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A UK website about atypical Haemolytic Uraemic Syndrome (aHUS)

Scotland looks into the way it does not approve ultra orphan drugs.

Scotland looks into the way it does not approve ultra orphan drugs.

Earlier this year, Dr Brian Montgomery was asked by Shona Robison, the Cabinet Secretary for Health, Wellbeing and Sport to lead an independent review of access to new medicines in Scotland and this is currently taking place. Patient groups, clinicians and pharmaceutical companies were invited to provide feedback as part of this review. This has given us an opportunity to comment on the SMC evaluation process of orphan and ultra-orphan drug, which is timely, given our recent experience with SMC and with NICE before that. As you will see from our comments, we do not think that the current process is fit for purpose, and we believe that many concerns are widespread amongst others who have experienced the current process. Dr Montgomery is expected to report back to the Scottish Government in a few months, and his findings will be published. It will be interesting to see what impact the review may, or may not have on future practice in Scotland.




April 2016


Dear Dr Montgomery


aHUSUK was formed in 2011 in response to the need to campaign for effective treatment for people affected by aHUS. Our small charity is run by volunteers, and also aims to support affected patients and families and improve knowledge and awareness of aHUS.


We are pleased to have the opportunity to make some comments for this review, largely based on our recent experience of the ultra-orphan evaluation process, which resulted in a decision by SMC not to recommend the medicine. This decision was at odds with that taken for the rest of the UK, where the medicine was accepted in 2015, having received broader consideration, and where there was greater inclusion of the patient voice and more flexibility in decision-making.


Currently, the need for a patient voice is acknowledged and we are grateful to have been able to participate in the evaluation process. However, the patient voice is limited at the moment by a restrictive format and we lacked sufficient opportunity to raise all the issues we felt needed consideration in order to meet the complexities and challenges of evaluating orphan and ultra-orphan medicines robustly.


In our case, there was no opportunity for proper consideration of the likely budget impact from the potential for reducing the dose of the drug or stopping treatment. Had the format allowed, we could have provided evidence from various trials which are already taking place in a number of countries to demonstrate the progress being made into the understanding of treatment options, thereby benefitting patients as well as budgets. We believe that this should have been an important real life consideration in a robust evaluation of an innovative, but expensive, new medicine, particularly as it provides the first and only clinically effective treatment and can transform people’s lives.


A more flexible PACE process and the participation of PACE representatives at the Committee meeting could have provided this opportunity. In fact, we were only able to briefly allude to the existence of trials in PACE, and we had to observe the lack of any reference to this important consideration during the course of the Committee meeting without being able to rectify this omission. As there was no reference within the DAD either, we are not aware that SMC members saw any evidence from these trials.


It is unclear what the real impact patient group submissions and PACE have had on decision making, even though this is supposed to be a major factor in the process. The current constraints of the Committee meeting do not permit adequate representation of their views and we do not feel that the brief summary of patient group input provided in the DAD “reflects the views of the group.” Time and effort had been spent trying to write as comprehensive an overview as possible within the confines of the format, and this was not properly reflected.



The right to appeal decisions should be extended to patient groups and clinicians if they have concerns about decisions. We have not been able to do this despite believing that more evidence should have been taken into consideration.


At the moment it is not possible for the SMC to be seen to deliver fair and equitable decisions because the current process is inadequate, overstretched and under-resourced. With such a workload, members cannot be expected to be able to give full consideration to all the available evidence.


The ability to make conditional recommendations could help improve access to orphan and ultra-orphan medicines in a timely manner. Account could then be taken of factors such as ongoing developments in knowledge and understanding of rare diseases and new medicines, and the likelihood of further new treatment alternatives in the future, with their associated potential for cost reduction. It could also facilitate ongoing reviews and dialogue with pharmaceutical companies. We believe that this is another reason why opposing decisions were reached within the UK in the two recent evaluations of the same medicine, for the same price.


Finally, under the current system, it would appear that there is a price threshold above which no medicine can be recommended, regardless of any other factors or the time and effort put in by the participants into the process. If there is no chance of justifying the treatment’s cost in relation to its health benefits, even when these are immense, there seems to be little point in evaluating the medicine in the first place.  Orphan and ultra-orphan medicines need a more relevant evaluation process if the unlucky few with rare diseases are to have any chance of equitable access to effective treatments. In contrast, the general population can access “cheaper” medicines, at great overall expense, even when they lack evidence of clinical effectiveness, and offer poor value for money.



















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