The Scottish Medicines Consortium (SMC) has issued 55 decisions on the 74 orphan drug indications approved by the European Medicines Agency (EMA) up to May 2011, while the National Institute for Health and Clinical Excellence (NICE) has assessed only five indications for orphan conditions, new research shows.

In fact, at May 2011 the SMC had issued a total of 69 decisions on the 74 approved orphan indications, due to some resubmissions and reviews, according to data produced by the Office of Health Economics  (OHE), in an update of research, originally published in October 2009, comparing access to orphan medicinal products (OMPs) in certain European countries. The update was produced at the request of the Association of the British Pharmaceutical Industry (ABPI)'s Orphan Medicines Industry Group (OMIG).

The OHE's research shows that for 62% of cases, companies submitted full submissions for OMPs to the SMC, compared to 55% for non-orphan indications, and that only 4% were abbreviated submissions, against 21% for non-orphan indications. This lower figure is possibly due to the fact that OMPs are more likely to be new molecules/active substances, the authors suggest. 

However, the study also shows that rejection by the SMC was more likely for OMPs, at 62% compared to 37% for non-orphan indications, and with positive decisions granted for only 12% of OMPs (31% for non-orphan indications) and restricted decisions for 26% (against 32% for non-orphan use).

Out of total of 43 OMP indications rejected by the SMC, 12 (29%) were due to non-submissions, while for the remaining 31, the key reason stated in the Consortium's Detailed Advice Documents (DADs) was lack of economic evidence. This means that the cost per Quality Adjusted Life Year (QALY) was too high, the model involved too much uncertainty or no cost-effectiveness model was provided, says the OHE. 

In 20 of the 43 rejected submissions, the SMC considered the clinical evidence to be sufficient, while for the remaining 23, the level of clinical evidence available at the time of the review was considered to be inadequate.

So far, NICE has assessed only five indications for orphan conditions, the OHE report goes on. These are for: - Pfizer's Torisel (temsirolimus) for renal cell cancer (RCC); - Bayer's Nexavar (sorafenib) for RCC and hepatocellular cancer; and - Novartis' Glivec (imatinib) for gastrointestinal stomal tumours and leukaemia.

In three of the five instances - for Torisel and both indications for Nexavar - the overall decision at NICE was not to recommend use of the medicine, while for the two indications for Glivec, use was restricted to a subgroup within the licensed indication, says OHE.

It also points out that two NICE Technology Appraisals (TAs) were initiated but not completed, either because the NHS had already decided on treatment protocols (pulmonary arterial hypertension) or the drug - Novartis' Tasigna (nilotimib) for Philadelphia chromosone-positive chronic myeloid leukaemia - was considered in tandem with other treatments.

- The OMG works closely with stakeholder group Rare Disease UK (RDUK), which earlier this year published a report containing proposals for bringing together existing knowledge and expertise "in a coherent, logical and equitable way so that the founding principle of the NHS can become a reality for all patients with rare diseases in the UK."

Currently, these patients are being denied access to orphan drugs that have been granted EU marketing authorisation, says RDUK, which sees an "urgent need" to reassess the mechanism and methodology for appraising the value of OMPs for reimbursement on the NHS.  The group calls for the evaluation methods and processes for such products to be refined, their funding to be organised nationally from a central source, and new value-based pricing (VBP) to consider issues specific to orphan drugs and ensure patient input.