On August 10, 2018, Alnylam Pharmaceuticals announced
that the FDA had approved its patisiran (Onpattro) to treat polyneuropathy caused by hereditary transthyretin-mediated (hATTR) amyloidosis. The approval was a milestone for patients with hATTR amyloidosis, as it is the first and only treatment for the rare, progressive, and often fatal condition.
The disorder is caused by genetic mutations that cause deposits of transthyretin (TTR), a protein present in human serum, in body tissues. These deposits produce sensorimotor disturbances as well as autonomic, cardiovascular, gastrointestinal, renal, leptomeningeal, bowel, and bladder dysfunction. Patisiran, a lipid complex, is an RNA interference therapeutic delivered by intravenous infusion. The drug suppresses the production of both mutant and nonmutant forms of TTR and has been shown to both improve polyneuropathy and reverse neuropathy impairment in a majority of patients, leading to quality of life improvements.
The approval was notable not only for the promise that it offers patients, but also for the strategy by which Alnylam hopes to sell the drug: In order to help payers cope with the $450,000 annual list price of patisiran, Alnylam is working with commercial insurance plans to offer value-based contracts for the therapy.
The contracts will tie the level of reimbursement to patient outcomes; if patisiran delivers the same benefits in real-world practice that it did in clinical trials, Alnylam will receive a higher level of reimbursement. If the drug does not perform as well, it will receive lower reimbursement.
Alnylam says that it is currently discussing value-based contracts with multiple health plans and has reached an agreement in principle on a contract with Harvard Pilgrim Health Care. Michael Sherman, MD, MBA, chief medical officer of Harvard Pilgrim Health Care, said
in a statement that linking reimbursement with outcomes will help the plan meet its patients’ needs by balancing access with affordability.
“We believe these initiatives are the right things to do for patients and will deliver value to the healthcare system,” Barry Greene, president of Alnylam, stated. Greene added that his company is also launching a patient support program that will offer case managers to help coordinate insurance benefits and financial support for eligible patients, as well as liaisons who will offer patient education on hATTR amyloidosis.
Alnylam’s attempt to secure value-based contracts is in line with new approaches to paying for such high-cost drugs that have helped address costs in the short term, but challenges with such contracts will still need to be addressed in the longer term. The Alliance for Regenerative Medicine’s Foundation for Cell and Gene Medicine, an organization that focuses on issues that face regenerative and advanced therapies, recently released a report
, prepared by IQVIA, that finds that outcomes-based contracts for products like tisagenlecleucel (Kymriah) and voretigene neparvovec-rzyl (Luxturna) have helped to overcome payers’ skepticism about high-cost drugs.
However, it also found that a lack of infrastructure to track patients and link their clinical outcomes to insurance claims complicates value-based contracts. Furthermore, innovative payment models may reduce the immediate budgetary impact of these products, but they fail to improve long-term sustainability of the healthcare system. A new framework for paying for these therapies, the report suggested, will be key.