Improving access and reimbursement for specialty therapies

As specialty therapies move toward long-term maintenance use and as treatment times grow longer, government and commercial payers are scrutinising the economic value of new treatments more closely. In addition to efficacy and safety, clinical trials increasingly must demonstrate a meaningful impact on patients’ lives. Several executives at ICON share their thoughts on how clinical development can better satisfy the evidence needs of health technology assessment (HTA) bodies and payers.

In what ways are R&D protocols not aligned with reimbursement and access processes, and how is this impacting the industry and patients?

Arabella Stanley: Historically, reimbursement was not a consideration during protocol design for clinical trials, but that must now change.

It’s worth noting that in 2017, of the 50 drugs submitted to HTAs, only 20 were reimbursed because of various evidence challenges. HTAs frequently question the relevance of endpoints, study populations, comparators, study geographies, and even the design of quality-of-life (QoL) questionnaires. They use the same process to review specialty drugs as non-specialty drugs, which doesn’t allow for any unique circumstances, such as, for example, when there might be data on only 20 patients in a rare disease study with no comparator. If they find any holes in the data, they tend to either restrict coverage to a subpopulation, or require more evidence – either from a retrospective real-world study or from a fresh trial. So, by not incorporating the needs of payers early into trial designs, you risk spending more money later on and delaying access to critical medicines for unmet medical needs.

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