Finding #4: An integrated approach for rare disease treatment is key
According to survey respondents, the best long-term approaches to address the high cost of rare disease treatment are more integrated approaches by benefit managers (43%) and deploying more expansive strategies to access risk (21%). Lagging behind in the survey were using benefit incentives to drive consumer engagement and higher-value care (18%) and employing government guidelines or regulations (16%).
More drugs exist to treat orphan diseases than ever, Rademacher says. Agents that treat rare diseases have various routes of administration (e.g., self-injectable, oral, intravenous) and therefore are reimbursed by both medical and pharmacy benefits. “It is essential to integrate the pharmacy and medical benefit to more closely manage these costly diseases across benefits and reduce the total cost of care,” she says. “Management of rare disease drugs is common, but given the lack of competition in many of the rare disease categories, contracting strategies are not generally employed. Expanding the risk-sharing deployed in other specialty disease states by employing value or performance-based contracts could lower costs as seen in other disease states.”
With the most fragile patient populations, healthcare costs are significantly increased by issues with persistence and adherence due to the difficulty of the drug regimen and the other challenges a patient may face (e.g., scheduling, time, coordination of care), Wilkinson says.
“Stronger patient support regarding drugs and assistance with care coordination can improve outcomes,” she says. “Improved outcomes may make payers less likely to establish conditions of coverage that restrict access and lead to prescriptions that are written but never filled. This will enable manufacturers to consider basing pricing on effectiveness, because patient support will increase effectiveness and the market will become more stable.”