Thank you: EMA MS Workshop

Are you for patient choice? The EMA is not; what should we do about it? #MSBlog #MSResearch #ClinicSpeak

"I would like to thank you all for completing the survey I posted last week to get some data for my presentation to the EMA on a MSers perspective in relation to the staggered 2-step approach. Whilst preparing my talk, which I did at the last minute, I realised that I made a mistake in accepting an invitation from the EMSP (European Multiple Sclerosis Platform) to speak on behalf of MSers. The EMSP should have taken the opportunity to have an MSer speak. I suspect the EMA did not agree - they don't like being reminded that their decisions impact on peoples' lives - hence my invitation."

"I tried to focus my talk on the early hidden disabilities that MSers suffer from, early in the course of  the disease, that indicates how serious MS is as a disease; i.e. employment, quality of life, anxiety, depression, fatigue, divorce, suicide, etc. If we apply  the staggered 2-stage approach to treatment, using the EDSS, as our yardstick by the time MSers start becoming disabled we have missed the boat. People with MS need the option of early highly-effective treatments and it should be up to them and their neurologists to decide. Our survey results are clear on this."



"More worrying is drug development using the staggered -2-step approach. The EMA want Pharma to develop drugs in series; i.e first do a trial in MSers who are more advanced and have or are failing current DMTs. Once your drug has been shown to be effective and safe in this population to only then start a trial in MSers who are naive to treatment. The serial, rather than parallel, approach will add an extra 4-5 years onto the time it takes to develop DMTs. This is unfair to MSers and Pharma. Why delay access to an effective treatment? The 4-5 year delay reduces the patent life of the drug and hence it will push up the price of drugs substantially. Pharma need to make money; if the can't see a finish line why take the risk?"

"I was criticised by one of my colleagues for using case scenarios to make a point. He said I sounded like a surgeon describing my last surgical success. It is difficult to give a patient perspective without giving real-life examples. The two cases I presented illustrate what could happen if you delay access to highly-effective therapies; you run the risk of acquiring fixed disability and never catch-up to those who access the treatment from the outset. Unfortunately, our predictors of who is going to do well or poorly on the staged approach are to unreliable to use clinically. The point I was making is that it is up to you as people with the disease to discuss  things with your neurologist, make and informed decision and choose one or other option. It is horses for courses."



CoI: multiple

Labels: , , ,