Our case for ending placebo controls in clinical trials of Nusinersen to allow for expanded access.

Why I’m petitioning for access to Nusinersen for all terminally ill children with Spinal Muscular Atrophy.

Unfortunately on the UK Parliamentary Petitions website I was restricted by word limits from explaining the case I am making to end the current placebo controls within clinical trials of Nusinersen, which would in turn enable expanded access for all suffering from Spinal Muscular Atrophy. My points for making this case are as follows:

  1. Nusinersen has not had a single drug related adverse effect in over 4 years of clinical trials.
  2. In type 1 cases of SMA, 80% of children have a life expectancy of less than 2 years, these children can’t wait until 2018 for treatment to become commercially available.
  3. A third of trial participants will receive placebo. These placebo controls are used in trials that are entirely made up of type 1 SMA. These children don’t deserve the distress of this during their short lives for a placebo and the emotional distress it would put their families through – to mistakingly think their child might get better and all the hospital trips etc, just for nothing. And to allow children to die alongside children gaining strength just to demonstrate how good the drug is – these are children, not lab rats!
  4. The earlier after diagnosis that Nusinersen treatment is started the more abilities are retained and regained. As an infant a child will see a significant gain in motor function treated early enough, an older child will see minimal gain in motor function. This is due to the nature of the way in which motor neurone cells degenerate, it appears they go into a “sleeping” state before they die completely. Not all damage to motor neurone cells can be undone with an increase in SMN protein levels, but the earlier in the process of damage occurring the better.
  5. Without placebo controls in current trials expanded access for all could be applied for and research could still continue.
  6. With expanded access to Nusinersen exploitative con artists would not be able to take advantage of the desperate situation in which families affected find themselves by charging extortionate amounts of money for bogus treatments.
  7. Many families affected by SMA give up everything they have in their desperation to raise funds to travel to other countries to gain treatment through clinical trials, some do this and still don’t access trial treatments and can be left grieving having given up everything in their pursuit of treatment.
  8. Surely saving lives is more important than a bureaucratic process? Isn’t saving/improving lives the whole motive behind developing new medicines in the first place? When did we lose sight of this and place more importance on tick boxes than life?
  9. Many parents of and SMA sufferers just want a chance. Many in the SMA community have been waiting 20 years+ and many of these years have been spent with the promise the treatment is just 3-5 years away, they have been patient and now they want results.
  10. Many pharmaceutical companies don’t want to carry out placebo controlled trials, but these are insisted upon by regulatory authorities.
  11. Research data of the natural history of disease progression in SMA is available. So we already know and have researched/documented how the condition progresses, surely this already tells us what will happen in the control groups.
  12. SMA is the Number 1 genetic killer of infants.
  13. Every day 1 child dies because of SMA.
  14. Sma affects hundreds of thousands of people worldwide – people with SMA and their families. In the UK alone there are between 2,000-2,500 people with SMA.
  15. What we are asking for is not unprecedented. Nusinersen has already undergone phase 1 and phase 2 trials and is currently in phase 3 trials. Kanuma was approved in December 2015 for a terminal condition with only 1 phase of trials, no placebo controls (natural history data was used) and small participant numbers in trials. Xuriden which treats a rare metabolic disorder affecting approximately 20 (yes 20) people worldwide was approved for use September 2015 with 1 phase of trials, no placebos. Tagrisso was approved for use in November 2015 as a treatment for lung cancer which 1 phase of trials and no placebo use. And other examples exist!
  16. For those of you economically minded. Yes, as an orphan drug, Nusinersen may cost a great deal when it is released, however SMA costs a great deal in care costs. As the symptoms worsen care needs increase. The many different consultants (neurology, respiratory, paediatric, community paediatric, ENT, palliative, gastrology and orthopedic), doctors (juniors and GPs), nurses (and specialist nurses), physios (paediatric and chest), OTs, feeding specialists, speech and language specialists, carers, educational assistants all run up a huge bill as their involvement starts and increases. As well as this there is the multitude of expensive equipment that needs to be supplied and many hospital stays. As the condition worsens all these costs increase, so perhaps stopping it or reversing it in it’s tracks then sounds much more cost effective?
  17. SMA makes a huge impact on families. As children become trapped inside their bodies and parents watch completely helpless. Often 1 parent stops working in order to carry out the full time care needs of a child with SMA. It affects the family psychologically, emotional, financially, in terms of their health and relationships and dominates their life and time.
  18. Death is permanent. Once we lose a precious life to SMA, we can’t get them back again once treatment becomes available and many hearts remain forever broken.
  19. In most recently published data, 2 type 1 children are taking supported steps for the first time. They are walking! This is entirely unprecedented in the entire history of SMA. Type 1 children don’t sit unsupported, they don’t crawl, they don’t stand and they definitely don’t walk! This is an enormous gain in strength in children that only lose strength. How else can this be explained if not proof of drug efficacy!?

Please read these considerations carefully to see if you agree with the case we are making to end the use of placebo controls in current trials which would enable the compassionate use of Nusinersen for all with SMA. If you would like to sign the petition, please visit https://petition.parliament.uk/petitions/121253 , it takes literally 1 minute of your time and it could make all the difference. Many thanks for your continued support.

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