what is a clinical trial
Dr. Bailey does an excellent job of summarizing the steps that are followed to bring new drugs to market, which follow a very predictable route.
| Phase | Target | Duration |
|---|---|---|
| Pre-clinical Trial | animals | on average 31 months |
| Phase 1 | healthy | several months |
| Phase 2 | carefully chosen | 1-2 years |
| Phase 3 | 1-3,000 | 3-5 years |
| Phase 4 | small general public | 1.5 years |
| FDA Approval | all general public |
Pre-clinical trial on animals to determine toxic dose and safe dose for humans, and look at side affects that may affect humans.
Phase 1 Clinical trial on small group of humans (20-80). Healthy individuals, who are paid for their participation.
Phase 2 Clinical trial on target patients to identify side affects and efficacy or how well the drug works (100-300 people). Participants are carefully chosen.
Phase 3 Clinical trial to measure effectiveness and monitor side affects (1000-3000 people). Some side affects take time to reveal themselves. Randomized, double-blind, placebo.
Phase 4 Public Trial – regulatory approval to use the drug in the general population under varied conditions, drug interactions and discover untoward affects that might have been missed in earlier trials. Continue to monitor side affects. Many drugs have shown harmful effects only during phase 4 trials and have subsequently been banned from use.
Informed Consent
Following the horrendous government practices during world war two, most countries signed the Nuremberg agreement to make participation an individual choice and after providing information about the risks of the procedure. Technically you can not be involved in a clinic trial without signing first an Informed Consent agreement.
Fast tracking
It can take 10-15 years to get a new drug to market and many will fail along the way.
FDA has a provision for Accelerated Approval before effectiveness is understood and allows for the drug to be used for new drugs for serious and life threatening illnesses that lack satisfactory treatments. In as early as 2.5 years. The drugs are continuously monitored for effectiveness and removed if they do not appear effective and confirm the initial results. This was used for HIV drugs for example.
Emergency Use Authorization – EUA
Special circumstances exist for approval and this was obtained by Pfizer for use in only 8 months.
It is very important to understand that Clinical Trials are still running while the product has already been purchased by government and is being given to the general public. The study that is currently running since April 2020 but is not actively recruiting patients. It is a phase one, two, and three study. It is just under 44,000 patients and is looking to find the correct dose and efficacy of the product in healthy people. It’s estimated to finish in January 2023 so we may not see any long-term safety data until that date.
Legal Liability
One of the reasons that clinical trials are so expensive is the pharmaceutical company is legally liable for injury and compensation during the trial and even continues after the drug goes to market.
There are exceptions to this rule and the govt has invoked the Public Readiness and Emergency Preparedness Act on Feb 4, 2020. The 2005 law empowers the HHS secretary to provide legal protection to companies making or distributing medical supplies, such as vaccines and treatments, unless there is “wilful misconduct” by the pharmaceutical company. This protection lasts until 2024.
https://www.youtube.com/watch?v=7h7mLhjYvF8
technical notes
https://www.cdc.gov/nchs/nvss/vsrr/covid19/excess_deaths.htm
https://www.cdc.gov/flu/symptoms/flu-vs-covid19.htm
https://www.cdc.gov/flu/about/burden/preliminary-in-season-estimates.htm
https://www.fda.gov/media/143557/download
https://vaers.hhs.gov/data.html
https://www.clinicaltrials.gov/
From FDA to MHRA: are drug regulators for hire? Patients and doctors expect drug regulators to provide an unbiased, rigorous assessment of investigational medicines before they hit the market. But do they have sufficient independence from the companies they are meant to regulate? Maryanne Demasi investigates https://www.bmj.com/content/377/bmj.o1538.long