Exclusive: NHS England proposes new rare condition drug access rules

The proposed new policy, which is expected to be signed off by NHS England chief executive Simon Stevens, follows allegations by some medical specialists that a delay to changing existing processes has meant patients with critical need “have [had] no hope of access to funding” despite facing a “high risk of preventable death”, and led to avoidable deaths.

NHS England has contested this.

However, HSJ has learned NHS England is preparing new standard operating procedures for the consideration of critically urgent cases where there is no established policy, with the intention of publishing it next week.

An email sent last week to NHS England’s patient and public voice assurance group by Fraser Woodward, head of engagement and partnerships for specialised commissioning for NHS England and seen by HSJ, said a new draft standard operating procedure for dealing with critically urgent cases was being developed.

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Mr Woodward said the new policy would require “sign off from Simon Stevens” but that once published, “would update and supersede” a circular on access for patients in urgent need, which was published in 2013.

The 2013 circular highlighted a problem for patients with rare conditions which had arisen from the Health Act 2012 commissioning restructure, and put in place what it said was an interim access process to deal with this, which it said would expire in March last year. It warned that when primary care trusts were scrapped, the number of patients applying for an individual funding request for interim access to drugs to treat rare conditions was aggregated nationally. This meant there were too many to be considered exceptional, which the policy limited to no more than five a year.

In recent months, several charities have told HSJ they believed this interim policy had expired in March 2014 as planned, and that this had led to patients in urgent need to being able to access drugs. NHS England has insisted that, despite the stated expiry date, the interim policy remains in place and was being used.

However, the Tuberous Sclerosis Association, which represents patients with tuberous sclerosis complex, told HSJ it believed the replacement policy which has now been proposed could be a “life saver” for these individuals, compared to the current situation.

Association chief executive Jayne Spink said it could be “a life saver for the adults and children with critical need and TSC”. She added: “We will be testing the policy on our 30-plus critical need patients. What we then need NHS England to do is to get on with developing the policy for non-urgent cases.”

TSC can be treated by the drug everolimus, which was licensed for use in TSC patients in 2012, but has not been considered by the National Institute for Health and Care Excellence.

Ms Spink said the proposal should also help patients in critical need with other rare conditions. HSJ has previously covered concerns about those with Morquio syndrome, and Duchenne muscular dystrophy.

NHS England has insisted there is already a process in place to consider cases such as these as “individual funding requests”. However, it said an example in February had seen a request patient denied treatment, despite a “massive life threatening bleed”, because it was “representative of a group of patients who have a similar condition and are at the same stage of that condition and who could potentially all request the same treatment”. This meant, it said: “In this circumstance, this is a request for a service development and is not appropriate for consideration as an IFR.”

NHS England also said an interim policy remained in place for access for critically urgent cases. During 2013-14 and 2014-15 there were 46 applications by doctors to treat TSC patients with everolimus, and 15 approved.