The association of health insurers (ZN) has announced a new pilot project, the Orphan Drug Access Protocol (ODAP), which is intended to make promising new drugs for rare diseases available to patients more quickly for a “socially acceptable price”. Under the pilot, patients will have access to eligible non-oncology orphan drugs but reimbursement will be delayed pending the collation of additional data on effectiveness.
Under ODAP, a protocol is drawn up “with criteria for starting, stopping and effect of a medicine. Appropriate use is thus realised immediately at the start of the treatment. The idea at ODAP is that it is agreed with the manufacturer to make the medicine available free of charge at the start of the treatment. Only when it has been demonstrated for the patient that the medicine has an effect will the health insurer take over the reimbursement for that individual patient”, states the ZIN.
According to the Amsterdam University Medical Centre (UMC) which will coordinate the pilot, to qualify for ODAP, a given orphan drug must meet the following criteria:
- registered with EMA or expected registration within 9 months
- intramural application (ie given/prescribed in hospital)
- cost less than € 10 million per year
- considered to be promising but with uncertain effect
- responds to an “unmet need” (defined as a new treatment with an expected high effectiveness for which there are no alternatives).
For eligible drugs, a protocol is drawn up which includes information about the disease to be treated, the position of the new drug in relation to other treatments, “start and stop criteria and evaluation criteria”. Subsequently:
Phase 1: The manufacturer makes the medicine available free of charge (for an unspecified duration).
Phase 2: Individual reimbursement by health insurers.
In case of proven effectiveness on an individual level, the health insurer reimburses the costs for that patient. Since the effectiveness of the drug at group level is not yet sufficiently clear, “reimbursement by health insurers during this phase must do justice to this uncertainty. Therefore, the price in phase 2 is lower than the requested list price”.
Phase 3: Group-level reimbursement
After a certain period of time, insurers decide on reimbursement at the group level. The time needed to reach a decision depends on the product and the robustness of the data collected.
The ZIN notes that the pilot phase involves three to five new medicines and several hundred patients.
Although the initiative is framed as accelerating patient access to orphan drugs, the question remains if, from a manufacturer’s perspective, there is an incentive to prioritise access in the Netherlands under these conditions?
- www.zn.nl, “Naar een nieuwe, gecontroleerde toegangsroute voor veelbelovende weesgeneesmiddelen”, 23rd May 2022
- https://medicijnvoordemaatschappij.nl/, “Naar een nieuwe, gecontroleerde, toegangsroute voor veelbelovende weesgeneesmiddelen”, accessed 26th May 2022
- EUCOPE, “Netherlands: Dutch health insurance companies announce a pilot project “ODAP” which will postpone reimbursement of non-oncology OMPs”, 25th May 2022