Author Peter Sutton
Manoj and Sejal Thakrar and Gary and Louisa Hill headed to the Department of Health on 8 September for a meeting with the Health Minister, George Freeman MP, as part of the campaign for access to emerging treatments for Duchenne.
Shiv Thakrar (aged 4, pictured) and Archie Hill (aged 9) both have Duchenne, and their families are amongst those fighting to speed up access to potential therapies, as well as raising funds for research into treatments.
George Freeman has set up a review on access to treatments, the Accelerated Access Review, which is set to make early stage proposals in October.
The Minister said that a potential recommendation could be a ring-fenced fund for rare disease drugs, as is the case in Scotland and Northern Ireland, which families and Muscular Dystrophy UK have long been pushing for.
Also on the agenda is strengthening existing programmes for early access to new drugs, such as the Early Access to Medicines Scheme (EAMS). Currently no treatments for muscle-wasting conditions have been put forward under EAMS, but it’s essential that it is made as attractive to pharmaceutical companies as possible. At the moment, drug companies, even small ones, have to pay to apply to the scheme. This could put them off, and we’re pushing for the Government to help fund applications.
The Minister was also pressed on access to Translarna. The first NICE committee meeting on this new drug will be held on 17 September, where Gary Hill and Muscular Dystrophy UK Chief Executive, Robert Meadowcroft, will be giving evidence. Draft guidance will follow in October and if this is positive, then we’ll be pushing for NICE to move to final guidance without further delays.
George Freeman MP then stated
Duchenne Muscular Dystrophy is a devastating condition. I would like to pay tribute to the Hill and Thakrar families and Muscular Dystrophy UK for their efforts to improve the lives of people with the disease.This Government is committed to accelerating access to innovative twenty-first-century medicines and technologies for all patients.
Fast Forward: fast track to specialist treatments
With some potential treatments for Duchenne in late stage clinical trial and one, Translarna, already available in some European countries it’s vital that these drugs reach families in the UK as quickly as possible.
Our Fast Forward campaign is joining with families like the Thakrars and the Hills to make this happen.
For more information and to get involved with the Fast Forward campaign on access to potential treatments, please contact Peter Sutton on firstname.lastname@example.org or call 020 7803 4838.