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Asembia 2021: When the Disease Is Rare, Communication with Specialty Pharmacy Shouldn’t Be


Because families and patients may experience many difficult — and confusing — years prior to a definitive diagnosis of a rare disease, specialty pharmacies need to do everything in their power to make the treatment as navigable as possible, advised Nick Calla, J.D., and Dave Frobel of Orsini Specialty Pharmacy.

When it comes to gene and cell therapies for rare diseases, specialty pharmacies need to focus on every patient’s individual situation and prioritize communication with families and manufacturers, according to panelists speaking today at Asembia’s 2021 Specialty Pharmacy Summit in Las Vegas.

Nick Calla, J.D., senior vice president of industry relations at Orsini Specialty Pharmacy in Elk Grove Village, Illinois, and Dave Frobel, senior vice president of specialty pharmacy and trade at the company, outlined key considerations these pharmacies must take into account when working with patients with rare diseases, many of whom are children.

Nick Calla

Nick Calla

Approximately 7,000 rare diseases have been identified but only 10% have a known treatment, they explained. Rare diseases sometimes develop slowly, and years may past from when early symptoms occur and a clinician accurately diagnoses the condition, Calla and Frobel said. For many gene therapies, timeliness is crucial because usually the treatment is more effective earlier in the course of the disease.

Because families and patients may experience many years of doubt and worry prior to a definitive diagnosis, specialty pharmacies need to do everything in their power to make the treatment as navigable as possible, Calla and Frobel said. By employing an empathetic care team and striving to strike the right balance between over- and undercommunicating, specialty pharmacies can help parents deal with this complicated phase in their child’s disease.

Communication with manufacturers, reimbursement teams, and case managers can also help ease this process. “It's a different dynamic than what you'd see in a traditional pharmacy manufacturer relationship,” Calla noted.

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Frobel shared findings from a 2018 survey that showed that manufacturers want specialty pharmacies to provide additional services including communication, improved responsiveness or turnaround of therapies to providers, and more data in the rare and orphan disease area.

“Manufacturers are really looking to trend data to get insights from what's happening with their patients,” Frobel said. This can be achieved via platforms made to customize data collection for these specific therapies.

The survey also how manufacturers are largely dissatisfied with hub services, a finding Frobel said indicates that standard hub services models may not apply to rare disease and may need to be customized. Hubs are patient and administrative support services that centralize adherence, patient support and a variety of other programs.

Specialty pharmacies should also keep open lines of communication with patient advocacy groups and disease foundations to stay up to date on patient concerns, advised Calla and Frobel.

Both large and small manufacturers are working to develop cell and gene therapies for rare diseases. “You are looking at hundreds of millions of dollars in (research and development) to support very small patient populations,” observed Calla.

Because these therapies can cost millions of dollars, ensuring a process is in place for storage, transportation and administration is crucial, the experts said. Logistical and billing factors all need to be considered. Nurses may need to have experience with pediatric populations and could be tasked with assisting patients with correct administration or training them to administer the treatment themselves.

Maintaining consistent control room and storage temperatures is also crucial as

some of the therapies for rare disease may need to be frozen or stored at extremely low temperatures. Specialty pharmacies may need to consider disposal of dry ice or traceability of the product as it makes it way to administration centers.

Thousands of gene therapies are in development in both the oncology and rare disease fields, Calla said. “At the end of the day,” he added, “what we're really looking at is the patient. We want to give the patient hope.”

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