Research and Development Policy Framework
Breakthrough science and robust biopharmaceutical R&D are revolutionizing patient care. Advances in genomics and immunotherapy are enabling personalized treatments and harnessing the power of the immune system to fight diseases like cancer and rare conditions.
Over the past decade, PhRMA member companies have invested over $900 billion in developing new treatments and cures, yielding groundbreaking results. The biopharmaceutical sector is the most R&D-intensive industry in the U.S., investing six times more in research than other manufacturing sectors.
This progress is transforming how we diagnose, treat, and cure diseases, offering hope for conditions once thought untreatable. With more than 8,000 medicines in clinical development, the future of medicine is brighter than ever.
Report
A New Era of Biopharmaceutical Innovation
Report
2025 PhRMA Annual Membership Survey
Innovation
Progress toward New Medicines and Vaccines
The biopharmaceutical industry utilizes its scientific and industrial expertise to build on and advance basic science research into safe and effective treatments and vaccines that can be made available to patients. The biopharmaceutical industry is uniquely positioned to take on the necessary risk to advance research into safe and effective treatments. For example:
- On average, it takes 10-15 years and costs $2.6 billion to develop one new medicine, including the cost of the many failures.
- Only 12% of new molecular entities that enter clinical trials eventually receive U.S. Food and Drug Administration (FDA) approval.
- Approximately 7,000 rare diseases exist today yet only 5% have an available treatment.
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.
Related Resources
Medicines in Development
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Accelerated approval Bringing patients access to needed medicines
Accelerated Approval: Bringing Patients Access to Needed Medicines
The accelerated approval pathway, formally established by U.S. Food and Drug Administration (FDA) regulations in 1992 and later codified in statute in 1997, enables expedited access to medicines that address an unmet medical need for serious and life-threatening diseases and conditions, while preserving FDA’s high standards for safety and effectiveness. This pathway has provided timely access to treatments for HIV/AIDS, cancers and rare diseases, leading to better health outcomes for millions of patients.
Learn more about the accelerated approval pathway in this fact sheet from PhRMA.
Accelerated approval: Bringing patients access to needed medicines image
Access to Medicines
Fact Sheet
Development of new medicines relies on private sector expertise and investment
America’s biopharmaceutical companies are at the heart of a research and development (R&D) ecosystem that develops more innovative medicines than any other country in the world. Critical to this ecosystem is the working relationship between industry and government agencies like the National Institutes of Health (NIH), academic medical centers and community-based research sites to further the translation of basic research into important medical products.
art of scientist looking through microscope
Resource
Biopharmaceutical Innovation in Fighting HIV/AIDS
The human immunodeficiency virus (HIV), the virus that causes acquired immune deficiency syndrome (AIDS), was first identified in the United States more than 40 years ago. Since then, expanded prevention and treatment options have helped patients by bringing down death rates, increasing adherence, improving the quality of life for patients and paving the way for future research focused on preventing the disease.
Biopharmaceutical Innovation in Fighting HIV/AIDS image
Disease
Fact Sheet
New report: Post-approval innovation delivers substantial patient benefits
Post-approval R&D is the continued investigation by biopharmaceutical companies, often through lengthy clinical trials, to find new uses and improvements for U.S. Food and Drug Administration (FDA)-approved medicines to better meet patient needs. Recently, there has been growing discussion among IP critics aimed at undermining the importance of post-approval R&D. Yet, despite critic’s dismissive claims, post-approval R&D is critical to advancing life-saving treatments and improving patients’ lives for the better.
In fact, a new report illustrates how post-approval R&D is essential to not only delivering substantial health benefits to patients but economic benefits to society at large, through:
- Expanding uses of existing medicines and improving existing formulations and dosage forms. Post-approval R&D increases treatment options for patients by demonstrating, for example, that an existing medicine can treat a different disease or stage of disease, a new dosage form is safe and effective or new formulations are safe and effective for children.
- Reducing health and economic burdens by advancing treatment options that are convenient and aligned with patients’ needs. For example, new dosage forms or formulations often provide greater convenience to patients by reducing treatment complexity, easing administration or reducing side-effects. These benefits in turn improve treatment adherence and disease management while reducing costly disease complications, hospitalizations and boosting productivity in the workplace.
