May 17, 2019
During a session at the 2019 Asembia Specialty Pharmacy Summit, Sami Khella, MD, professor and chief of Clinical Neurology at the University of Pennsylvania, and Karen Thomas PharmD, PhD, pharmacy supervisor with the University of Utah Health Specialty Pharmacy Services, discussed data that supports the use of current and emerging therapies for hereditary ATTR (hATTR) amyloidosis treatment as well as ways payers can effectively manage rare diseases.
Dr Khella kicked off the session by defining the pathophysiology of hATTR, a multi-system disease. He explained a multi-system disease affects many organ systems, including peripheral nerves, sensory neuropathy, amyloid motor neuropathy, and amyloids.
Dr Khella broke down the meaning of amyloid. He explained that there are two categories that relate to amyloidosis—hereditary (variant), and acquired. He said that after a diagnosis is made, which can be challenging, he highlighted that there are 2 recently approved medications that are available for the treatment of this multi-system disease. The treatments include:
- Patisiran (Onpattro), which was approved August 10, 2018; and,
- Inotersen (Tegsedi), which was approved October 5, 2018.
He then highlighted the available oral therapies that are available, which include:
- Diflunisal (Dolobid)—Peripheral Neuropathy;
- Doxycycline-Tauroursodeoxycholic Acid (TUDCA)—Cardiomyopathy; and,
- Tafamidis (Vyndaqel)—Peripheral Neuropathy (Europe) and Cardiomyopathy (US).
Dr noted that clinical trials for ATTR Fibril Disputer include Doxycycline and tauroursodeoxycholic Acid (TUDCA) for the reduction of nonfibrillar TTR deposition.
In the second part of the session, Dr Thomas highlighted considerations for both payers and specialty pharmacy.
According to Dr Thomas, the global prevalence of hATTR is approximately 50,000 patients. She explained that hATTR is heterogeneous, debilitating, and progressive. She continued and said that there is a significant demand—physically, mentally, emotionally, and financially—on both patients and caregivers. Dr Thomas highlighted that patients often report high health care utilization and impaired quality of life, and caregivers report poor mental health and reduced work productivity.
Although limited data is available about the cost of managing hATTR amyloidosis, Dr Thomas provided estimates. She said that the estimated cost of a liver transplant in the United States is $1,427,805 per patient. Further medication costs for blocking synthesis of TTR and TTR tetramer stabilization include:
- Blocking synthesis of TTR
- Patisiran, inotersen—estimated maximum of $450,000 per year
- TTR tetramer stabilization
- Diflunisal—estimated $1200 per year
- Tafamidis (not available in the US)—estimated $170,000 per year, which is roughly $14,175 per month.
Dr Thomas then explained medication access considerations, direct and indirect costs, managed care strategies, and challenges and successes related to rare diseases. She explained that common payer requirements for biologics and injectable medications often require things like step therapy, prior authorizations, specialist referrals, and shifts between medical and pharmacy benefits. She noted that there is a need for patient payment assistance because the cost of care is often high for rare diseases. She said that there are often more apparent costs, such as medical equipment, insurance premiums, hospitalizations, and medication copays. Additionally, Dr Thomas noted that there are also hidden, or indirect monetary costs, often associated with rare diseases.
Dr Thomas highlighted managed care strategies for patients with rare diseases. She said it is important to identify patients who will have high utilization and spending early one. She encouraged the use of specialty pharmacy and care management teams as well as care coordination. She also said that it is important for managed care professionals to work with patients to identify feasible goals of treatment and to consider adjusting mediation tiers, reducing coinsurance, and evaluating out-of-pocket policies that may create insurmountable barriers.
The last topic Dr Thomas discussed was the role of the pharmacist in rare disease treatment. She explained that the pharmacist must provide excellent care, including empathy and compassion, participate in care coordination and collaboration, assist with comorbid condition management, ensure continuity in prescription drug coverage and prior authorization approvals, and assist in patient counseling.