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Close-up doctor's hand giving pills in blister pack to patient; specialty pharmacy distribution concept

How Will Orphan Drugs Fare in an Era of Rising Costs and Wider Use?

Specialty pharmacy distribution is changing as specialty drug treatments become more commonplace. Many biosimilars have entered the rare disease market, directly competing with biologics like Humira that represented breakthroughs in their time. New treatments for a wide range of conditions are also available, including biologics, cell and gene therapies for rare diseases. 

Wider adoption of these treatments occurs alongside rising drug prices — and rising overall pharmaceutical costs. As legislatures and other parties seek to rein in costs, patients with rare diseases may face challenges in accessing the treatments they need. 

Specialty Pharmacy Trends: Rising Costs, Rising Adoption

Drug costs continue to rise, despite legislative attempts to address these costs in various ways. Approximately 50 percent of total pharmacy benefits plan costs in 2023, for example, are driven by just two percent of the plan’s prescription drug claims, writes clinical pharmacist Dr. Kelly Stuart for RxBenefit. These claims are for specialty medications, including gene and cell therapies, all of which carry significant price tags.

Overall costs are driven by both rising prices and rising utilization. A study of drug cost trends by Dr. Eric M. Tichy and fellow researchers found that overall U.S. pharmaceutical expenditures increased by 9.4 percent between 2021 and 2022. Utilization drove the bulk of this increase, accounting for 5.9 percent of the total. Higher prices accounted for 1.7 percent of increased expenditures, and new drugs accounted for 1.8 percent. 

As overall drug costs rise, the cost of orphan drugs continues to stand out. A 2023 study by Dr. Hana Althobaiti and fellow researchers discovered, for example, that the median annual cost was $218,872 for orphan drugs but $12,798 for non-orphan drugs. Althobaiti et al. further found that biologics, orphan drugs, and medications for chronic conditions, oncology, and genetic disorders were more likely to carry high price tags than other drugs. 

Rising drug costs for the rare disease market have been in the headlines for some time. Attempts to address the issue seek to balance costs involved in specialty pharmacy distribution, specifically access for patients. These attempts can have an outsized impact on orphan drugs, which often have outsized costs but relatively small patient populations.

Mother and daughter sit on chairs, waiting for the pharmacy counter to provide services to patients in medical center; specialty pharmacy distribution concept

The Changing Landscape of Rare Disease Treatments and the Role of Specialty Pharmacy

Legislative responses to rising drug prices include reviewing drug prices and, in some cases, reducing them. Yet these moves may spark specialty pharmacy trends such as a “ripple effect,” which sees some medications no longer being available in states that set the price too low. 

One example is Trikafta, a medication for cystic fibrosis, which is under price review in Colorado. The review is leading many to worry that Vertex Pharmaceuticals, Trikafta’s manufacturer, will simply decline to sell the drug in Colorado if it cannot command a price for the drug it deems adequate, writes Markian Hawryluk in KFF Health News. 

Setting price caps on expensive drugs is one way to address rising prices, but it can have undesired side effects. As noted, in some cases adjusting prices hinders patients from receiving treatments instead of facilitating access: Trikafta recipients risk their payers dropping the drug from coverage, forcing patients to seek coverage through the drug’s manufacturer, charity programs, or other sources.

Meanwhile, patients are changing the way they interact with the healthcare system. In one study, about 72 percent of adult U.S. respondents said they’d be open to having medications prescribed by a specially-trained pharmacist instead of a doctor. Younger respondents are more willing to embrace specialty pharmacies as sites of medical care. “Gen Z and Millennials are thinking differently about the who and where of healthcare and medication,” says Dr. Peter Bonis, adjunct professor of medicine at Tufts University School of Medicine .

To manage these complex changes to specialty pharmacy distribution for patients, everyone — providers, payers, pharmacies and distributors — must work together. Hub platform technology allows participants to communicate easily and effectively about a patient’s needs. Through the Hub, patients can connect in real time with their payer, provider, and specialty pharmacy — ensuring they receive the treatment they need when they need it. 

Medications like Trikafta can be life-changing for patients with rare diseases, but their hefty price tags can also make them a target for legislatures, payers, and other entities seeking to rein in healthcare costs. Efforts to address these rare disease market costs are likely to disrupt treatment access until a balance can be reached. 

Payers, providers, and pharmacies can help patients maintain access and possibly influence specialty pharmacy trends by using digital platforms to facilitate communication and keep patients connected to the treatments they need. 

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