Outcomes-based Agreements

January 23, 2020

Woman holding digital medical devices globe

Denizar Vianna, the appointed head of Science, Technology and Strategic Input Secretariat (SCTIE) in Brazil, is expected to lead the changes within the Brazilian healthcare system, also advocating for the implementation of risk-sharing agreements (RSA). Read here how the first RSA pilot went.

The newly-elected government in Brazil is expected to bring changes to the health care system landscape, with a higher focus on more affordable and fair public reimbursement. Denizar Vianna is expected to lead these changes. He has made public statements regarding his focus on defining public priorities, budget constraints, considerations of unmet needs and the future technologies landscape, and is also a strong advocate for the implementation of risk sharing agreements (RSA) in Brazil. The first RSA pilot was closed in June 2019 for Spinraza in Spinal muscular atrophy (SMA) types II and III.

SMA is a rare genetically-inherited neuromuscular disease that causes progressive muscle weakness and loss of movement, and is the most common genetic cause of death in infants. There are 4 types of SMA, and type I is the most severe and common. Type I patients never develop the ability to sit unsupported, and typically do not survive after the first 2 years if no intervention is provided. Spinraza is the first treatment approved beyond supportive care, and got a fast-track approval 3 months after submission by ANVISA (the Brazilian Health Regulatory Agency). Access was granted in November 2017.

The first submission for public funding happened in January 2018 for SMA type I, however CONITEC (the Brazilian HTA agency) gave a negative preliminary recommendation due to fragile clinical evidence, lack of demonstrated clinical improvement, inconsistent cost-effectiveness relation, and evidence that it could compromise the financial sustainability of SUS (The Unified Health System). In January 2019, Biogen made a new submission proposing a 62 percent discount vs. list price and achieved a positive funding recommendation 3 months later for SMA type I. The fast assessment by CONITEC was a result of the high awareness and perceived unmet needs for SMA. The public consultation for the first submission achieved a record of 37K contributions, followed by 42K contributions for the second public consultation, which received 95 percent in favor of incorporation.

For SMA types II and III, due to uncertainties on efficacy and budget impact, the MoH decided to implement the first RSA pilot in the public setting, which is the biggest innovation in the public sector since the early days of tech-transfer. For Denizar, it is important that each contract is tailored case-by-case and with transparency. He also gave public statements about being open to discussing ideas and building future RSA models. The RSA reached with Biogen consists of a patient cap based on epidemiology, a negotiated price discount, and payment based on performance, which will be monitored by the MoH through Albert Einstein Hospital. The data will be used by CONITEC for a new assessment in three years. Since the beginning of November, patients with SMA types II and III have been able to request access to Spinraza and be directed to a reference center by the MoH.

Although there are still several challenges for rolling out outcomes-based contracts, including infrastructure availability for results monitoring, patient database development, and legal constraints (e.g., the MoH cannot receive paybacks in the case of treatment underperformance), this is a new access opportunity in Brazil that is especially relevant for orphan drugs. Offering the MoH support for developing the required capabilities and having high public awareness, perception of unmet needs, and indication severity can improve chances of success. Denizar highlights the importance of the MoH and industry engaging in discussions to improve access that guarantees the sustainability of the public system.

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