KEY POINTS OF CONSIDERATION To prepare for the introduction of gene therapies in haemophilia care, healthcare frameworks for evaluation and valuation will need to evolve to address the unique requirements of current and future innovations for treating this rare disease. The papers in this supplement provide an insightful and comprehensive state‐of‐the‐art assessment of these requirements and challenges. In terms of evaluation, the definition of a patient‐defined value framework that captures multi‐dimensional, patient‐centered outcomes is an important first step for determining the full benefit of gene therapy for persons with haemophilia. In terms of valuation and rewards for innovation, health systems will need to develop alternative payment models for risk‐sharing that will allow payers and society to address uncertainties about the ultimate clinical and economic value of these innovations. And health technology assessment authorities will need to exercise greater flexibility in evidence requirements given the unique features of data collection for a potentially curative therapy for a rare disease with long‐term uncertainties about durability of impact. Collaboration among stakeholders will be essential for developing the critical evidence requirements and providing the incentives needed to achieve sustainable budgets and broad access for persons with haemophilia worldwide.
Advances in the digitization of health systems and expedited regulatory approvals of innovative treatments have led to increased potential for the use of real-world data (RWD) to generate real-world evidence (RWE) to complement evidence from clinical trials. However, health technology assessment (HTA) bodies and payers have concerns about the ability to generate RWE of sufficient quality to be pivotal evidence of relative treatment effectiveness. Consequently, there is a growing need for HTA bodies and payers to develop guidance for the industry and other stakeholders about the use of RWD/RWE to support access, reimbursement, and pricing. We therefore sought to (i) understand barriers to the use of RWD/RWE by HTA bodies and payers; (ii) review potential solutions in the form of published guidance; and (iii) review findings with selected HTA/payer bodies. Four themes considered key to shaping the generation of robust RWE for HTA bodies and payers were identified as: (i) data (availability, governance, and quality); (ii) methodology (design and analytics); (iii) trust (transparency and reproducibility); and (iv) policy and partnerships. A range of guidance documents were found from trusted sources that could address these themes. These were discussed with HTA experts. This commentary summarizes the potential guidance solutions available to help resolve issues faced by HTA decision-makers in the adoption of RWD/RWE. It shows that there is alignment among stakeholders about the areas that need improvement in the development of RWE and that the key priority to move forward is better collaboration to make data usable for multiple purposes.
Objectives: South America (SA) is a continent with 400 million people and occupies 12% of the world's territory. It is composed by 12 countries and 6.75% of its population is below the poverty line, as defined by UN. The proper distribution of financial health resources, through an HTA process in public systems is potentially essential to improve the quality of health care expenditure. The objective of this study is to understand the incorporation process of new health technologies and compare the general health status in each country, regarding the Objectives of the Millennium (OM). MethOds: A public data collection was performed in official sources linked to UN, to governments of SA and the Unión de las Naciones Suramericana (UNASUR). Results: The public health financing in SA countries was between 2.43% and 6.20% of the GDP. An HTA process in an institutionalized and specialized form is in place in only 3 countries (Argentina, Brazil and Peru). Bolivia, Chile, Colombia, Paraguay and Venezuela do not have a specialized HTA process and the other four countries have no HTA process at all. Regarding the OM the decrease in child mortality, increase in vaccination, increase in malaria and tuberculosis treatments are among the closest to be achieved in all countries. There is a linear positive correlation of OM with the Human Development Index and with the percentage of GDP invested in public health but not with having a HTA process in place. cOnclusiOn: At this moment, there is no evidence that an HTA process in place helps SA countries to achieve the OM.
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