharmaceutical manufacturers and foundations. These programs may provide opportunities for patients to access drugs when the health plan doesn’t cover it.
Drug affordability and health care costs continue to be a topic of intense discussion on both the federal and state level. Unfortunately, access to costly therapies is likely only to get more difficult. Rare disease patients will need to rely on what they have in the past: education and engagement. Taking charge of one’s health care means all aspects from treatment, from making doctor’s appointments to knowing what drug is covered. Exclusions make the treatment journey more complex but, for the empowered patient, far from impossible.
Jeremy Schafer, PharmD, MBA, is SVP at Precision for Value.
More from U.S. News
Exclusions: Adding Complexity for Patients With Rare Diseases originally appeared on usnews.com