Access to Medicines Hearing: A report on the APPG on Rare, Genetic and Undiagnosed Conditions

Access to Medicines Hearing, APPG on Rare Genetic and Undiagnosed Conditions, 11 October 2017, Committee Room 9, Houses of Parliament

Report by John H Sharpe, Chairman, Reverse Rett.

The first three speakers at the hearing were from biotech companies and all had essentially the same message which was that there was inordinate delay between the licensing of new medicines by the EMA and the commissioning of these medicines by NHS or NICE.

These speakers were:

Sally-Anne Tsangarides, Santhera
David Lewis, Bio Products Laboratory
Mark Bell, Sobi

Santhera had developed a medicine for a genetic condition known as LHON (Leber’s hereditary optic neuropathy). This condition starts with blindness developing in one eye, often in teenage years, which quickly spreads to the other eye. Typically blindness can develop in between two to four months. The drug was approved by the EMA in September 2015 and two years later has still not been accepted in the UK. It has been referred to  Clinical Reference Group (CRG) but after 15 months there has been no communication from the group.

The second speaker was from Bio Products Laboratory who have developed a medicine called Coagadex

On March 17th 2016 the European Medicines Agency granted marketing authorisation for Coagadex. Coagadex is indicated for the treatment of bleeding in patients with hereditary factor X deficiency. Coagadex is the first and only treatment licensed specifically for this rare bleeding disorder in Europe. 18 months later this medicine is still not permitted in the UK despite the fact that medicines for Factor 8, 9 and 11 are all available. The CRG studying this application has been disbanded, NHS England changed their mind during the process and have retrospectively changed the rules for Haemophilia products.

SOBI are an integrated biotechnology company dedicated to rare diseases. They have a medicine to treat Ammonia buildup called Ravicti which is much more pleasant to take and will cost no more that the currently available medicine which is extremely unpleasant to take, especially for children. The EMA authorisation was given in November 2015. Despite no extra cost and being much easier to take, two years after the EMA approval, the medicine has not been approved for use in the UK.

The next two speakers on the agenda were from NICE and the NHS. Unfortunately, neither of these speakers was able to attend. The NHS said they could not find a senior enough person to attend, and NICE said they could not cope with the change of time from the afternoon to early evening.

In view of the constant criticism that both organizations came under this was extremely disappointing as they could not attempt to answer some of the criticisms.

There was considerable discussion as to whether it was bureaucratic inefficiency or lack of money which was leading to these delays in authorisation for the UK. The NHS processes were not transparent, they were far too complicated and the patient’s voice was not heard at any stage of the process. One person present summed up the situation by saying that the NHS was not interested in patient groups or new medicines or rare diseases.

Research commissioned by Shire found that of 143 medicines approved by the EMA, Germany had approved 133, France 116 and the UK only 63. Looking at the time between EMA approval and national authorisation the situation was that Germany took 12 months (not completely sure of this figure), France 19.5 months and the UK 47.6 months.

The UK clearly has a lot to learn from Germany and France.

Germany and France have both given a much clearer political priority to the treatment of Rare Diseases. Only if the UK brings sustained political pressure to bear will NICE or the NHS make any changes. At the moment they are too strong and bound in their ways to expect any change unless they are put under concerted political pressure

The APPG together with support from the Genetic Alliance, after discussion with stakeholders and reviewing the German and French processes will develop a greenfield process for the authorisation of drugs approved by the EMA and take it to ministers and the NHS and NICE to try and get a fundamental change in the way this process is handled in the future.

You can read Genetic Alliance UK's own briefing re the Access to Medicines Hearing held on Oct 11th 2017 on the Genetic Alliance UK website here.