Recent Statistics on Orphan Approvals in Scotland and England

In October 2009, the OHE published research that compared access to orphan medicinal products (OMPs) in selected European countries.  At the request of the UK Orphan Medicines Industry Group [1], OHE recently updated to May 2011 some of the data included in that research.  Using the same methodology, this new analysis focuses on decisions taken on orphan drugs by the Scottish Medicines Consortium (SMC) and the National Institute for Health and Clinical Excellence (NICE).  Included are the 74 orphan indications approved by the European Medicines Agency (EMA) up to May 2011.

Decisions by the SMC

The SMC has issued 55 decisions for the 74 orphan indications.  Because of resubmissions and reviews for some, a total of 69 decisions have been published.

As figure 1 shows, companies most often submitted full submissions for OMPs to the SMC.  Compared to non-orphan products, the proportion of non-submissions is slightly higher, perhaps because the expense of HTA may be prohibitive relative to the prospects for approval.  Abbreviated submissions were less frequent for OMPs, possibly because OMPs are more likely to be new molecules/active substances.

Figure 2 shows that rejection is more likely for orphan than for non-orphan indications. (Note that ‘not recommended’ includes products that were automatically rejected by SMC because the manufacturer did not make a submission to it.)  Of the total number of orphan rejections (43), 29% were due to non-submissions (12). For the remaining 31, the key reason for rejection stated in the SMC’s Detailed Advice Documents (DADs) was lack of economic evidence (‘economic case has not been demonstrated’); this means that the cost per QALY estimate was too high, or the model involved too much uncertainty, or no cost effectiveness model was provided. In 20 of these 43 rejected submissions, the clinical evidence was considered sufficient.  For the remaining 23 rejected submissions (more than half of the rejections), the level of clinical evidence available at the time of the review was considered by SMC to be inadequate.

Four of the decisions classified as ‘restricted’ in the DADs are effectively recommendations because the restriction coincides with the licence, i.e. the ‘restriction’ is that the medicine can be used only by a specialist in the relevant medical area.

To limit the analysis to one SMC decision per orphan indication, only the most recent submission is included in figure 3.  This shows approval was unrestricted for 10 orphan products and restricted for 14.  Thirty-one orphan products were not recommended.

As part of the submission, manufacturers supply details about the budgetary impact expected during the first and the fifth years after launch. These estimates may include the number of patients receiving treatment. Table 1 summarises statistics for patient numbers extracted from the 41 submissions that included this information.

Table 1. Range of reported patient numbers per indication (total indications = 41)Year 1Year 5
Average across indications2943
Minimum12
50 percentile1225
Maximum179179

NICE Decisions on OMPs[*]

NICE has assessed nine indications for orphan conditions:

  • azacitidine for mylelodyplastic synromes
  • imatinib for gastrointestinal stromal tumours and leukaemia
  • lenalidomide for multiple myeloma
  • sorafenib for RCC and hepatocellular cancer
  • temsirolimus for renal cell cancer (RCC)
  • trabectedin for ovarian cancer and soft tissue sarcoma

In four of the nine indications (temsirolimus and sorafenib, for both indications, and trabectedin for ovarian cancer), the overall decision was to not recommend use of the medicine. In three instances (imatinib, for both indications, and lenalidomide), the decision was to restrict use to a subgroup within the licensed indication. In two cases (trabectedin for soft tissue sarcoma and azacitidine), the treatment was recommended for use subject to a Patient Access Scheme.

Two NICE Technology Appraisals for other products were initiated, but not completed either because the NHS had already decided on treatment protocols (pulmonary arterial hypertension) or the drug (nilotinib) was considered in tandem with other treatments.

[*]This section was updated with new data on 9 January 2012.

Contact Martina Garau at the OHE with questions or for additional information.


[1] The Orphan Medicines Industry Group (OMIG) consists of orphan drug manufacturers that are members of the Association of the British Pharmaceutical Industry (ABPI).

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