Due to the limited availability of treatment options for rare indications, such as certain cancers (e.g. hairy cell leukaemia), there is a substantial opportunity for the introduction of new interventions. However, the need to improve access to efficacious and safe therapies for the treatment of these indications is accompanied with numerous challenges, that make research and Health Technology Assessment (HTA) in rare diseases difficult. Key challenges faced by pharma companies looking for access for their new interventions in rare diseases are depicted in Figure 1.

Authors: Jatin Gupta, Vishnu Priya Wahal

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