OBJECTIVES : Pharmaceutical product lifecycles are normally characterised by protracted clinical development followed by marketing under exclusivity/patent protection during which returns on investment (ROI) can be realised. However, we are entering an era of transformational therapies with curative potential (e.g. gene and CAR-T cell therapies) for whom the window to realise returns on investment may be greatly curtailed. This also impacts emerging new standard-of-care which could rapidly become superseded. Nusinersen is the first and only EC-approved therapy (2017) to treat spinal muscular atrophy (SMA), a rare and fatal genetic disease. Nusinersen has demonstrated transformational patient benefits but requires chronic treatment. However, a potentially-curative gene therapy for SMA, onasemnogene‑abeparvovec-xioi, threatens to surpass nusinersen, first launched in 2019 (US). This research aims whether the reimbursement strategy and payer/HTA responses for nusinersen have been impacted by the impending launch of a potentially-curative competitor.

METHODS : Publicly-available HTA appraisal documents for nusinersen from EU5, Nordic countries, Australia and Canada were identified, and key information extracted.

RESULTS : HTA reports for nusinersen were identified from 12 countries, which were all were positive, despite the complexity of the disease state, lack of long-term supportive data, and high-cost of therapy. Recommendations were limited to SMA subtypes (17%: type 1; 33%: types 1-2; 25%: types 1-3; and 25%: other restrictions/conditions). The mean delay from marketing authorisation to first positive HTA recommendation was only 299 days (120-715 days).

CONCLUSIONS : Nusinersen has successfully translated marketing authorisation into rapid reimbursement across a wide range of payer bodies. However, restrictions and discounts have been necessary to facilitate this, such flexibility may have been driven by the impending launch of a competing potentially-curative gene therapy. As more potentially-curative therapies come to market, such approaches may become increasingly commonplace among recent entrants to maximise ROI under time-limiting circumstances.