Tag: PPMS

For English PPMSers

“I love deadlines. I like the whooshing sound they make as they fly by” Douglas Adams, Hitchhikers Guide to the Galaxy.

There is one deadline that happened quitely earlier this month. NHS England turned on the blueteq form ocrelizumab treatment for PPMSers living in England; they did at the last possible timepoint dictated by the law. The good news is that the requirements for someone with PPMS to be treated are relatively simple. All that is required from us is the following:

1. Confirmation the patient has a diagnosis of early PPMS with active disease, defined by the appearance of new lesions confirmed by two MRI studies taken at least 6 months apart OR one or more gadolinium enhancing lesions on one MRI over the last three years.

2. EDSS score <= 6.5

3. A decision agreed at an MS multi-disciplinary team (MDT) meeting.

4. Recording of all the above in the patient’s records (for audit purposes).

This has been a long time coming and I want to thank all my colleagues who signed-up to the open letter we sent to the Chief Medical Officer (see below). I suspect the letter may have helped find a funding solution. It is clear that when there is a will there is a way.

I am also beginning to realise that NHSE is actually on the side of the patient and contrary to my previous perceptions NHSE want patients to access effective treatments. The problem is they have limited budgets and are beholden to the Department of Health and the political system of the day.

We may have won one battle, but the war continues. PPMSers in Scotland still have no access to ocrelizumab. How can we live in a country, with a socialist healthcare system, that allows your place of abode to dictate your access to treatment? If there is anything we can do to help PPMSers living in Scotland please let us know.

Open Letter

Professor Dame Sally Davies
Chief Medical Officer
Department of Health
Room 114, Richmond House
79 Whitehall
London SW1A 2NS
Email:CMOweb@dh.gsi.gov.uk

24 September 2018

Dear Dame Sally

Ocrelizumab for the Treatment of Primary Progressive Multiple Sclerosis

We are writing to you as concerned healthcare professionals for help. We are neurologists, nurse specialists and allied healthcare professionals who specialise in multiple sclerosis (MS). Although patients with the relapsing-remitting type of MS have access to many disease-modifying treatments no treatments until now have been licensed for those with the primary progressive type of MS (PPMS). In its final appraisal document [FAD ID938] NICE has not recommended the first available licensed treatment ocrelizumab, for treating early PPMS.

The problem with NICE’s appraisal determination (FAD) is that the price for ocrelizumab had already been set for treating relapsing-remitting MS (RRMS, FAD TA533), but this price was considered not cost-effective for the treatment of PPMS based on its efficacy in the PPMS trial. As there are currently no licensed treatments for PPMS ocrelizumab had to be compared with best supportive care or no treatment. In comparison, when NICE appraised ocrelizumab for RRMS it was compared to all the other licensed disease-modifying therapies (DMTs).

We have been told that Roche had then agreed to lower the price for ocrelizumab so that it would be cost-effective for the PPMS indication. If this was accepted it would mean ocrelizumab having two NHS prices, a higher price for the treatment of RRMS and a lower price for the treatment of PPMS. Apparently, the Department of Health is not prepared to support differential pricing, despite having a mechanism with the blueteq system for tracking prescribing for the PPMS and RRMS indications.

NICE’s decision in association with the Department of Health’s Rules means an irrational decision has been made. It also creates inequity. People with PPMS who are prepared to pay for ocrelizumab privately will be able to receive the therapy, potentially in an NHS institution. Similarly, those who are fortunate may be able to move and be treated with ocrelizumab in another country where ocrelizumab is covered for the PPMS indication as other UK and EU countries have different decision-making processes.

We would appreciate it if you could review the Department of Health’s position on differential pricing as a solution for people with PPMS being treated with ocrelizumab? We are convinced that there must be a solution that will allow our patients with PPMS to be appropriately treated under the NHS.

Despite the pharmaceutical company conceding to the NICE target and the drug receiving a European license, it is now the government who is preventing patients receiving appropriate treatment for their MS simply due to a rule, arguably an irrational rule, created by the Department of Health.

We look forward to hearing from you.

Yours sincerely

Concerned NHS HCPs.

CoI: multiple


25 years

I mentioned in a comment yesterday that “MS is unrecognisable to what it was 25 years ago. It is all about early diagnosis and early effective treatment; if you miss out on these it is not good news”. In response, a commentator said “what it really was 25 years ago … and what you remember are going to be different” and “if someone has PPMS …. it’s not”. 

This commentator is wrong on both accounts.

When I started my training as an MSologist working for W. Ian McDonald at Queen Square I recall us having to see MSers with very severe and disabling relapses on a weekly basis. We always had one or two MSers on the wards; admitted to hospital to manage severe relapses, pressure sores, fractures and severe contractures. This rarely happens now. The only patients with relapses that get admitted tend to be patients coming via A&E with their first attack, i.e. their initial presentation. DMTs don’t only reduce relapses they reduce the severity of attacks. I estimate that the use of high-dose steroids to treat relapses has dropped off by over 80%. Most of our RRMSers are being treated to target and are NEDA and relatively stable. We know that prognosis for relapse-onset MS has changed. With DMTs, in particular, high-efficacy DMTs, we have revolutionised the outcome for people with the disease; fewer relapses, milder relapses, less disability, fewer people with SPMS, improved survival, better symptomatic treatments, etc. 

What about PPMS? Firstly, the proportion of people being diagnosed with PPMS has fallen from about 15% of the new, or incident, cases to 5%. Why? This is because neurologists don’t label people as having PPMS so that they can offer them treatment. Even those with a diagnosis of PPMS are being treated in most countries, with either licensed DMTs or off-label treatments such as rituximab. At Barts-MS we have a cohort of our PPMSers on rituximab and an increasing number with active PPMS on off-label cladribine. I also have quite a large number of PPMSers who I still follow who I treated with mitoxantrone. We are also offering an increasing number of PPMSers HSCT. In fact, I saw one of my patients with PPMS, who had HSCT in February this year, in clinic last week. She seems to have done well; i.e. she is stable and says her fatigue levels are much better. And finally, we have ocrelizumab licensed and available to treat early, active, PPMS. Ocrelizumab delays the need for a wheelchair by about 5 years and maintains arm and hand function by about 9 years. Is this not an improvement on what was available 25 years ago? In addition, there is a lot of activity in terms of PPMS trials. We are about to start ORATORIO-HAND, the second ocrelizumab in PPMS trial, and I am aware of at least three other PPMS trials in development. 

Despite the advances in the treatment of PPMS mentioned above we have done many other things. For example, we have improved the recognition and diagnosis of the condition. Also, the symptomatic treatments for PPMS have improved. There are newer antispastic agents (Sativex, intrathecal baclofen, gabapentin), drugs and devices to improve walking (fampridine, functional electric stimulators, botox, better splints), better drugs and devices for bladder and bowel dysfunction, better drugs for pain, better drugs for osteopaenia, and this list goes on. We are improving outcomes by managing MS holistically and this is why the life-expectancy of PPMSers has improved substantially and is now only 3-4 years below what is expected for the general population; 25 years ago life expectancy was about 8 years lower.

So this commentator, who is clearly a cynic, should think a little more deeply and look at the facts before commentating.

Maybe the following quote from Bill Gates’ is apt: “we always overestimate the change that will occur in the next two years and underestimate the change that will occur in the next ten. Don’t let yourself be lulled into inaction”.

So if you have been diagnosed with PPMS in the last few years your outcome is so much better than it was 25 years ago and will continue to improve over the next 25 years. Please remember that innovation is relentless, even if you have PPMS, so never give up hope!

CoI: multiple

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