Written by Iro Malekou Email: [email protected]
What is happening?
OD (orphan drug) privilege status under the AMNOG procedure means that ODs are currently exempt from providing comparative data for their assessment. They are assumed to have a positive added benefit (with the lowest rating possible being a positive but unquantifiable added benefit), even when the data is insufficient to support this. If sales over 12 months surpass the €30 million mark, this privilege is revoked, and a full assessment is performed based on comparative data.
However, Germany’s IQWiG director, Thomas Kaiser, reiterated decade-old calls for reforms in OD assessments in the German HTA. He stated that OD privilege status in the German HTA will be at least “questioned” with the start of the EU joint evaluation in 2025.
While EU evaluation with joint clinical assessments (JCA) of ODs is planned to begin in 2028, assessment of oncology drugs and advanced therapy medicinal products (ATMP) with an OD status is expected to begin in the next two years. JCA will provide analyses including comparisons with standard therapies without a rating recommendation. This will then be considered by EU member states for their pricing and reimbursement decisions.
The changes brought by the implementation of the EU joint HTA could alter in the current German procedure. Concrete adjustments will be discussed in due time with the Health Ministry, G-BA and IQWiG.
“The orphan privilege is basically aimed at financially promoting orphan drugs. Our primary concern is not to question this objective at all and not to say that orphan drugs should be available at low cost. It is to say that we want to disclose the knowledge about newly introduced orphan drugs in the same way as we disclose the knowledge about drugs that are not for rare diseases”, Kaiser explained.
What are the implications?
It is thought that revoking the OD privilege will prevent unfair assessments compared to non-ODs which can be rated negatively, and will help differentiate the ODs that have a real added benefit and represent real progress.
Having robust comparative data between ODs and other currently available treatments would likely improve decision making in terms of pricing, reimbursement, and patient care. Another potential benefit could be a reduction in the strain placed on health budgets, as fewer OD’s will be successful in securing a positive rating and consequently reimbursement.
However, requiring full assessments for all OD’s from their launch might prove expensive and extremely difficult for manufacturers, due to multiple reasons.
Firstly, there may be issues in effectively demonstrating a clinical benefit in comparison to the standard of care. This could be due to difficulties in running the appropriate randomised controlled trials (RCTs) due to prohibitive sample sizes and costs.
Secondly, even if an RCT is performed, it is still possible that the assessment outcome is negative due to other factors (e.g., selection of an appropriate clinical comparator, choice of endpoint, etc.). Evidence from a 2021 IQWiG study showed that 54% of the ODs originally assessed with the positive rating “unquantifiable added benefit” could not prove superior therapeutic benefit when assessed under the regular procedure in Germany. This indicates how many ODs could be affected by a further reduction in flexibility within the German HTA, if the OD privilege is revoked entirely. It will be pivotal to see whether the JCA and in turn the German HTA adapt to accommodate for cases where the collection of comparative data is not feasible.
Overall, these considerations may encourage manufacturers to allocate more resources in designing and running comparative clinical trials to secure a positive rating. Alternatively, it is possible that manufacturers reconsider their plans to launch in Germany. This will limit access to life-changing treatments for patients and might have downstream effects on how the European market is perceived.
P4A are supporting clients launching in the EU during this transition period, by offering tailored solutions such as utilising rare disease registries to power RCTs or adopting alternative statistical methods. Reach out to us to see how we can support your new drug launches for cell & gene therapies and rare diseases.
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