Orphan drugs & rare diseases
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
Some of the key challenges for OMPs are highlighted below:
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.
Nordic HTA has great expertise in OMPs and finding pragmatic solutions e.g. through innovative agreements and supportive RWE.