European Union Health Technology Assessment Fit for Rare Diseases

Following the work on the Orphan Drug Incentives, Copenhagen Economics worked together with the OD Expert Group on market access for rare disease treatments addressing challenges at the health technology assessment stage: clinical evidence requirements and stakeholder representations.

Clinical evidence

Health Technology Assessment (HTA) processes typically require evidence generated through randomised controlled trials (RCTs) which is considered the gold standard for evaluating the efficacy and safety of medical interventions. However, in the case of rare diseases, due to the small population size RCTs are often not feasible, or the outcomes of such trials cannot be interpreted using standardised statistical significance thresholds. This leads to a lengthier assessment process and creates uncertainty around the benefit of the treatment impacting pricing decisions.

To address this issue, the OD Expert Group proposes implementing a two-step procedure to assess treatment for rare diseases under the EU HTA. The first step involves a feasibility dialogue aimed at determining whether a standard data dossier is feasible or if alternative evidence sources and methodologies should be considered. If a standard data dossier is deemed unfeasible during the feasibility dialogue, then in the second stage, the HTA body and developer collaborate to define what a feasible dossier based on complementary evidence should look like.

The OD Expert Group provides specific guidance documents to inform and facilitate dialogue at each stage.

Stakeholder representations

In the field of orphan medicinal products, there are few subject matter experts and small patient populations, drastically confining the pool of relevant stakeholders who are in a position to provide useful input to EU HTA Joint Clinical Assessments (JCAs). The limited number of experts are prone to having experiences that could constitute biases.

To handle and mitigate the interests of stakeholders, the OD Expert Group proposes the following actions:

Download the reports below.

Related work

European Expert Group on Orphan Drug Incentives Orphan medicine incentives