There is something weird with clinical trials on neurodegenerative diseases, they seem to always fail. For example there were ~50 completed interventional phase III ALS clinical trials and ~170 completed interventional phase III Alzheimer clinical trials. On overall there were nearly 2,500 interventional clinical trials on Alzheimer disease. For diabetes it is even worth: ~ 1,500 completed interventional phase III studies!
To understand those failures, there are some aspects that are important to realize:
- Chronic diseases, and specially neurodegenerative diseases will certainly not show obvious improvement during the 6 months duration of good clinical studies. Such clinical trials must last years to show some improvements, and indeed today's short term clininal trials are designed to fail.
- To minimize costs, phase I or phase II drugs are not needed if there were already done, as they are not specific to any disease.
- Industry and investors will never try non patentable drugs. Yet there are many non patentable drugs that will never be tested by the industry. For example it is obvious that many diabetes drugs could be tried in ALS or other neurodegenerative diseases. One of the too rare cases is University of Florida studying Metformin in NCT04220021.
- Tools are needed to find non patentable drugs that could potentially be repurposed. Example of such drugs are repurposed drugs with expired patents (in particular patents by universities), abandoned drugs (at least 800 drugs are abandonned), drugs which were described in academic prior art but were never tried (ten of thousands), or natural products such as TUDCA. It's even better if those drugs went through a successful phase I and II trial. ~35,000 phase II studies where completed, it covers a lot of drugs.
- The industry look down on academic research which they feel as being of poor quality. Moreover, academic proposals are ignored due to risk of prior art.
- Most drugs used in neurodegenerative diseases have strong side effects such as they can't be seriously considered for long term use. This lead to reluctance to prescribe by primary care physicians. Drugs for chronic diseases should only tested in matched combinations.
- Clinical trials should rely on the medical professionals who will interact with patients on a day to day basis.
- Clinical trials should use Healey platform-like methodologies to make sure that no patient is left only with placebo.
- Hosted software should manage the double blinded procurement of drugs.
We will propose here a tailored low cost Clinical trial management system that fits those requirements.
- The idea would be to let primary care practitioner (GP in UK parlance) to take part in the clinical trial. A MD treats their own patients. Doctors are carefully indentified, they agree to follow the clinical trial guidelines.
- The clinical trial would typically last several years.
- The doubled blinded study would use a volunteer organization to blind drugs and send it to medical doctors who would administrate it their patients.
- No patient should be let only with a placebo, a Healey platform design-like is mandated.
- The software solution take care of regulations and comply with them.
- The software solution is operated under the control of an ad hoc organization with clear roles.
You can mail us at contact@padiracinnovation.org