Six months ago we launched our campaign – and today we are delighted to announce our first significant victory in our Fight4Treatment.  The Health Service Journal (HSJ) had today reported that NHS England are to publish a policy that will allow patients with urgent need to get funding for Everolimus for those who’s health means they cannot wait for a national policy to emerge.  

Having supported the development of a service specification and prescribing policy for Everolimus for submission to NHS England (NHSE) we learned that delays and inefficiencies in their work programme meant no decision would be made in the foreseeable future. 
We were also horrified to learn that under NHSE’s own policies the Individual Funding Request (IFR) route had been effectively sealed off at the point where more than 5 patients had requested access, thereby triggering the need for a national policy.     
Up until March 2014 there had been a policy for urgent and critical need patients to access Everolimus.  This policy was not updated or reissued, resulting in funding requests for urgent need being screened out rather than considered – with no right of appeal.   
We owe a substantial debt of gratitude to those of you who have protested, petitioned, enlisted the help of your MP, written to the Prime Minister and shared your stories.  This is a first and substantial step forward.  It’s critical now that NHS E get on with review of national policies and a service specification for TSC.  Your help is still needed.    Look out for further updates and subscribe to our e-newsletter if you do not already receive it [insert link].   
We won’t stop until our NHS clinics are properly supported and resourced and all people with TSC have access to the best in care and treatment.  Keep up the brilliant campaign work – write to your MPs, share your stories and keep on supporting the #Fight4Treatment campaign. 
Health Service Journal Article

Exclusive: NHS England proposes new rare condition
drug access rules

May, 2015 | By Shaun

England is set to publish new guidance, described by charities as ‘life
saving’, on access to licensed drugs for patients with rare conditions and in
‘critical need’, HSJ has learned.

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.

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.

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

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.

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.

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.

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

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.”

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.

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.

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.”

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.

relation to the new guidance for critically urgent cases, an NHS England
spokesman said: “We are currently refreshing the standard operating procedure for
considering these cases, as well as producing information on it for patients
and clinicians, and will be publishing these shortly.”

The article is available to view online here