Hey. Back to the “Considerations Regarding Food & Drug Administration Review and Regulation of Drugs for the Treatment of Amyotrophic Lateral Sclerosis”. We need help. It would be fantastic if we had an advocacy group taking the lead here but alas…. let’s move on.
When it comes to drug/ therapy development we are failing. We have two approved drugs to “treat” ALS. One “may” extend life 3 months. One “may” slow down progression (while fleecing everyone involved). There is no question – ALS is a difficult disease to target. However, we are failing before we begin. We are currently using archaic protocols in drug development. The measure of success will never be met because we are using a yardstick that does not correspond to the disease. If we are ever going to make a dent in the utter havoc this disease wreaks on everyone it touches, we MUST change the way we approach it.
We have one month left to make comments. PLEASE comment on the guidance for industry the FDA has proposed. I am including some suggestions below to get your thoughts rolling. Feel free to cut and paste any combination, should you agree with them. I am also including some complete comments. Change them up, use completely different ones. use them exactly. Whatever works for you. PLEASE speak up, share your thoughts, be heard.
The FDA must encourage innovative clinical trial designs tailored to the population living with the disease and reflective of the heterogeneous nature of its presentation.
This disease demands urgency. Use clinical trial designs which reflect the same.
- Push for the use of trial designs that realistically represent the number of people living with this disease at one time.
- Push for mobile travel sites in to allow a larger number of participants.
- Push for remote data collection to make it easier on families to participate.
- Use historical controls from the PROACT data base vs. placebos.
- Widen participant eligibility criteria.
- Choose an endpoint other than death... a predesignated drop in FVC or specific point loss of ALSFRS.
- Consider taking guidance from people living with this disease.
Please do not accept this guidance as is or we will be having the same conversation in another 50 years.
This guidance does not reflect the advances in technology, science, or data collection we have made this last century. We must encourage the industry to embrace innovate trial design that correlates to the number of people living with the disease at a given time. Use historical data, mobile trial sites and remote data collection. Change eligibility criteria so more people can participate without compromising quality of life interventions that currently exist like the feeding tube which allows people to maintain hydration and minimize the thickening of secretions. We already ask too much of these families - do NOT ask them to give up the few things we can offer.
This proposed guidance will do nothing to accelerate the development of viable therapies to treat ALS. The status quo is NOT working and this guidance IS the status quo. We need the FDA to encourage and support innovative trial designs that make sense with a rare and terminal disease with rapid progression. Widen the base of participant eligibility, use mobile trial sites and remote data collection to include people who do not live near trial sites. Use historical controls. encourage accelerated approval. We must change the way this disease is approached because what we have done the past 50 years, what we are doing RIGHT NOW, is not working.
The guidance presented is antiquated, out of date, and demonstrates zero use of updated technology and data collection methods. This is a rare and fatal disease with no significant therapy. Trials used in drug development must reflect the number of people living with this disease at one time - the duration of the disease - the wide geographic locale of those diagnosed. There should be mobile trial sites and remote data collection, the use of historical data vs. placebo arms, eligibility criteria reflective of a savagely progressive disease. Please do not allow this blatant disrespect and disservice to the people from whom we already ask too much.
Click HERE to submit your comments.
Click HERE to submit your comments.
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