Case Studies Archive - Alacrita

EU value proposition and market access plan for orphan disease drug

Written by Cort | Jul 25, 2023 10:00:00 AM

Challenge:

A Swiss biopharma company was developing a therapeutic for a severe orphan disorder. The company had successfully obtained Breakthrough Therapy Designation by the FDA and PRIME status by the EMA. While the new drug promised to improve mortality rates, improve the efficacy of another existing drug when used in combination, deliver a more favourable side effect profile and lower infection rates, achieving reimbursed pricing in Europe represented a challenge. The drug was set to deliver the most benefit to those patients getting the lowest dose (infants) but pricing per course was perceived to be difficult.

Alacrita was asked to develop a robust HTA-rationalised value proposition, an evidence generation and market access plan, and provide ongoing market access support.

Solution:

Alacrita performed three streams of work: 

Stream 1: We critiqued and revised existing value propositions and associated evidence generation plans, particularly with reference to our experience and by comparison with health technology assessment (HTA) requirements. Specifically, we developed a value proposition incorporating all different target patient profiles, including unmet needs, the solution (describing the benefits of the new drug) and the value (the economic impact).

Our methodology ensured that the necessary and sufficient evidence for HTA submission purposes was identified investment in evidence generation was prioritized, recognizing that not all evidence gaps will be filled due to budgetary constraints; and any remaining evidence gaps were highlighted. Different domains of evidence (e.g. clinical, real-world evidence, patient-centred outcomes, economic) were considered. 

Stream 2: We organized a Pan-European full-day advisory board to pressure test plans. We put together a panel of advisors that included current and ex members of HTA bodies with representation from the major European HTA markets. The panel also included different clinician profiles treating the target indication. 

Stream 3: We provided ongoing support, comprising ad hoc support and advice throughout the execution of the market access, pricing and reimbursement plan and in particular, advice on appropriate vendors to support evidence generation work streams.

 

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