• Research Topics

  Double blinded clinical trials for ALS patients

  Posted by Igor on June 5, 2020 at 11:42 am

  Disclaimer:

  1. All ideas below are only my opinion
  2. All information was taken from public Internet. There is not any spoiler here
  3. I can be mistaken as any ordinary people
  4. English is my second language. So, be patient if you notice some mistakes

  I analyzed quiet a lot of clinical trials on clinicaltrials.gov and found such restriction:

  • Patient should have ALS diagnose up to 12 or 24 months, not more
  • No feeding tube, no breath ventilation
  • No usually radicawa or edavarone treatment
  • Placebo group in some phase 2 trials and in all phase 3 trials ( I think for FDA)

  Companies offer patients win-win scheme, but it is not for ALS patients.

  Trials usually go on up to 6 or 8 months. It is a life lottery for ALS patients who has fast going degradation or live with ALS more than 12 months.
  I think companies and FDA should redesign double blinded placebo tests for ALS patient. They should find the way how to check drug or cell therapy efficiency using equipment, not just wait time and see result

  So I’ll prefer not blinded phase 1 and phase 2, but there is not a lot of them.
  I saw a good news about 2 Bills on alsnewtoday, but how much time will it take to sign them and FDA starts accepting drugs and therapies. I think a year as a minimum and have a risk to meet very high price of drugs or therapies.

  For example:
  “uniQure introduced Glybera in 2012, it had a price tag of $1.2 million per patient”
  Gilinea – 3000 Euro for 3 months package

  We have to reduce such risk and I’ll come to this idea later.

  Igor replied 3 years, 10 months ago 0 Member · 0 Replies
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