Orphan Drugs & Rare Diseases

A different approach.

Rare is the new normal

There is an increased focus on rare diseases, and consequently more products receive the Orphan Medicinal Product (OMP) designation.

The marketing authorization incentives for OMPs such as an expedited process and more limited evidence than other medicinal products result in misalignment between the clinical trial results and the requirements from the various HTA-bodies. Additionally, the nature of rare diseases often sets limitations for which evidence is feasible and ethically responsible to collect, given the high unmet need.

Some HTA-bodies has acknowledged the natural limitations for OMPs when meeting the standard HTA requirements and have tried to address this by relaxing some of the standard requirements under certain circumstances, e.g. The Process for STAs of medicinal products for very limited patient populations with severe diseases in Norway and the NICE Highly Specialised Technologies reviews in the UK.

However, there is a high unmet need for solutions on how to overcome the evidence gaps and how to effectively assess OMPs within the normal value framework

Key Challenges

Some of the key challenges for OMPs are highlighted below:

Small samples, lack of statistical power
Uncertain clinical pathways & lack of comparative data
Limited/short trial duration
Limited clinical and Pro/HRQol evidence
Uncertainties in cost effectiveness modelling
High cost – often not within standard cost-effectiveness thresholds

Consequently, there is a high need for pragmatic and manageable solutions for rare diseases in the HTA-assessment process, such as supportive Real World Evidence (RWE) and Innovative agreements structures.

NHTA has great expertise in OMPs and finding pragmatic solutions e.g. through innovative agreements and supportive RWE.