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6ER-008 Development of a risk-sharing model based on the clinical performance of onasemnogene abeparvovec (Zolgensma)
  1. AC Martins De Figueiredo1,
  2. RP Pinheiro Gonçalves Marques2,
  3. AP Mecheiro De Almeida Martins Silvestre Correia1,
  4. AF Cosme Silva2
  1. 1Faculdade de Farmácia da Universidade de Lisboa, Lisbon, Portugal
  2. 2Centro Hospitalar Universitário de Lisboa Norte – Hospital de Santa Maria, Lisbon, Portugal


Background and importance Zolgensma is an innovative gene therapy for spinal muscular atrophy (SMA) infants. Nevertheless, the life-long clinical follow-up needed for understanding the long-term effectiveness of Zolgensma in combination with an exceptionally large single payment represents scientific and financial challenges for the pharmaceutical industry, regulators and payers. The so-called Performance-Based Risk-Sharing Arrangements-Performance Linked Reimbursement (PBRSAs-PLR) are financial models that have been developed for reducing uncertainty through greater investment in evidence collection, while a technology is used within a healthcare system.

Aim and objectives The scope of this investigation comprised the development of a hypothetical PBRSA-PLR for Zolgensma Gene Replacement Therapy (GRT).

Material and methods A review of the literature was constructed, comprising five phases: (a) identifying the research question; (b) searching for relevant studies; (c) selecting studies; (d) analysing data and (e) presenting the results. A comprehensive English-language literature search of the electronic databases PubMed and Science Direct was undertaken to identify published papers. Data were collected and analysed until May 2021.

Results We propose an outcome-based scheme based on Zolgensma performance in terms of sustainability of the clinical effect. The relevant outcomes should be the subsequent for a given SMA infant: (a) overall survival and (b) event-free survival. We further suggest an annuity-based payment scheme to reduce the consequences of the annual budget impact with a pay-over-time of 5 to 15 years to increase patient access. More favourable outcomes could be achieved if SMA infants started treatment earlier. Thus, we propose a maximum 50% refund for Zolgensma early dosing in SMA infants (until 3 months old), and a maximum 25% refund for Zolgensma late dosing in SMA patients (after 3 months until 9 months old), if Zolgensma fails to meet the agreed-upon outcomes and predefined timing of outcome assessments.

Conclusion and relevance We conclude that it would be possible to mitigate uncertainty around the incremental budgetary impact and cost-effectiveness of Zolgensma GRT. Nonetheless, it should be outlined that innovative payment schemes should only be applied in circumstances where there is scope for such mechanisms to effectively reduce decision uncertainty so that the probability of long-term cost-effectiveness can be improved.

Conflict of interest No conflict of interest

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