info@rareimpact.euA review of the challenges and proposals for improving patient access to advanced therapeutic medicinal products in Czech Republic
Executive Summary
RARE IMPACT is a multi-stakeholder initiative working to improve patient access to advanced therapy medicinal products (ATMPs). This patient-focused initiative aims to assess challenges and propose actionable solutions to concerns regarding access to these transformative rare disease treatments in Europe. Through engagement with health technology assessment (HTA) agencies, regulatory bodies, payers, patient groups, clinicians, manufacturers and other experts across Europe, RARE IMPACT partners identified challenges and have proposed solutions for better access to ATMPs in Europe.
This report aims to stimulate multi-level stakeholder discussions on patient access to ATMPs and is not intended to capture all challenges to patient access to ATMPs. The RARE IMPACT initiative was launched at the European Conference on Rare Diseases and Orphan Products in 2018.
The data on patient access to orphan products points to an association between economic status of a country and the accessibility to orphan products
For example, the public expenditure on orphan drugs as a proportion of all pharmaceutical expenditure in the Czech Republic is 2.25% compared with 3.84% and 6.5% in Austria and Belgium, respectively. The Czech Republic has provided access to CAR-Ts recently, suggesting a willingness to provide access to innovative drugs. But there are challenges related to the assessment of ATMPs, affordability / willingness-to-pay and healthcare service infrastructure to address in order to provide sustainable patient access to these treatments.
There is no clear pathway or exemption in the assessment process for ATMPs in the Czech Republic. Direct negotiations between manufacturers and insurers are required to secure patient access. This requires a cost-effectiveness and budget impact analysis. For highly innovative products, where effectiveness is not well understood at the time of application for reimbursement, conditional reimbursement can be obtained for up to three years. A compassionate use programme (CUP) is also possible in the Czech Republic in advance of launch. Leveraging these two programmes to develop an adaptive assessment pathway would allow mature data to be developed to better inform reimbursement decisions.
With regard to affordability, the Czech Republic use a series of measures to control prices due to a focus on budget impact. Consequently, the introduction of managed entry agreements (MEAs) and innovative funding options should be explored to control expenditure over time
Payers are more likely to find the annuity model of payment less risky if it is contingent on health outcomes being met, which will become evident over time. Hospital budgets will initially be responsible for paying for ATMPs, as health care providers have limited budgets for reimbursement of medicinal products (contracted lump sum from health insurers). A state contribution to the hospital drug procurement budgets for ATMPs could address this.
The Czech Republic is familiar with using bilateral agreements to provide access to healthcare innovation to citizens. This will be of importance for ATMP when few treatments centres will be available across Europe. Czech authorities should proactively prepare for this scenario by establishing protocols for providing timely access.
To date, the Czech Republic has one certified CAR-T treatment centre and as hospitals are technologically advanced, more are in place to become certified centres. However, this will require planning to understand which ATMPs being developed could be provided within the Czech Republic, the typical support services required, and the geographical dispersion. Identifying infrastructure and/or services that can be reconfigured for ATMPs will utilise what is already in place in order to reduce the burden on capital infrastructure. The impact on health service should be addressed as early as possible through horizon scanning to better prepare providers and reduce the time to patient access.
|
Domain (Impact)* |
Challenge |
Proposed Solution |
Feasibility** |
|---|---|---|---|
| Assessment | |||
| 2 | No formal HTA for ATMPs identified, and direct negotiations with health insurers requires HTA and budget impact assessments | Widen the population covered under temporary reimbursement and CUP to generate data to inform an adaptive assessment process | ++ |
| Affordability | |||
| 4 | A focus on budget impact does not capture long-term value of ATMPs | Innovative payment options such as risk-sharing agreements should be explored | + |
| 4 | Hospitals are pressured and incentivised to keep costs low (covered under single health insurer lump sums) | A state contribution to hospital budgets to support the reimbursement of ATMPs | + |
| Availability | |||
| 4 | The use of cross-border incentives to get patient access is uncertain | “Gemeinsam Grenzenlos Gesund” (Unlimited Health Together) cross-border scheme between Czech Republic and Austria could be reconfigured and expanded for ATMPs | ++ |
| Accessibility | |||
| 4 | It is unclear if infrastructure required for ATMP delivery is available | Identify infrastructure/services that can be reconfigured for ATMPs | + |
Notes
*The working group assessment of the relative impact of the challenge of each domain on patient access is represented by Harvey balls from highest (represented by a full blue Harvey ball) to lowest (represented by an empty, white Harvey ball); **Feasibility: Working Group assessment of feasibility of solutions to be implemented. + low feasibility, ++ medium feasibility, +++ high feasibility.
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