- Increasing therapeutic options and driving competition. Payers leverage choice in the marketplace to negotiate rebates and discounts off the prices of medicines which can lower costs by as much as half. Post-approval R&D to develop competitive alternatives to meet a wide range of patient needs contributes to optionality and enhancing brand-to-brand competition and savings to the health care system.
And as we dig deeper, the case for post-approval R&D gets stronger—particularly when you consider the many therapeutic areas that rely on it to advance new treatments.
For instance, a study that reviewed 155 oncology medicines first approved between 2000 and 2021, found that 9 out of 10 FDA-approved cancer medicines underwent further trials after approval, with over half of all uses approved by the FDA post-approval. Additionally, 51% of these new uses were approved in new disease areas than the initial approval, often involving unique types or subtypes of cancer.
Similarly, another study found nearly half of cardiovascular drug new uses are identified more than seven years after initial FDA approval. What’s more, 82% of pediatric trials on existing medicines started about six years after the medicines first received approval, highlighting the importance of post-approval R&D, particularly in the later stages of a product’s lifecycle.
The evidence is clear: Post-approval innovations extend far beyond minor adjustments; they often represent significant breakthroughs for patients while providing widespread health and economic benefits to society. Intellectual property protections, including patents, are what incentivize manufacturers to continue to make investments to improve their medicines and explore how they may help a broader range of patients.
Organizations like I-MAK that downplay post-approval R&D—particularly when it leads to patent protections for new uses—fail to recognize the essential role of these innovations. This ongoing R&D, often involving additional costly and lengthy clinical trials, highlights the necessity of strong patent protections to safeguard scientific progress and ensure real-world benefits reach patients. Strong IP protections create the foundation for ongoing scientific discovery – encouraging companies to invest in new medicines that can reach more patients. Undermining this system poses a real threat, potentially freezing the progress of vital research and leaving groundbreaking treatments and cures undiscovered.
Post-approval innovation isn’t a loophole. It’s a lifeline. And it deserves the same protection and recognition as the first breakthrough.
Andrew Powaleny
Advances in Treatment
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Revolutionizing Cancer Care with Immunotherapy
In the early 2000s, the potential of immunotherapy—harnessing the body’s immune system to combat diseases—was met with skepticism and limited understanding. At the time, only a handful of immunotherapy drugs had FDA approval.Fast forward to today, and immunotherapy has transformed oncology. Groundbreaking advancements like therapeutic cancer vaccines, immune checkpoint inhibitors, CAR-T cell therapies, cytokines, and oncolytic viruses have redefined how we fight cancer.1 Since 2010, over 30 immunotherapy drugs have been approved to treat 25 types of cancer.
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Turning HIV/AIDs into a Treatable Chronic Condition
Just a few decades ago, the HIV/AIDS epidemic gripped the U.S. with devastating force. HIV cases soared, with limited understanding of the virus and a scarcity of effective treatments. At its peak, HIV was the leading cause of death among men aged 25–44.Fast forward to today, and the landscape is dramatically different. Thanks to expanded prevention strategies and groundbreaking treatments like antiretroviral therapies (ARTs) and pre-exposure prophylaxis (PrEP), death rates have plummeted, adherence to treatment has improved and life expectancy for those living with HIV has increased significantly. These advancements have not only saved lives but have also given hope for a future without HIV/AIDS.
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Gene Therapy Transforms Rare Disease Care
Gene therapy is changing the game for patients with rare diseases, offering hope and tangible results for those who once had few, if any, treatment options. This groundbreaking technology works by repairing faulty genes in the body, targeting the root cause of many rare diseases and offering the potential for life-changing benefits.Unlike traditional treatments, gene therapies have the potential for long-term, even curative effects. This means that patients can enjoy longer, healthier lives while avoiding the immense burden and high costs that often come with current standards of care. For many, gene therapy isn’t just a treatment; it’s a new lease on life.
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Committed to Research
All of PhRMA’s member companies meet the following criteria:
- A three-year average global R&D to global sales ratio of 10% or greater; and
- A three-year average global R&D spending of at least $200 million per year.
PhRMA member companies have invested over $1 trillion dollars in R&D since 2000, establishing the biopharmaceutical sector as the most R&D-intensive industry in the U.S. economy.
Through investment in R&D, biopharmaceutical researchers and scientists have gained a better understanding of diseases and a greater ability to harness new scientific advances, leading to tremendous progress in the development of treatments and cures for some of the most debilitating diseases patients face.