Value-based pricing for advanced therapy medicinal products: emerging affordability solutions

Abstract

The emergence of advanced therapy medicinal products (ATMPs), a disruptive class of health technologies, is generating important challenges in terms of value assessment and their high prices introduce critical access and affordability concerns. The aim of this article is to analyze the challenges of traditional value assessment and price and reimbursement methods in the evaluation of ATMPs and to characterize the current and prospective financing solutions that may ensure patient access and affordability for these health technologies. Standard Health Technology Assessment (HTA) is not designed for ATMPs, and may delay access to these health technologies, thus a broader concept of value is required. As a consequence, value-based pricing methodologies have been gaining terrain to cope with the specific challenges of ATMPs. The pricing and reimbursement framework should ensure the balance between encouragements to innovation and maximization of value for money for payers, through the attribution of a fair price to new health technologies. Early scientific advice by regulatory and HTA bodies to developers is key, as it will contribute to diminish the perspective gap between developers, regulators and payers. The high efficacy/high price dynamic of many advanced therapies will demand novel financing models, both in the EU and US. Managed entry agreements (MEA), with financing being conditional to the submission of additional evidence, associated with methods of leased payments, may offer effective strategies to address the uncertainties caused by the evidence gap associated with ATMPs, ensuring affordable and sustained access.

Addressing Risks Derived From the Commodification of Substances of Human Origin: A European Proposal Applicable Worldwide

“…33 ATMPs are not strangers to this reality, with prices that bear no obvious relation to their aggregated costs of development and manufacturing. 43 A recent example is the academic chimeric antigen receptor (CAR) T-cell therapy granted hospital exemption by the Spanish Agency of Medicines and Medical Devices after demonstration of quality, safety, and efficacy. This academic CAR-T is reimbursed by the Spanish National Healthcare System at one third the price of the commercial CAR-Ts available in Spain.…”

Feasibility Study Protocol: Investigating Family Reported Outcome Measures (FROMs) in the Estimation of Societal Value of Advanced Therapy Medicinal Products (ATMPs)

“…Therefore, in the context of ATMPs broadening the de nition of "value", to capture elements not considered by the QALY, such as societal outcomes, may be necessary [26]. Doing so could support outcome-based pricing models, where consideration of the value requires demonstration of positive or desired outcomes beyond cost-effectiveness, are expected to become more prevalent [30].…”

Análisis estratégico para la implementación de las terapias avanzadas en España

“…En este sentido, en la literatura reciente publicada, distintos autores han comentado que los métodos convencionales de evaluación económica pueden no ser totalmente apropiados para las terapias avanzadas u otras terapias complejas, requiriendo ciertas adaptaciones, como la utilización de perspectivas de análisis más amplias, utilizar análisis para mejorar el manejo de la incertidumbre o la incorporación de los resultados reportados por los pacientes y las experiencias reportadas por los pacientes (PROMs y PREMs, por sus siglas en inglés respectivamente). En este aspecto, la recogida de datos como la carga de la enfermedad, la vía de administración y el impacto en los cuidadores ayudaría a capturar mejor el valor del medicamento (Tabla 7) 55- 58 .…”

“…Es por ello que algunos autores opinan que se deben utilizar métodos alternativos de financiación para estas terapias 58, 97 . Dichos esquemas, condicionados a la remisión de evidencia adicional, alcanzar los resultados previstos y/o a realizar el pago de manera diferida, pueden ofrecer estrategias efectivas para manejar la incertidumbre, compartiendo riesgos entre las partes y permitiendo así un acceso más asequible y sostenido a los pacientes 58 . Además, con los actuales marcos de evaluación, se dificulta la posibilidad de capturar el valor asociado a estas terapias, principalmente…”