What does FDA approval mean?
FDA approval is required for Class III medical devices (like heart valves) and treatments for diseases. It is not required for things like fitness trackers and dietary supplements.
Clinical trials are run for treatments like the recent Covid vaccines, and on average take 12 years to complete.
The 3 central stages in clinical trials test whether the treatment is safe(Phase I), best dosage and whether it seems to work(Phase II), and a large randomized study to really test if the treatment seems to work (Phase III).
Correction from the last email: we mistakenly wrote that plastics labelled 3 were recyclable - they are generally not! Thanks to sharp-eyed Darren for spotting this
Thanks also to good friend Semmie for reading through this current mail post and keeping us honest.
I hear the term “FDA Approval” every so often, but never really looked into it. From time to time, you’ll hear that a drug got approved. Or maybe someone was inside trading on FDA news and was going to jail. Either way, I just knew it was a big deal, took a long time, and spelled large differences in outcome for health-related companies. So what is it?
FDA approval is pretty specific, and only required for substantial situations. For medical devices, approval is needed for Class III items: things like pacemakers or implanted prosthetics (versus, for example, class II devices like syringes or contact lenses). It’s also required for disease-treating therapies and drugs (like the covid vaccines, or drugs treating Alzheimer’s disease). But it’s not required for things like dietary supplements or multivitamins, where packaging makes general wellness claims (strengthen your immune system), as long as they don’t make specific disease claims (treats the common cold).
Clinical trials are an important part of the approval process. Generally, the steps are:
Preclinical (sometimes): Prove that it’s safe and effective in animals.
Phase 1: Administer it in healthy humans. This is done for a small number of people (<100 or so), and is done to show safety; you don’t want your drug to hurt people.
Phase 2: Administer it to people who have the disease (or in a vaccine’s case, to people. This can be done in a randomized control trial on a relatively small number of people (up to a few hundred usually). It tries to suss out if the treatment seems to work or not (it’s not worth doing a large-scale study if your treatment isn’t showing ). It’s also where they try to find out correct dosage for the treatment.
Phase 3: Basically, a scaled-up phase 2 trial. Thousands of people are given the treatment and observed for safety and effectiveness. This is usually done with randomized controlled trial, where half the group gets the real treatment and half the group gets a placebo (a procedure where the active ingredient is missing but they think they got it).
After a treatment passes phase 3, the therapy-makers can submit how they ran these tests and what the results were to the FDA, upon which they might get approval. After approval, they’re allowed to start marketing the therapy.
Phase 4: Observation after the fact. Sometimes, long-term effects of a treatment might not show up until years later. The Covid vaccines have only been around for a few months, so there’s no telling what will happen in five years. Even after public consumption, the FDA will have to monitor health reactions and see if it’s still safe.
On average, this process (from research to approval) takes about 12 years. Getting enough people and data for phase 2 and 3 clinical trials takes a really long time, and a lot of money. In a survey of pre-drug costs, the median cost across Therapeutic Area(a statistic of a statistic, sorry) was $59 million. This is obviously quite expensive, and companies do tend to IPO before they can even sell their therapies (check this for a spread of public biotech companies and the stages that they’re in now). It’s quite different from the software world...
On a current note: it was obviously a large and concerted effort to get the Covid vaccines out. There’s a timeline of the Moderna vaccine here, which I enjoyed. It also pretty cool that it took just 25 days to go from sequencing to shipping the first vaccine candidates.
Thanks for reading this far! We’re pretty intrigued about the ramifications of the AlphaFold results from this week. If you’re knowledgeable here, please ping us at davidren.mailbox@gmail.com.
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Some references for the interested reader:
https://www.fda.gov/consumers/consumer-updates/it-really-fda-approved
https://www.fda.gov/drugs/development-approval-process-drugs
https://www.prevention.com/health/a32132101/fda-approved-definition/
https://en.wikipedia.org/wiki/Phases_of_clinical_research
https://www.modernatx.com/modernas-work-potential-vaccine-against-covid-19
https://www.nytimes.com/interactive/2020/science/coronavirus-vaccine-tracker.html
https://www.sofpromed.com/how-much-does-a-clinical-trial-cost/
Nice summary! After 9 years in the pharma commercialization industry, I'd like to add that sometimes Phase II/III occur together for rare diseases since it takes so long and is so hard to enroll all patients. Data releases from P2 and P3 are huge days for pharma companies.