• Letter says rare conditions drug policy is a “matter of extreme urgency”
  • Doctors warn patient access could be delayed until 2016-17
  • NHS England to set out timetable for new specialised commissioning process soon

Twenty senior doctors and experts have written an open letter to the NHS England board warning of ‘a high risk of preventable death’ for some patients due to delays in drawing up an access policy for a licensed drug.

The consultants urge NHS England to act now over what they say is a “matter of extreme urgency” to ensure patients in critical need receive the drug everolimus.

The drug has been shown to improve outcomes for children and adults with tuberous sclerosis complex, which causes growths in organs such as the brain and kidneys. The authors say that, without the drug, patients could suffer avoidable harm and death.

The letter, addressed to 17 NHS England board members, is signed by doctors and experts working across the NHS including specialists at Alder Hey Children’s Hospital and consultants in paediatrics, neurology and kidney disease. It is also signed by senior geneticists, scientists and clinical leaders.

In the letter they say: “The current position is this: around 32 patients with critical need, whose doctors believe everolimus treatment is their best or only option, have no hope of access to funding.

“Most have been waiting many months. Approximately half of these patients are at imminent risk of a catastrophic event (renal bleed or kidney failure) with a high risk of preventable death.

“It has been made clear by officials that ministers cannot intervene and that the power to introduce an interim funding policy to avoid needless suffering and death lies with NHS England. With power resides the responsibility and we urge you to act upon it.”

Their letter, sent in support of the charity Tuberous Scelerosis Association, follows a two year delay in drawing up a policy for everolimus which has already been blamed for at least two “absolutely preventable” deaths by a consultant.

In most cases patients with TSC cannot show they are exceptional and therefore don’t qualify for individual funding requests. An NHS England spokesman said in the past two financial years 46 applications were made by doctors to treat TSC patients with everolimus and 15 were approved.

In a specialised services circular from NHS England to its area teams in September 2013, seen by HSJ, the organisation accepted there was an issue with patients accessing drugs to treat some rare conditions.

It said: “In situations where we don’t have a policy and these patients are part of a cohort we are unable to approve funding through the [individual funding request] process. This means that patients who would have been treated in the past are now being refused access to treatment, not because it isn’t indicated but because a clinical access policy is yet to be written.”

In their letter, the consultants warn that access for the remaining patients in need could be delayed until 2016-17 and said the annual cost of the drug, at £36,000, is similar to the cost of treating renal failure and dialysis, which is a major risk for TSC patients.

They say: “Whilst we entirely accept that there needs to be a process of evaluation, the delays in NHS England’s work programme have resulted, and will further result in, avoidable harm. This issue affects real people. They are mothers and fathers with small children. They are young adults struggling to be well enough to access education and employment.

“They cannot afford to sit it out whilst NHS England resolves its current issues.”

NHS England has not put in place an interim funding policy for access to drugs to treat rare conditions after the previous process was abolished by the Health and Social Care Act 2012. Its replacement process was scrapped by NHS England last year amid claims it was discriminatory against people with rare conditions.

It is now consulting on a new policy for prioritising funding for specialised services, which is due to end later this month.

A spokesman for NHS England said: “We have received the letter and will be responding shortly. NHS England’s regional panels have approved 15 applications for everolimus to treat tuberous sclerosis over the last two years, as well as three for sirolimus [another drug sometimes used to treat TSC].”

“We will shortly be setting out our timetable for establishing the new specialised commissioning prioritisation process, and will continue to work closely with stakeholders on this important work.”