I am preparing myself to let my patients with PPMS down. Are you? #PatientSpeak, #MSBlog #Ocrelizumab
I saw one of my patients the other day who has PPMS. His disability is worsening and clearly wants something done about it. He was very excited about the news that the FDA had licensed ocrelizumab for PPMS and wanted to know when ocrelizumab would be available under the NHS. I explained to him that ocrelizumab had yet to be licensed by the EMA and as the EMA tends to be more conservative than the FDA it wasn't certain by any means that it would be licensed in Europe for PPMS. Even if ocrelizumab is licensed for PPMS, I suspect the EMA won't give it as broad a label as the FDA has done and may limit it to a particular population of pwPPMS. If this happens I informed this patient he may fall outside of the licensed indication, for example his PPMS may not be active enough.
In addition, if and when the EMA licenses ocrelizumab for PPMS then NICE has to green-light it for use in the NHS. The latter may be a problem, particularly if ocrelizumab is priced to be cost-effective for RRMS. At the RRMS price NICE will need to compare it to best supportive care (no DMT) for PPMS and it is unlikely to be cost-effective using this comparator. I have been arguing for sometime now that this may be the time for Pharma to explore differential pricing and charge the NHS less when ocrelizumab is prescribed for PPMS.
When I told this patient about the various hurdles that will need to be overcome for me to prescribe ocrelizumab for him he was very disappointed, so I asked him to put something down in words to start a conversation with other pwPPMS who live in Europe.
Please note that Mark has given me permission to publish his name. In fact Mark sits on the Barts-MS Advisory Group and wrote a blog post on this in December 2015.
A patient-with-PPMS's Perspective on Ocrelizumab
"Some 4 months after I was diagnosed with PPMS in May 2008, I remember speaking to a man who had had MS for a number of years and he said that I would be one of the lucky ones – there would be treatment for PPMS in the next ten years or so. I have always remembered this but of course with less and less conviction as the years have gone by. Now I think I will be just another of the countless unlucky ones, there will be no treatment for me.
So when Ocrelizumab was announced as a possible treatment for PPMS I was overjoyed and thought, you know, maybe he was indeed correct. However, as ever with this disease any joy is always mixed with disappointment and so it was when I discovered that I was too old to be considered for the trial – I was 57. This was a bitter disappointment as I do not consider myself to be old, let alone too old. Indeed I have taken part in 3 trials since being diagnosed, I am generally fit, look after myself, lost weight, go to the physio twice weekly and maintain a good positive outlook. I do “my bit” but, there you are, I am too old.
Following the Ocrelizumab trials I like others with PPMS are very keen for the drug to licensed as currently it is our only hope. I understand that whilst it is not particularly effective where MS has taken a hold - eg on the lower limbs, it is effective on the upper limbs. For this reason alone it should be prescribed as our upper limbs must be saved they act as our legs by allowing us to use a stick and maintain some independence by using mobility scooters and other aids. Once our arms are as stiff as our legs we are effectively quadriplegic - if that can be avoided or at least delayed then surely we should have the drug. After all if there was a cancer treatment that whist not effective on the primary tumour but reduced or delayed the spread of secondary tumours, I am sure that would be licensed."
Mark Harrington, May 2017, London
Labels: #MSerSpeak, #PatientSpeak, ocrelizumab, PPMS