The Food and Drug Administration (FDA) Drug Approval process is long and involved. The public (as well as the president) knows that. On Wednesday, the president signed into law the “Right to Try” bill – allowing patients with life-threatening diseases to access experimental drugs. Patients who have lost hope and exhausted all options will now be able to receive treatments that have gone through Phase 1 approval.
This sounds like a great idea on the surface, right? Patients will have another option when they feel like they have tried everything. Loved ones may be able to squeeze a few more months or years out of life. Remaining days could have reduced suffering and better quality. That all sounds amazing! But before you sign up for a “right to try” make sure you read below.
The FDA drug approval process consists of 3 phases of clinical studies:
Phase 1: Clinical studies involve only 20-100 people and last only a few months. The purpose of these studies is to evaluate safety and proper dosage. 70% of drugs make it past this phase.
Phase 2: Clinical studies involve several hundred people and last up to 2 years. The purpose of these studies is to evaluate efficacy and side effects. Only 33% of drugs make it past this phase.
Phase 3: Clinical studies involve up to 3000 people and last up to 4 years. The purpose of these studies is to further evaluate efficacy and side effects. Only 25-30% of drugs make it past this phase. Also, according to the FDA’s website, “Phase 3 studies provide most of the safety data. In previous studies, it is possible that less common side effects might have gone undetected. Because these studies are larger and longer in duration, the results are more likely to show long-term or rare side effects”.
Based on this information I would argue that Phases 2 and 3 are the most critical phases of clinical trials. Of the 70% of drugs that get approved through Phase 1, only 10% go to market. Does this give you confidence in a drug that has only passed Phase 1 trials? For me…NO.
Back in April, we found out that a drug called Nuplazid (used to treat Parkinson’s disease) was reported in 700 deaths. This drug had gone through an expedited approval process after family members of patients with Parkinson’s pleaded with the FDA for help. The drug went through 3 unsuccessful clinical studies and was approved as a “breakthrough therapy” based on a single 6-week study of only 199 people (half were given the drug and half were given placebo). 10% of patients experienced serious adverse events. Death was not one of them.
The purpose of this blog post is not to deter anyone from enrolling in a Right to Try program. I personally believe that this is a viable option for families when all hope is lost. Right to try gives people access to experimental drugs who are more than likely too sick to get accepted for enrollment in clinical trials. However, I firmly believe in giving the general public the FULL STORY. It is important to know that if you take experimental drugs, that there are risks – sometimes very significant ones.
If you are interested in enrolling in a Right to Try program or learning more, please visit www.righttotry.org. Anyone who is considering the program should review Phase 1 clinical data here and understand any serious adverse reactions that were observed during the trial for the drug of interest.
Do your research. Make informed decisions. And as always…