Drug Approval Process

robertbanking

Hello you wonderful and intelligent people that make up this forum. I sincerely hope you are doing well and having a wonderful weekend. 

I recently have been looking at pharmaceutical company stocks, such as Crispr Therapeutics. I note that it recently had a approval for a breakthrough gene editing therapy for Sickle Cell Disease. I am kindly wondered please if anyone knows any books or articles i can read up about the drug approval process please, as i find this really interesting? I note for Pharmaceutical type stocks you would require some special knowledge to evaluation them, so interested in learning further. If anyone kindly had any thoughts on this i would be forever grateful and thankful it would mean the world to me. 

Sending you lots of good wishes and i very much hope you continue to have a wonderful life. Very best wishes to you, enjoy the rest of your weekend. All the very best. 

elsien

Similarish previous thread.
https://forums.moneysavingexpert.com/discussion/6497484/outlook-therapeutics-fda-rejection#latest

As you are already aware of the FDA, you’re probably also aware  that the drug approval process will differ between countries. So no one size fits all answer.

You could probably also dispense with the rather flowery but identical first, and last paragraphs in all your questions. 

xylophone

https://www.biopharma-reporter.com/Article/2024/01/04/casgevy-approval-unlikely-to-be-followed-up-by-another-crispr-drug-in-near-future


https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/evaluation-medicines-step-step

https://pubmed.ncbi.nlm.nih.gov/20590071/

https://www.ataxia.org/from-research-to-reality-the-5-steps-in-drug-development/

masonic

Another good resource for this, covering preclinical development and the clinical programme is the ICH website https://www.ich.org/page/ich-guidelines

As well as the approval process differing depending on the regulatory authority a pharma or biotech company applies to, it will also differ depending on the disease indication and type of treatment. The classic example is the ICH S9 route to delivering treatments for advanced cancers, where the guidelines seek to accelerate the process and remove the need for certain safety studies to be conducted, as the clinical need outweighs these risks.

It's a huge area, which changes over time, and professionals will build their knowledge over years of 'on the job' learning.

Clive_Woody

Lots of good advice already, but think carefully about investing in single stocks rather than funds. 

Having worked in drug development for more than 24 years I would struggle to pick a winner when it comes to picking shares in a particular company. There are multiple approval pathways and rare and orphan disease get special treatment. Different countries/regions have different approval processes and timelines. Once approved there are also reimbursement negotiations that can drag on, then you need to consider the risk of side effects that didn't show up in clinical trials, competitors already on the market and other competitors in development launching soon, patent life, long term efficacy, etc...then there's also pipeline considerations for the company or are they a single product company that could well be acquired.