Health Technology Appraisal and Access To Medicines; Experience of The All Wales Medicines Strategy Group, 2002-2014

Introduction: The All Wales Medicines Group (AWMSG) was established in 2002 to advise Welsh Government on the introduction of new medicines into Wales. We analysed the acceptance rate of all medicines submitted by manufacturers to AWMSG for health technology appraisal (HTA) in NHS Wales, as well as separately for a sub-group of those medicines indicated for the treatment of rare conditions (orphan medicines, licensed for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating and affects fewer than 1 in 2,000 people in the European Union[EU]).

Methods: HTA was conducted by AWMSG in its monthly meetings in public. “Recommended Medicines” were those approved either according to the full licensed indication or for a restricted (optimised) indication. Base caseincremental cost-effectiveness ratios (ICERs)for 26 of the 34 orphan medicines (including those classed as ultra-orphan medicines, a sub-set of orphan medicines defined as being used to treat conditions with a prevalence of ≤ 1 in 50,000 in the EU) were taken from the submissions by the manufacturer.

Results: In the period September 1st 2002 to December 31st 2014, 184 (81%) of the 228 HTA’s resulted in the medicine being recommended, either in full or with restrictions. In contrast only 20 (59%) of the 34 orphan medicines were recommended either in full or restricted (Fisher’s exact test, two-tailed p = 0.0072 compared with non-orphan medicines). In the case of ultra-orphan medicines, 8 (73%) of 11 medicines were recommended in full or with restrictions (Fisher’s exact test, two-tailed p = 0.2948, non-significant compared with the other 23 orphan medicines).

The base case ICERs for 26 of the 34orphan medicines appraised by AWMSG to date with the recommendations are shown in Table 1. Negative ICER values indicated that the medicine was claimed by the marketing authorisation holder to be more effective and less costly than the comparator (“dominant”). A Mann-Whitney test indicated that there was no significant difference between the ICERs for orphan medicines recommended and not recommended (U = 948, p = 0.88076).

Table 1. Base case ICERs for the 26 orphan medicines (including ultra-orphan medicines) appraised by AWMSG between 2002 and 2014,where ICER data was available.

Conclusions: Significantly fewer orphan medicines were recommended (either in full or for optimiseduse) than non-orphan medicines. The similarity of the median ICER of the recommended andnon-recommended medicines suggests that cost-effectiveness may not be theonly criterion for acceptance or rejection of an orphan medicine. New approaches arebeing piloted to ensure thatsocietal issuesarefully taken into account in AWMSG’s decision-making processes for orphan (including ultra-orphan) medicines.