This year’s end of February wasn’t only about Rare Disease Day. We also bade farewell to the European Medicines Agency which on 28th February turned off the lights in its London headquarters. Starting 1st March, the Agency is officially headquartered in Amsterdam, The Kingdom of the Netherlands.
The European Medicines Agency (EMA) reviewes medicines and issues recommendations for the European Commission (EC) to grant or revoke marketing authorisation (MA) – effectively, a license for drugs to be traded across the EU. Whilst medicines for common diseases may be individually approved by national regulators, all orphan drugs – drugs for rare disorders – have to go through the centralised procedure at EMA. This is to speed up their path to the patient.
EMA has traditionally been unsually supportive of new medicines for SMA. Against widespread expectations and despite scarce trial data, the agency approved nusinersen (Spinraza) in the widest possible indication and in record time. It has closely co-operated with SMA Europe. Currently EMA is reviewing Zolgensma – gene therapy for SMA. Should a no-deal Brexit take place, the EU marketing authorisation for Zolgensma will not be valid in the UK, unless the UK Parliament decides to accept the future EC decisions as binding on the UK.
Joseph Irwin, TreatSMA trustee and regulatory consultant in pharmaceuticals, wrote:
As someone involved in the development of medicines the European Agency has been a place I have regularly attended for treatments for cancer, heart disease, paediatric medicines and many others including for spinal muscular atrophy. Though it will continue to provide a great service for the registration of medicines it is sad to see the staff leave for the Netherlands. It has been a great success and I am sure the EMA will continue to be a success bringing treatments to patients across the EU.
What will be the situation now? Joseph writes:
To be fair, the MHRA – the UK medicines agency – was a lead contributor to the assessment of medicines. It will continue to do this, but we will miss out on collaboration and have a double assessment. Also UK as a 60 million population market is a small percentage of the 500 million patients across the whole of the EU. The current trend in the UK is to delay access to medicines like Spinraza. Though the NICE process is highly regarded it is being used by the NHS to delay medicines becoming available. The Secretary of State talks of the UK being a global leader in healthcare, yet for children with a rare disease like spinal muscular atrophy it has taken over 2 years, since registration, to grant access. They have elected to use the EAP to keep medicines available using the company’s good will to provide medicine for free. Yet elsewhere in the EU it is paid for by governments. So either the UK steps up and is a world leader in medicines or becomes a backwater for healthcare.
The Secretary of State talks of the UK leading in the genomic projects. We did RD-Connect which is a EU-funded genomic project for rare diseases led by Newcastle University for the EU. Like TREAT-NMD, these were collaborations using the best EU scientists and clinicians to bring forward world-beating research and development of medicines. I know as I have been part of their programs. Spinraza came out of these collaborations. To collaborate is to succeed.
As the UK and EU part their ways, we wish all the EMA experts a smooth transition to a new country; energy levels as high as previously; and as much dedication to orphan drug approval as they had in London.