Developing a new medicine begins with understanding the disease or condition as thoroughly as possible. Basic research provides clues about how to treat diseases and potential ways to target the symptoms or underlying causes. Armed with an idea, researchers work to understand biological targets for a potential medicine. A “drug target” can be a protein, RNA, DNA or other molecule that is somehow involved in the disease. Researchers conduct studies in cells, tissues and animal models to determine whether the target can be influenced by a medicine. They then look for a lead compound — a promising molecule that could influence the target and, potentially, become a medicine. This is the first step in the discovery and development process, which spans from initial research to the delivery of life-saving or life-enhancing new medicine1. It is worth mentioning that for every 10,000 molecules identified only one will make it all the way to being approved by the regulatory authority and prescribed for patients by a physician. There are a number of phases in that journey2.
In order to address further issues of uncertainty surrounding the benefits and risks of a new treatment, it needs to be tested within the framework of a clinical trial. Prior to embarking upon the commonly three-stage clinical trial process itself, a company or academia must submit a protocol for a regulatory assessment, which details what is going to take place in the trial. The protocol is evaluated by an ethics committee, which consists of independent experts and representatives of the lay public.
Clinical trial starts with a small trial in healthy volunteers (phase 1) before going in to increasingly large trial in phase 2 and phase 3 which are often multi-center trials involving sometimes thousands of patients and conducted in multiple countries and regions and having the objective to find out if the new medicine is safe and effective. All trials need to be registered in the publicly available database before start of the trial2.
Clinical trials are the most time- and resource-intensive part of the research and development process for a new medicine, and biopharmaceutical manufacturers support and conduct the majority of this important work. Yet, without clinical trials, new medicines could not be approved and–most importantly–made available to patients who need them. From drug discovery through regulatory approval is a long journey.
Developing a new medicine takes at least 10 years on average and costs an average of $2.6 billion* while less than 12% of the candidate medicines that make it into Phase 1 clinical trials will be approved by the FDA. Since 2000, PhRMA member companies have invested over $800 billion in the research and development of new treatments and cures in the US, including an estimated $71.4 billion in 2017 alone3.