Access to orphan medicines: rare expertise and patient experience needs to be core part of decision

Blogpost by Leela Barham, Independent Health Economist and 2020health Research Fellow for Empower Access to Medicines

Orphan medicines are used to treat some of the rarest conditions; conditions that affect less than 5 per 10,000 people in Europe.  Some of these conditions are so rare that in parts of the UK access is not even about hundreds but tens of patients recieving a medicine, and in some conditions that amounts to a handful of families. Deciding who should get what in the NHS is always controversial as there is a limited amount of money to go around, and that’s perhaps more acutely being felt now than ever before.  Some orphan medicines cost a great deal (but by no means all of them), so it is right to explore their value for money.

But one of the particular (but not only challenges) for orphan medicines is that there is limited expertise; few clinicians who see enough patients and few patients who can put forward their case for the benefits that orphan medicines can bring and hence make the case why they should be able to access them.  In some instances, there may not be a patient organisation who can speak up for affected patients at all.  This means that the benefit side of the equation may be less clearly interpreted than for medicines for common diseases, whereas the cost of the orphan medicine is much clearer.  Knowing the true benefit of an orphan medicine is a particular issue when the evidence base can be limited too;  because there may be few patients who can be identified, willing and able to take part in trials. Some products are seen as promising but are not fully worked up into cost effectiveness analysis in terms of the cost per Quality Adjusted Life Year (QALY).  That doesn’t mean that there isn’t a benefit that is worth it; but that we’re not yet able to know for sure.

The UK has developed over time more sophisticated approaches to make decisions about access to all types of medicines.  Agencies such as the Scottish Medicines Consortium (SMC), the All Wales Medicines Strategy Group (AWMSG) and the National Institute for Health and Clinical Excellence (NICE) have all looked at some of the orphan medicines currently licensed.  They each take into account value for money in making recommendations for their use to the NHS, but some have allowed for some of the particular challenges of making the case for orphan medicines.  The SMC for example, also considers whether the drug: treats a life threatening disease; substantially increases life expectancy and/or quality of life; can reverse, rather than stabilise, the condition; or bridges a gap to a “definitive” therapy.  And they all bring in expertise and try to involve patients and the public in their decision making.

But more needs to be done; with clinicians and patient organisations still telling of their frustration that they have to make their case over and again to different parts of the NHS across the UK, and that some decisions are reversed by virtue of the right expertise being brought to bear.  It must also surely make economic sense; having a single national view of the evidence done once, and done well, will avoid time spent repeating the same efforts across the UK and within the devolved regions especially when there are so few who can contribute to each process. That should encourage consistent access where the experts and patients feel that it is worth it. It could also free up time to look again in the future, as the evidence base improves and experience by clinicians and patients builds up over time to help know what the value for money really is; and that means knowing what the benefits of the medicine are to patients and carers.

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1 Response to Access to orphan medicines: rare expertise and patient experience needs to be core part of decision

  1. John Mills says:

    There is a national organisation called Rare Disease UK which helps to support and co-ordinate all rare diseases, including setting up a database of all those suffering from different rare diseases so that all the information from those isolated cases can be be brought together. Many of the drugs that are used in treating rare diseases have been developed and licensed for treating other, more common conditions. It is the use of these drugs “off label” ie for uses for which they are not approved and licensed, that causes problems. International cooperation is also important as a disease where there are only a handful of cases in the UK, when looked at globally, probably has several handsful of cases.

    John Mills – Vasculitis UK (Vasculitis is a “rare disease”!).

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