Even though they only represent a little more than two percent of prescription volume in the U.S., specialty medicines have become the most significant and fastest-growing segment of the U.S. pharmacy market.

The specialty drug market has grown 1,200 percent since the mid-90s. In 2019, 36 percent of the U.S. pharmacy industry’s revenue (totaling around $161 billion) came from specialty drugs.

For all its growth, though, the specialty medicine market has been somewhat stagnant in what it was used for. Until recently, specialty medicines were reserved for rare diseases that did not have other treatment options.

Today, however, thanks to advancements in technology and innovation, more pharmaceutical companies can use specialty medicines to target beyond rare diseases.

Specialty Drugs and Rare Diseases

A disease needs to affect less than 200,000 people to qualify as rare. And so, the need for specialty drugs to treat rare diseases is acutely felt among populations with rare diseases like Multiple Sclerosis (MS), HIV, and Gaucher’s Disease.

Altogether, 25 to 30 million people in the U.S. suffer from a rare disease. In comparison, around 2 million people in the U.S. receive a cancer diagnosis annually.

Because so few people are diagnosed with rare diseases each year, patients have limited options for treatment outside of specialty drugs.

The process of researching, developing, testing, and launching these drugs on the market has historically been very costly. However, the increasing use of digital tools in pharmaceutical manufacturing, such as artificial intelligence (AI) and machine learning (ML), will decrease costs and shorten the time it takes to get a drug developed, tested, and on the market.

This means that there will be new opportunities to create specialty drugs for illnesses and diseases that are not considered rare. For example, experts anticipate that by 2023, 65 percent of new medicines will be for specialty therapies that indicate beyond rare diseases to include cancer and other illnesses.

What Separates Specialty Medicines from Everything Else?

Specialty medicines have several features that distinguish them from other types of treatment. For one thing, they are often more scientifically complex than other types of drugs because they go beyond simply managing symptoms to treating a disease’s underlying cause.

In addition, specialty medicines have an option to get priority status from the Food and Drug Administration (FDA), which accelerates the review process and allows them to get to market faster. However, because specialty medicines are designed to treat diseases with a small patient population, it can take longer to go through clinical trials.

Finally, many specialty medications can’t be developed in pill form. Instead, patients may need to get their treatment through infusion, injection, or nebulization. This can increase the cost of specialty therapies. For example, in 2017, the average annual cost of specialty medication was over $78,000.

Takeaway

While there are many opportunities for pharmaceutical companies to branch away from rare diseases when creating specialty medication, there is also a need for manufacturers to consider budgetary constraints.

Keeping communication lines open between all stakeholders, including payers, health systems, governments, and pharma manufacturers, will prove critical as we move into this new era of pharmaceutical possibilities.