Consultation on idebenone for treatment of LHON

Idebenone is a drug used in the treatment of Leber’s hereditary optic neuropathy (LHON), a mitochondrial disorder that causes vision loss. LHON usually starts with blurred vision in one or both eyes and often leads to blindness in both eyes. This can happen quickly (over a number of months) or may take longer to progress (over a number of years). Most people with LHON will have both eyes affected within 1 year of diagnosis.

There have been a number of clinical trials to determine the clinical effectiveness and safety of idebenone in patients with LHON. These studies have provided some evidence to show that idebenone may help improve vision in people with LHON who have experienced vision loss, and help prevent people from becoming blind. There are currently no available treatments other than supportive care.

Idebenone is licensed to treat visual impairment in patients with LHON and is currently available across Europe, including Scotland.  NHS England have recently reviewed evidence prepared by NICE to decide whether idebenone will be routinely commissioned for LHON. The report concluded that there was not enough evidence to consider making this treatment available.

NHS England are proposing to not routinely commission idebenone for LHON. There is now a public consultation for stakeholders to provide their views on the evidence used to reach this decision.  The consultation is open for 30 days and will close on 22nd August 2019.  We would encourage anyone with views on this to submit a response using the online form that can be found here:

The LHON Society have provided guidance to their members for completing the survey, which they have kindly shared below:

The documents available on the consultation website contain some very technical reports containing more than 100 pages.  The survey itself is quite short and not as scary as you might first think.  The documents you mostly need to be concerned about are the Policy Proposition and the Clinical evidence criteria (although all of them are worth reading if you have the time).

The first two questions in the survey are about you (the person submitting their views). The rest of the survey is made up of five questions numbered 3 to 7. The guidance below may help you complete these.

Question 3.  Has all the relevant evidence been taken into account?

You should make your own judgement on this question.  We would argue that it has not, as the main evidence relied upon is an admittedly flawed survey that is now 10 years old.  Had negative information been unearthed since then it would have been taken seriously, whether peer reviewed or not, but where positive information exists it is effectively ignored if not peer reviewed.  The international consensus statement of leading clinicians worldwide for LHON is an important document that should be considered.

Question 4.  Does the impact assessment fairly reect the likely activity, budget and service impact?

Rather a debateable point.  Given that the treatment is being rejected there is effectively no impact on budgets etc, and given the fact that most patients give up on visiting their ophthalmologist in the absence of any treatment, then there is no ongoing cost to the NHS that could be saved by approving the treatment.  Abandonment of patients is a poor argument, however, and this is essentially what is happening at the moment.

Question 5.  Does the policy proposition accurately describe the current patient pathway that patients experience?

This is under page 16 in the policy proposition and there is no description at all.  I would invite you to describe your own “Patient pathway” here, or your LHON story.  Write as much as you like – this subject is largely unknown and unquantified and the real cost of LHON is buried and ignored as a result.

Question 6.  Please provide any comments that you may have about the potential impact on equality and health inequalities which might arise as a result of the proposed changes that have been described?

We believe it is unacceptable that only those with the money or skills to purchase idebenone themselves will have access to it.  This also potentially exposes these individuals to unacceptable risks of receiving fake or contaminated supplies.  The fact this treatment is approved in 16 countries (including Scotland and Ireland) yet the UK is one of the wealthiest countries in the world is also unacceptable.

Question 7.  Are there any changes or additions you think need to made to this document, and why?

Feel free to go off on one!

Anyone can complete the survey and it contains free text boxes for you to add your own feelings and views, which we would encourage you to do.  It is not essential to answer every question and you can also explain if you do not understand any of the questions.