Review of Access to New Medicines

6.3 How the new approach to assessment of ultra-orphan medicines is operating in practice

6.3.1 Discussion with stakeholders on this aspect of the Review produced a variety of responses but running through the responses was a consistent theme that, while the new approach was welcomed and had brought about some improvements, it was not yet suitable for all ultra-orphan medicines.

6.3.2 At the heart of the concerns was the issue alluded to in the section on definitions namely that medicines for very rare conditions, the so-called "true ultra-orphans" are still disadvantaged by the new approach and as such access, if not denied, was being made more difficult as patients and treating clinicians were reliant on IPTR and PACS.

6.3.3 The T&FG discounted QALY weighting for the new approach as applied to ultra-orphans, opting instead to recommend that a decision-making process not based on the cost per QALY should be used for medicines defined as ultra-orphans. The preferred approach was for SMC to use a framework of explicit criteria for evaluating these medicines, without performing weighting and scoring. This was seen to parallel the approach being adopted by NICE and consequently would guard against inequity of access between the home countries of the UK.

6.3.4 There is a widespread feeling that this has not had the expected impact and that an unstated price threshold exists and remains the predominant influencer when true ultra-orphan medicines are assessed. Some take the view that despite the modifications in process and the resultant increased flexibility offered, those making submissions have still not been able to make sufficiently strong clinical or commercial cases to support the use of the medicines under consideration.

6.3.5 Closer examination of the data on the twenty-two SMC decisions since the introduction of the new approach suggests that the success rate for recommendations of ultra-orphan medicines is relatively high at 62%. However, when the medicines are sub-classified as ultra-orphans used at the end of life, ultra-orphans used in the treatment of cancer and other malignancies and ultra-orphans used in the treatment of very rare conditions then the figures for each sub-grouping are starkly different.

6.3.6 Only one out of seven medicines (14%) used in the treatment of very rare conditions was approved despite supportive PACE statements. This compares with 11 out of 12 (92%) approved amongst medicines being used at the end-of-life and 2 out of 3 (67%) approved in the treatment of cancer. The widely held perception is that ultra-orphan medicines used in the treatment of very rare conditions, are all extremely expensive and this is the barrier to their approval. The full break down of approval figures for ultra-orphan medicines is given in Table 2 below ( Paragraph 6.4.9).

6.3.7 This finding ties in with the use of the term "true ultra-orphan" in several of the stakeholder discussions. This term acknowledges that there are a small number of high-cost medicines for which the cost-effectiveness data is at a level that, even with supportive PACE statements and the application of modifiers, the medicines still do not attain approval.

6.3.8 Despite the T&FG recommendation that QALYs would not be used in the assessment of ultra-orphan medicines, cost effectiveness remains an important consideration as part of any health technology assessment.

6.3.10 On the basis of this observation there would be merit in considering not just adopting a new definition of "true ultra-orphan" medicine as proposed above, but also exploring the development of an alternative assessment and approval pathway for these medicines.

6.3.11 Despite the new approach the SMC process is not providing patients with increased access to true ultra-orphan medicines. It does seem that even though QALYs are not being applied these medicines are failing to satisfy any test of cost effectiveness. As stated elsewhere this potentially calls to question the role of SMC in the assessment of these medicines. The ability of SMC to undertake a robust assessment of clinical and cost effectiveness is not in question however, it may be that SMC should no longer be the group or process responsible for making the final decision about availability.

6.3.13 The situation to be avoided is creating a series of individual arrangements for true ultra-orphan medicines as each new one is licensed. While it is now being accepted that one size may no longer fit all it is still important to restrict the number of alternative sizes ideally to one.

Recommendations

11 Develop and implement a new assessment and approval pathway for true ultra-orphan medicines that restricts the role of SMC to health technology assessment and places the responsibility for the final decision on availability elsewhere.