The National Health Care Institute has, for the second time, evaluated its procedure for conditional inclusion (CI) of orphan drugs, conditionals and exceptionals. The report contains the evaluation questions and subsequent conclusions and action points.
CI procedure for orphan drugs, conditionals and exceptionals
The CI procedure makes patients with a serious, often rare disease eligible for temporary reimbursement of promising medicinal products whose effectiveness is still uncertain. The CI procedure allows time to gather evidence on the effectiveness while patients have access to the medicinal product. The National Health Care Institute tests the effectiveness on the basis of the criterion ‘established medical science and medical practice’. Only when a medicinal product or treatment meets this criterion will it be permanently eligible for reimbursement from the basic health care package.
Second evaluation CI procedure
The National Health Care Institute has prepared 5 evaluation questions. To answer these questions, we have conducted desk research and submitted questions in internal consultations and to external parties. Based on the answers, we have identified several areas for improvement.
No, not all medicinal products that meet the CI selection criteria will enter the CI procedure.
Follow-up action:
The National Health Care Institute wants to improve the selection of CI candidates. To that end, we will:
- Actively look for potential CI candidates.
- Find out how to resolve bottlenecks when conditionals are involved in a CI procedure.
Medicinal products that are not an orphan drug, conditional or exceptional, are not currently eligible for CI. Adjustment of this criterion may be needed, but adjustment of the other CI selection criteria is not necessary.
Follow-up action:
- The Ministry of Health, Welfare and Sport will take this point into account in the policy evaluation of the CI procedure.
Yes, the research proposal is assessed within the target lead time. And no, the agreement on a price arrangement and the drafting of a covenant are not completed within the projected 6 months.
Follow-up actions:
- The National Health Care Institute examines how to facilitate the drafting of the covenant and how to reduce the workload for the CI procedure.
- The National Health Care Institute indicates to the Ministry of Health, Welfare and Sport that marketing authorisation holders of medicinal products are asking for more transparency about budget considerations when agreeing to a reduced price for the medicinal product during CI.
The experiences of 3 CI procedures (4 medicinal products) with the procedure during a CI procedure are generally positive, but there are areas for improvement:
- Participation in a CI procedure involves a lot of time for attending physicians and the patient associations, but funding for this is not provided in the CI procedure. Treating physicians and patient associations indicate that it is important for them to be financially independent from the marketing authorisation holder.
- The mandatory secondary research was not considered useful in all procedures.
Follow-up action:
- The National Health Care Institute will discuss with the Ministry of Health, Welfare and Sport the need for independent funding of patient organisations, professional associations and data collection.
The action points from the previous evaluation have been largely addressed, but not all problems have been fully resolved. The main remaining issue, in addition to the above-mentioned need for independent funding of patient organisations, professional associations and data collection, is that while there is more clarity about the differences and similarities between CI, the drug access protocol (DAP) and the orphan drug access protocol (ODAP), streamlining is desirable.
Follow-up action:
- The National Health Care Institute, together with the Association of Dutch Healthcare Insurers (ZN) and the Ministry of Health, Welfare and Sport, wants to explore the possibilities for a joint procedure for conditional inclusion. The Ministry of Health, Welfare and Sport will take this point into account in the policy evaluation of the CI procedure.
Continuation
The National Health Care Institute will start working on the areas for improvement in 2025. In 2026, we will review the implementation of the conditional inclusion procedure again.