All Party Parliamentary Group hearing on access to medicines in England

How can we improve access to medicines for rare disease patients in England? How does the current commissioning system differ from those in the devolved nations? What are the biggest issues facing patients, families, clinicians and industry during the evaluation of rare disease medicines?

These were just a few of the questions raised at the All Party Parliamentary Group (APPG) on Rare, Genetic and Undiagnosed Conditions hearing on access to medicines, which took place in the Houses of Parliament on Tuesday 11 October. Parliamentarians including Baroness Neville-Jones, Cheryl Gillan MP, Mark Durkan MP, Greg Mulholland MP and Julian Sturdy MP were given the opportunity to hear from an expert panel of patient representatives, clinicians and industry members in what was the first of a series of events seeking to change the way rare disease medicines are commissioned in England.

Chaired by Ben Howlett MP, the APPG was established in February 2016 to increase awareness of rare, genetic and undiagnosed conditions in parliament and help to ensure that patients and families affected by these conditions have access to appropriate care and support.


Following an introduction from Ben Howlett MP, attendees heard first from Janis Clayton, General Manager at PTC Therapeutics; Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy UK; and Dr Adnan Manzur, Consultant Paediatric Neurologist at Great Ormond Street Hospital. All three have been involved in the evaluation process for commissioning ataluren, a medicine licensed for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). When used successfully, the medicine slows the progression of nmDMD, enabling children affected by the condition to maintain the ability to walk for longer.

This medicine was initially selected for evaluation in July 2014 through NHS England’s specialised commissioning route. However, following legal challenges to the validity of this process in December 2014, which halted the evaluation of a number of medicines through this route, George Freeman MP requested that ataluren be reviewed by NICE, despite it not meeting the selection criteria for the Highly Specialised Technology (HST) evaluation process. The decision for a positive recommendation for ataluren was finally published in April 2016 – almost two years after the European Medicines Agency had approved the medicine. During this time, patients, clinicians and industry were forced to overcome a number of significant challenges that were costly not just financially, but to the health of the patients.

The average lifespan of people with nmDMD is less than 30 years.  Two years is a long time in the life of a patient with nmDMD. Janis Clayton estimates that during the time lost to the weak process, around seven children with nmDMD have lost the ability to walk. The delay in access to ataluren carries significant implications for these young lives. Nic Bungay shared the experiences of patients and families working with Muscular Dystrophy UK, including those of Louise and Gary Hill, whose eleven-year-old son Archie has nmDMD. They stated:

“Being told your child will probably die before you has to be one of the most devastating things you can tell anyone. The impact it had on us could not be put into words.”

Dr Adnan Manzur also discussed the impact of the delay in commissioning the medication on families of patients, who attend hospital every six months for monitoring, and always see a decline in their child’s condition.

“Being told your child will probably die before you has to be one of the most devastating things you can tell anyone. The impact it had on us could not be put into words.”

the Hill family

The hearing discussed a number of issues in the evaluation process and commissioning routes for ataluren, including the way decisions about Independent Funding Requests (IFRs) and Clinically Critically Urgent (CCU) applications are made. They also discussed the need for greater integration and communication between NICE and NHS England, a theme that permeated the discussion about everolimus, a medicine approved for the treatment of tuberous sclerosis complex (TSC)-related brain tumours in September 2011.


“Over the past four years, it feels as though we’ve been forced into playing an exceedingly cruel and extended game of policy snakes and ladders, where the snakes predominate”, said Jayne Spink, CEO of the Tuberous Sclerosis Association (TSA), describing the journey patients and clinicians have undergone throughout the evaluation process for the commissioning of everolimus for tuberous sclerosis complex-related brain tumours. Following the introduction of the Health and Social Care Act 2012, NHS England became the body responsible for developing a commissioning policy for everolimus through its specialised commissioning route. However, in December 2014, this work was suspended and the clinical reference group (CRG) leading on the work was formally disbanded in February 2015. As the policy had not yet been completed or approved, no funding was available for everolimus. The process was not restarted until July 2015, following much campaigning by the TSA, significant media and political interest, and a letter from 20 NHS consultants to the Board of NHS England highlighting the situation. In this time, the TSA also learned via a Freedom of Information request that around 30 IFRs had been declined by NHS England.

“Over the past four years, it feels as though we’ve been forced into playing an exceedingly cruel and extended game of policy snakes and ladders, where the snakes predominate”

Jayne Spink – CEO, Tuberous Sclerosis Association

The everolimus for TSC-related brain tumours policy was finally included with a significant backlog of policies sent to the June 2016 meeting of the Clinical Priorities Advisory Group (CPAG), to be considered for funding for 2016/17. These policies were subjected to a new prioritisation mechanism which significantly disadvantages medicines for rare diseases. When the provisional outcomes of the CPAG prioritisation process were announced in July 2016, everolimus for inoperable TSC-related brain tumours was listed as having been ranked in priority level 5, meaning that in the opinion of the CPAG, the policy was of the highest cost and lowest benefit grouping. Patients were shocked at this result. As Jayne Spink noted, the consequence of denying access to patients is death in many cases. Dr Chris Kingswood, a Consultant Nephrologist who has been involved in the commissioning process of everolimus and also presented on the panel, had been surprised by the result, given that research shows everolimus is almost 100% successful for TSC treatment. Dominic Wake, Government Affairs, Patient Advocacy and Communications Manager at Novartis, the pharmaceutical company that produces everolimus, highlighted a number of issues with NHS England’s evaluation of everolimus regarding transparency, timeliness, stakeholder engagement and quality. He suggested the solution was “to increase NICE capacity and adapt its methods so NICE becomes the only body to assess medicines for use in the NHS.”

At present, it remains unclear what the final outcome of the evaluation process will be. Meanwhile, as Jayne Spink pointed out, “the clock is ticking on the lives of 20 young people.” Attending the hearing were a family whose eight-year-old son has TSC with related brain tumours. The family was keen to remind those present that the four years that have passed since everolimus was first licensed by the EMA is a long time in the a child’s life. In the case of their own son, it is half his life.

“The clock is ticking on the lives of 20 young people.”

Jayne Spink – CEO, Tuberous Sclerosis Association

The panel then listened to the views of a number of industry experts, as well as patient representatives, who have been involved in the commissioning processes for a number of rare disease medicines. There was broad agreement that key issues surrounding the current system include integration and communication between NICE and NHS England; the transparency of processes managed by NHS England; the perceived lack of clinical expertise at critical stages of the medicine’s review; and differences between the systems in the devolved nations, which many argued were more streamlined than those currently in place in England. At the end of the hearing, the panel agreed to meet again for a second hearing with representatives from NICE and NHS England.

You can read what Jayne Spink, CEO of Tuberous Sclerosis Association, had to say at the hearing here.