There are over 7,000 medical conditions classified as rare diseases, according to the CDC. And while each condition is, in and of itself, rare, only affecting fewer than 200,000 people by definition, when you put them all together, rare disorders affect more than 350 million people worldwide.
Historically, orphan drug development has not been a top priority for the pharmaceutical industry. After important legislation like the Orphan Drug Act, the Pediatric Voucher Program, and the Orphan Drug Modernization Plan were enacted, incentives were put in place to help encourage the development of new potential treatments. And, when you look at the 2019 pipeline, some of those incentives appear to be finally bearing some fruit.
Related article: How Immuno-Oncology Biomarkers Are Changing Cancer Care
Gordon Vanscoy, PharmD, CACP, MBA, Chairman and CEO of PANTHERx Specialty Pharmacy, said it looks that there will be several breakthroughs that will help provide treatments for rare disease conditions that previously had none. And at the 2019 Asembia Specialty Pharmacy Summit, held in Las Vegas, Nevada, in a session entitled, “DELIVERxING Hope to Patients with Rare Diseases: Improving Orphan/Precision Medicine Channels,” Vanscoy discussed the role that specialty pharmacy can play in facilitating contemporary rare disease treatment and channel strategy approaches in the future.
Vanscoy argued that pharmacists are in a unique position to help rare disease patients connect with lifesaving treatments. He introduced his talk showing an x-ray from a pediatric rare disease patient, Edie. And he said patients like Edie often have to travel quite far to receive the care they need—if a therapy is even available.
“These patients go to Centers of Excellence all over the country,” he said. “They may receive healthcare in Chicago but live in North Dakota. That leaves them without their specialist so specialty pharmacy can play an incredible role, particularly in this model of providing a care bridge to these patients and ensuring they stay healthy and continue on with their therapies.”
He noted that medical advancements in precision medicine require careful management due to the costs to payers, patients, and society. The smaller the patient population, he said, the better off the industry is with a semi-exclusive or exclusive channel, allowing for better data management and better flexibility and rapidity in identifying trends in those patients.
“These are the ideal characteristics for exclusive: you have smaller populations, products that require high touch, white-glove service, increased real-time access to data, and greater control over distribution,” said Vanscoy. These are also the characteristics that require an ideal specialty pharmacy with a good reputation and high patient satisfaction that has a customizable software platform.
“You’ve got to make sure the basic block is happening—licensure and payer coverage,” he said. “You’ve got operational redundancy and accreditation. Our accredited bodies are now looking at accreditation distinction in rare disease—I predict that will be available in the next 12 months.”
Related article: Understanding Both Sides of the Copay Accumulator Debate
For patients like Edie, who Vanscoy said is thriving on an orphan drug medication, he said it is imperative that specialty pharmacists help to get these rare treatments out to the patients who need them the most. And they are ideally suited to do just that.
“This is why we do what we do,” he said. “We do it because we have the opportunity to deliver hope—and we do it on a daily basis.”
Kayt Sukel is a science and health writer based outside Houston.