American patients get access to new medicines faster than anyone else in the world. It’s the result of the most complex, scientifically innovative ecosystem in the world. This process brings together government, academia and industry in a continuous cycle of discovery, development and delivery of safe and effective medicines to American patients.
Why it matters: America's innovation ecosystem doesn't just produce innovative medicines that save lives. It produces hope for patients and families, delivered through an intricate process of drug discovery and development.
The big picture, in motion: Bringing a new medicine to market takes 10 to 15 years of research and development and costs $2.6 billion, on average. Medicines go through distinct stages to reach patients, each one a chapter in a much larger story of American scientific ingenuity. PhRMA’s new drug development lifecycle module offers an interactive, stage-by-stage look at the time and resource intensity it takes for this innovation.
- Basic research: It starts with scientists asking fundamental questions about how diseases interact with the body and where we might intervene.
- Discovery: From there, researchers may screen thousands of compounds, searching for the candidates that could become tomorrow's treatments.
- Pre-clinical research: Every promising candidate is rigorously tested for safety before it is ever tested in a human.
- Clinical trials: The candidate moves through Phase 1 to test safety and dosage, Phase 2 to further explore efficacy and side effects and finally Phase 3 for large-scale efficacy and safety assessment.
- FDA review & approval: Companies submit the full body of evidence to FDA reviewers who conduct a rigorous evaluation of safety, efficacy and manufacturing quality.
- Post-approval safety monitoring: The work doesn't stop at approval. Sponsors are subject to detailed safety monitoring requirements helping to ensure patient safety.
- Post-approval research: And the science keeps going, with researchers studying new uses and formulations for the drug to help more patients.
By the numbers:
- Only about 1 in 10 medicine candidates that enter clinical trials are ultimately approved by the FDA, a reminder of how much scientific persistence and risk-tolerance the system demands.
- Over half of new uses for cardiovascular medicines were discovered after the initial FDA approval, proof that innovation doesn't end at initial product launch. It continues forward.
What makes this an American story: This ecosystem exists because of a foundation no other country can match. It’s built on strong intellectual property protections, a research infrastructure that spans academia and industry, a gold-standard review and regulatory system that is respected worldwide, and a market that rewards the enormous risk of discovery and development.
The patient advantage: Our unique ecosystem has built a world-leading medicine development ecosystem, with every stage designed to benefit patients.
- Clinical trial participants often gain early access to promising treatments before they’re available to anyone else. For patients with rare diseases or conditions that haven’t responded to existing therapies, that early access can be life changing. The data they help generate can transform care for thousands, even millions, of future patients.
- The robust post-approval safety reporting requirements help to ensure patient safety.
- Post-approval research continues to identify new information about treatments for patients that matter in everyday life: more convenient dosing, clearer understanding of side effects, or a new indication that opens up treatment options for more patient populations, like children.
The bottom line: We are living in an era of scientific breakthroughs – medicines that are curing cancers, reversing diseases once thought untreatable and giving patients years of life they weren’t supposed to have. None of that happens without the ecosystem behind it: Scientists studying diseases, researchers running trials and regulators reviewing drug safety, efficacy and quality.
Biopharmaceutical manufacturers take tremendous risks developing and manufacturing innovative medicines, often investing billions of dollars with no guarantee that a single compound will ever reach a patient. It’s why American leadership matters – so the next breakthrough goes from a promising idea to a patient’s hands.
Explore the interactive module to trace a medicine's full journey from the lab to the medicine cabinet.
Drew Voytal
Drew Voytal, MPA is a Senior Director of public affairs at PhRMA focusing on advocacy and policy communications on key issues, including 340B. His career in health care advocacy, policy and public affairs spans more than a decade in Washington, DC where he has built relationships with key government and industry partners to advance public policy priorities. Drew has personally experienced the benefits of biopharmaceutical research and innovation and is passionate about telling the story of this vital industry.
Drew Voytal