Drugs access: Why the NHS is failing those with rare conditions

Over the past few years, there has been a lot of negativity surrounding NHS England’s specialised commissioning programme.

We have been keeping a watching brief on this, and recently found ourselves in the thick of it, explaining to patients why two treatments - Humira and Remicade for severe refractory uveitis - were rejected for use on the NHS.

This decision is likely to affect around 100 children and 220 adults each year, putting them in imminent danger of losing their sight.

So the difficulty for us was not just breaking this news, but also trying to explain why this decision had been made.

NHS England said there was insufficient evidence to support the approval of these treatments; yet they are readily available in Scotland, Germany, France, the US and Japan.

‘Are patients missing out because of processes being operated by NHS England?’

Patients know this and we have already heard of one parent who is considering a move to Scotland in a desperate bid to save their child’s sight. So what really happened here? Have patients missed out because of the processes being operated by NHS England?

Understandably, people are anxious to know why NHS England made that decision.

They have a right to know. The NHS constitution states: “The system of responsibility and accountability for taking decisions in the NHS should be transparent and clear to the public, patients and staff.”

Yet, the exact opposite is happening with NHS specialised commissioning. 

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Lack of transparency

Since 2013, when NHS England took responsibility for this area of work, its decision making processes have been “iterative”. The goal posts have continually moved, the rationale behind their decision making remains unclear and we are unsure if the ethical framework was used to make decisions.

In January this year, NHS England launched a consultation on how it prioritises which specialised services and treatments to invest in. This was in response to a legal challenge made in 2014, where it was forced to abandon its controversial “scorecard” used to assess treatments for use on the NHS.

Sadly, its response to the 12 week consultation provided little clarity about future spending decisions. NHS England has recently stated that it will develop a new process for deciding which specialist treatments to fund but is yet to commit to a timeframe for this work.

‘There are insufficient numbers to make randomised controlled trials feasible for rare conditions’

To add to this, NHS England’s specialised commissioning web pages have not always offered up to date information, and this has led to confusion for patients and the public.

In fact, it remains unclear how patient case studies, feedback and involvement are used in decision making processes.

This contrasts sharply with the Royal National Institute of Blind People’s experience of working with the National Institute for Health and Care Excellence, where clear guidance states what evidence is required from patients and experts to inform health technology appraisals.

We also want clarity about the criteria used to assess treatments for rare and non-rare conditions – they must not be the same.

Rare conditions, by their very nature, mean that there are usually insufficient numbers to make randomised controlled trials feasible. In turn, this results in limited published evidence on the clinical effectiveness of treatments for these conditions.

We have additional misgivings about the lack of a formal appeals process to contest NHS England’s decisions around specialised commissioning.

Delays have a real impact on patients

We are not the first to raise concerns.

Treatments for patients with Morquio syndrome, Duchenne muscular dystrophy and Tuberous sclerosis complex recently hit headlines when the prime minister intervened and called on NHS England to “set out clearly the process and the timetable” for decision making.

The health service responded by creating an in-year commissioning policy for one of the drugs in question and referred the other two to NICE for assessment. Sadly, this has introduced further delays for patients as two of the treatments will now need to be reviewed by yet another committee.

‘Delays to decision making have a real impact’

In the case of severe refractory uveitis, further evidence is being submitted and NHS England will reassess its decision and may approve the treatments this financial year. This feels uncertain, however, as we remain in the dark about how and when the new evidence will be reviewed.

NHS England must not forget that there are real patients at the end of their processes and delays to decision making have a real impact.

Without rapid treatment, individuals with severe refractory uveitis could go blind.

Sight loss in childhood has major implications. It can hamper their motor and cognitive development, impede their educational and emotional development, and have a negative impact on their social interactions.

For adults, it can mean giving up paid employment and dependence on benefits, loss of independence and reliance on family members to undertake daily tasks.

NHS England must take action

There is simply no time to waste and this is why RNIB has joined a growing number of organisations calling on NHS England to urgently overhaul its decision making processes.

‘NHS England owes it to patients to fix this situation now’

We understand that NHS England has difficult decisions to make and is under severe financial pressure.

We also know that there is not a bottomless pit of money to fund every treatment.

However, NHS England owes it to patients, their families, as well as the taxpayer, to fix this situation now.

Clara Eaglen is policy and campaigns manager for the Royal National Institute of Blind People