SMA treatment and CAR T-cell therapy are both innovative and expensive therapies. The EU, Australia and Canada carry out health technology assessments or other analyzes for treatments that have a financial impact on health budgets, either at the individual or population level .
Nusinersen is an antisense oligonucleotide that consists of a series of injections, which are continued depending on the response.
Tisagenlecleucel, a chimeric antigen receptor (CAR) cell therapy, is a single-use cell therapy approved in the European Union for refractory / relapsed acute lymphoblastic B-cell leukemia in patients up to 25 years of age or for adults with relapsed / refractory diffuse large B cell lymphoma.
The researchers said the goal of their work was to identify best practices that can be factored into OBMEA implementation methods, as well as to encourage extensive collaboration and information sharing.
They chose nusinersen and tisagenlecleucel because they represent different types of disease, type and class of therapy, and intended treatment population.
Building on previous work from the UK and Italy that looked at how evidence coverage is used for medical devices, researchers interviewed those involved in health technology assessments, expert payers, market access experts and academics to compile what is known about how OBMEAs are used. for these 2 therapies.
The authors noted that it is difficult to find information about these agreements and what they contain, sometimes due to the fact that it is confidential business information.
The responses they received discussed how countries were implementing OBMEAs and what was being done to overcome the challenges. OBMEAs based on individual results were aimed at ensuring appropriate use and managing continuation of treatment; in 2 cases, they were linked to payment schedules. The population-based agreements included coverage with the development of evidence.
For nusinersen, population-based OBMEAs are found in Belgium, England and the Netherlands, while Bulgaria, Ireland, Italy and Lithuania have used individual agreements. Individual agreements sometimes included budget ceilings which were used in price and reimbursement renegotiations.
A few other EU countries have provided non-specific information about their OBMEAs due to privacy concerns. Countries with decentralized systems, such as Canada, also have confidential agreements.
Australia, France, Germany, Hungary, Norway and Slovenia have covered nusinersen without entry agreements but may have used strict criteria regardless, the authors said.
For tisagenlecleucel, Australia, Belgium, England and France used population-based agreements and Italy and Spain used individuals-based agreements.
When it came to creating an OBMEA, only England and the Netherlands included patients in the process; for the most part, these agreements have usually been concluded between the marketing authorization holder and the payer.
Most countries attempt to collect data on these treatments for continuous evaluation, but the quality of the systems and platforms used varies, as does the type of information collected. Only England, the Netherlands and Spain intended to capture patient-reported results.
“The processes to ensure the quality, completeness and sufficiency of the data for reanalysis after coverage with the development of evidence were not always clear, nor were the analysis plans,” the authors noted.
The implementation of OBMEAs has proven to be tedious; To encourage transparency and reduce duplication of work, the authors said it would be ideal to create an international public portal for these agreements and related data, including the sharing of interim and final reports.
Facey KM, Espin J, Kent E, et al. Implementation of results-based managed entry agreements for rare disease treatments: Nusinersen and tisagenlecleucel. Pharmacoecon Open. Published online July 7, 2021. doi: 10.1007 / s40273-021-01050-5