How Are Medicines Discovered?

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 medicine.


The Biopharmaceutical Research & Development Process

From drug discovery through FDA approval, developing a new medicine takes at least 10 years on average and costs an average of $2.6 billion.* Less than 12% of the candidate medicines that make it into Phase 1 clinical trials will be approved by the FDA.


Clinical Trial Diversity

Increasing diverse enrollment in clinical trials is a critical step to improving access to medicine development and the biopharmaceutical industry has taken action to enhance clinical trial diversity. The demographics of a clinical trial should, to the extent possible, reflect the intended treatment population. In addition to individual company efforts, PhRMA and its members have voluntarily developed and adopted industry wide principles on clinical trial diversity. These principles, which took effect in April of 2021, focus on four main areas: building trust and acknowledging the historic mistrust of clinical trials within Black and Brown communities, reducing barriers to clinical trial access, using real-world data to enhance information on diverse populations beyond product approval and enhancing information about diversity and inclusion in clinical trial participation. Yet despite progress and the steps taken by the FDA and the biopharmaceutical industry, clinical trials do not always reflect the demographics of the intended treatment populations.

Stakeholders must continue to come together to address systemic barriers to participation if we are going to make further progress. That’s why PhRMA, in July of 2022, funded a grant to launch Equitable Breakthroughs in Medicine Development. The initiative is a comprehensive effort to increase diversity in clinical trials and address systemic barriers to participation by communities of color. This effort seeks to help underrepresented patients be more involved in the research and development of potential life-saving medical treatments.


Clinical Trial Data Transparency

Clinical trial data transparency aims to improve access to clinical trial research and results in order to inform medical decision-making, advance scientific discovery and accelerate the development of new treatments to benefit patients. The biopharmaceutical industry has long-established commitments to facilitate appropriate transparency throughout the lifecycle of a clinical trial including registration, results reporting, and data sharing, and the biopharmaceutical industry is at the forefront of initiatives to improve access to clinical trial data in order to advance medical research.

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