OBJECTIVES

NICE’s managed access programme includes arrangements for 34 cancer therapies and 5 orphan treatments. These time limited arrangements enable patient access to promising therapies whilst further evidence is generated to address outstanding clinical uncertainties. Increasingly, health technology assessment bodies are presented with submissions using immature evidence without evidence from randomised clinical trials. MAAs can help to bridge evidence gaps using data from a real world clinical setting. NICE’s approach to managed access for cancer and orphan treatments has evolved in parallel. Both approaches have relative strengths and areas for improvement.

METHODS

All MAAs in orphan, ultra-orphan, and cancer therapies in England were reviewed and key data extracted and compared on (i) data sources for evidence generation, (ii) outcomes collected, (iii) length of agreement, and (IV) time to produce agreement (from final NICE committee meeting date to MAA publication date).

RESULTS

More cancer MAAs (28/34 [82 percent]), (orphan 0/5) had an ongoing clinical trial as the primary data source. In MAAs that included Real World Evidence (RWE) collections (cancer [91 percent], orphan [100 percent]) the mean number of outcomes collected were 2 in cancer compared to 11 in orphan MAAs. Cancer agreement lengths were shorter (median of 26.4 months), (orphan 60 months). Median time to agreement publication was 71 days (cancer) compared to 68 days (orphan).

CONCLUSIONS

Orphan MAAs were observed to have longer, more complex data collection arrangements, in part due to a greater reliance on RWE collection (usually via a registry). A wider range of clinical uncertainties identified in orphan medicine evaluations translates to the collection of a high number of outcomes. Comparatively, cancer MAAs operate RWE collection via one national registry, with standardised short-term outcomes to validate an ongoing clinical trial. Publication times were similar. Orphan MAAs are distinct from cancer MAAs and will require bespoke arrangements with comprehensive expert consultation.