Rare diseases and orphan drug therapy: four things to know

If you haven’t heard much about orphan drugs before, that’s about to change.

One of the biggest trends in the development of new pharmaceuticals relates to orphan drugs, highly specialized medications used in the treatment of rare diseases. These medications often need to be combined with other drugs or therapies. As a result, they can come with a high price tag.

As a plan sponsor, you might wonder what orphan drugs mean for your drug plan. How likely is it that any of your members could be prescribed orphan drug therapy? What could the effects be? And what can be done to manage them?

Here are four things you should know about rare diseases and the orphan drug therapies being used to treat them.

1. Rare diseases aren’t as “rare” as you might think.

The word “rare” can be deceiving. One rare disease may affect just a sliver of the population, but there are many different types of them (approximately 7000). Here’s another way to think of it: rare diseases are rare, but there are many rare disease patients. The European Organization for Rare Diseases (EURORDIS) calls this the “paradox of rarity”1.

But how many Canadians are actually affected by rare diseases? According to the Canadian Organization for Rare Disorders (CORD), nearly three million Canadians fit the criteria – that’s 1 in 122.

Some examples of rare diseases include cystic fibrosis, which affects the respiratory and digestive systems; Huntington’s disease, which affects the brain and nervous system; and muscular dystrophies, which affect the muscles.

2. Orphan drugs are often part of complex treatment plans.

The potential cost impact of rare diseases is significant, not only because the drugs themselves are often expensive, but because they are recommended to be used as part of combination therapies with other expensive drugs.

For example, Cotellic (cobimetinib) a cancer drug for the treatment of metastatic melanoma (skin cancer that has spread to other places in the body) has recently been approved by Health Canada to be used in combination with Zelboraf (vemurafenib), another cancer drug3. Each of these drugs is expensive on its own, costing more than $100,000 annually. When both are used as part of a combination therapy, the total cost doubles to more than $220,000 per year.

Combination therapy is not a trend specific to cancer drugs, nor does it necessarily involve only two drugs. For example, in Europe, orphan drugs have recently been recommended as part of a triple therapy regimen for the rare disease pulmonary arterial hypertension4.

As treatment guidelines continue to evolve, we expect that the demand for combination therapies could grow in the near future.

3. The demand for orphan drugs is growing.

In order to understand drug spend trends and put in place the right programs to support your drug plans, Manulife closely monitors drugs that are currently under review with Health Canada. Based on recent results, we expect about 50 per cent of the drugs introduced to the Canadian marketplace within the next several years to be orphan drugs for the treatment of rare diseases. Among these new orphan drugs, about half will be used as part of cancer treatments.

As orphan drugs grow in number, their possible uses have been growing as well. Experience with these drugs has been steadily increasing and treatment guidelines have been evolving, leading to more frequent uses of combination therapy. Orphan drugs are also being introduced even earlier in treatment programs than before.

For the foreseeable future, orphan drugs like these can be expected to play a major role in the world of pharmacy benefits.

4. There are ways to manage the cost impact of orphan drugs.

As your drug benefits provider, Manulife is constantly looking for ways to help keep your drug plan sustainable.

The number of orphan drugs is growing, and with it, the need to closely monitor how they will affect the drug landscape. Manulife DrugWatch™ is a program that can help by examining certain new, high-cost drugs, considering both their effectiveness and their financial impact to ensure you are receiving the best value for your drug benefit dollars.

To manage the costs associated with complex drugs, we also offer Specialty Drug Care with Bayshore HealthCare Ltd. Through a preferred pharmacy network, preferred pricing and case management services, Specialty Drug Care can lead to better health outcomes for specialty drug patients, and cost savings for members and their plans.

To learn more about the Canadian pharmacy landscape, visit manulifebalance.ca