We Drive Innovation

Robust investment in research and development by biopharmaceutical companies has resulted in advances and discoveries unlike anything we've seen. In the last decade, we have invested half a trillion dollars, and these investments are just beginning to pay off.

The progress we're seeing today is revolutionizing how we treat disease. These investments are saving lives and improving quality of life and public health by tackling a broad range of chronic and rare diseases. And today, there are about 7,000 medicines in development, with about three quarters having the potential to be first‑in‑class treatments.

Innovative new medicines keep patients healthy and out of the hospital, reducing the need for expenses such as ER visits, hospital stays, surgeries and long-term care - ultimately saving money for patients and the nation's health system.

America's biopharmaceutical companies invest about $75 billion annually in research and development in the U.S. and support 4.7 million jobs across the country
We Support a Strong Economy

Investments in biopharmaceutical innovation are not only improving and saving lives, but they are driving tremendous contributions to the American economy - all while solidifying America's role as a leader in medical innovation. Thousands of biopharmaceutical researchers work every day to develop new treatments and cures for patients.

We Support Patient Access to Care

The growing use of deductibles and coinsurance for prescription medicines creates affordability challenges for many patients. Biopharmaceutical companies are committed to ensuring that patients have access to medicines. We offer copay assistance to patients with insurance and additional support so underinsured or uninsured patients can get the medicine they need for free or nearly free.

We Believe in Our Market-Based System

New medicines are transforming care for diseases like cancer, hepatitis C and high cholesterol. But medicine costs are growing at the slowest rate in years. That's because the market-based system in the U.S. promotes incentives for continued innovation while leveraging competition to control costs.

While our current system for paying for medicines works well to hold down costs, it is complicated and can result in uncertainty and frustration for some patients. We believe insurance companies should pass on more of the discounts they receive to patients in the form of lower out-of-pocket costs, just like they do for other health care services.

Profile Toolkit: Innovation

Medicines are Transforming the Trajectory of Disease

Progress against Disease

Medicines have played a central role in transforming the trajectory of many debilitating diseases, resulting in decreased death rates, improved health outcomes and better quality of life for patients.

• Cardiovascular Disease: Tremendous strides have been made in reducing death rates for cardiovascular disease over the last 40 years, due in large part to advances in treatment.

• HIV/AIDS: Once considered acutely fatal, HIV/AIDS is now a chronic and manageable disease.

• Hepatitis C: Today, a broad range of treatments with minimal side effects and cure rates approaching 100% are available for patients with all forms of the disease.

• Cancer: Since peaking in the early 1990s, the U.S. has witnessed a 25% decline in cancer death rates.

Recent Approvals

In 2016, the U.S. Food and Drug Administration (FDA) approved 27 new medicines, including 22 new medicines approved by the FDA Center for Drug Evaluation (CDER). Among CDER's approvals, 36 percent were first‑in‑class medicines, representing entirely new ways of treating disease.

Examples of novel therapies that became available to patients in 2016 include:

• The first and only treatment for the rare and often fatal genetic condition known as spinal muscular atrophy (SMA). The new medicine is an antisense oligonucleotide designed to treat the disease caused by mutations in the chromosome 5q, which leads to a key protein deficiency.

• A first‑in‑class personalized medicine to treat chronic lymphocytic leukemia (CLL) patients with a specific chromosomal abnormality which is detected by a genetic test. The drug is an inhibitor of the BCL-2 protein, a protein that blocks "programmed cell death," including in cancer cells which can be overexpressed in some CLL patients.



of medicines in development have the potential to be first‑in‑class treatments.

The Future Has Never Been Brighter

There are about 7,000 medicines in development globally which could impact U.S. patients.

Medicines in development include:

• Blood cancers: CAR T-cell therapy is an emerging approach showing tremendous promise. It involves removing immune-boosting T-cells from a patient, engineering them to recognize and kill cancer cells, and returning the cells back to the patient.

• Migraines: After decades of research into a signaling pathway that appears to play a central role in migraines, calcitonin gene-related peptide (CGRP) inhibitors are offering new hope.

• Crohn's disease: A new approach works by blocking production of a protein called SMAD7, which is present at high levels and blocks pathways that keep immune cells in check.

• Alzheimer's Disease: One approach in the pipeline involves blocking the BACE1 enzyme that is integral to creating amyloid-beta, which makes up the plaques involved in the progress of the disease.

Profile Toolkit: Savings

Better Use of Medicines Can Improve Health Outcomes and Reduce the Use of Costly Medical Care

The Economic Burden of Chronic Disease is Substantial

The cost of treating patients with chronic conditions accounts for 90 percent of the nearly $3 trillion spent on health care in the U.S. each year. Patients with multiple chronic conditions are a significant driver of health care costs. In fact, the 12 percent of people with five or more chronic conditions account for 41 percent of total health care spending and spend 14 times more on health services than people with no chronic conditions.

  • 60%

    of American adults have at least one chronic condition.

  • 42%

    of American adults have at least two chronic conditions.

Significant Gaps in Optimal Use of Medicines

Nearly 75 percent of American adults do not follow their physicians' prescription orders, including not filling their prescriptions or taking less than the recommended dose. More than one quarter of newly written prescriptions are never brought to the pharmacy to be filled, including those for common conditions such as high blood pressure, diabetes and high cholesterol.

Patients with chronic conditions with recurring annual costs for medicines are disproportionately affected by declining generosity of coverage. This can lead to poor utilization of prescribed treatments and eventually poor health outcomes.

Better Use of Medicines Can Improve Health Outcomes and Reduce Health Care Spending

A large body of evidence demonstrates how better use of medicines can lead to reductions in other sources of health care spending across a broad range of chronic conditions.

Patients with less common chronic diseases may also reduce their health care spending through better adherence. For example, Medicare patients with Parkinson's disease, adults with Crohn's disease, children with cystic fibrosis and patients with advanced melanoma have all been shown to achieve health care savings through improved use of medicines.

Today and in the future, medicines will play a central role in making our health care more sustainable. The development of medicines will be critical in addressing the most costly and challenging diseases of our time.



of medications for chronic disease are taken as prescribed.


  • Better use of medicines could eliminate $213 billion in U.S. health care costs annually, amounting to 8 percent of the nation's health care costs.

  • But if we develop one new medicine to delay the onset of the disease by just five years we counld save the healthcare system $367 billion by 2050

  • Spending just $1 more on medicines for patients with high blood presure or diabetes can generate $3-10 in savings on ER visits and hospitalizations.

Profile Toolkit: Economy

The Biopharmaceutical Industry's Role in Fueling the Economy and Global Competitiveness

America's biopharmaceutical Industry Is the Most Research Intensive

The biopharmaceutical industry is the global leader in R&D, and its research intensity is unparalleled in the United States economy.

As a result, U.S.-based biopharmaceutical companies invested about $75 billion in R&D in 2015, with most of these investments made directly in the United States.

  • Relative to other manufacturing industries, the biopharmaceutical industry invests 12 times more in R&D per employee

  • At 25 percent biopharmaceutical companies had the highest growth rate in R&D investment across all manufacturing industries.

  • The biopharmaceutical industry is the single largest funder of medical and heal R&D in the United States, accounting for about half of all such research in the country.

The pharmaceutical Industry is one of the most research-intensive industries in the United States. Pharmaceutical firms invest as much as five times more in research and development, relative to their sales, than the average manufacturing firm."
Congressional Budget Office
The U.S. Biopharmaceutical Industry Is the Global Leader in Biomedical Innovation

The United States leads in both overall clinical trial activity and in early stage clinical research, but it also claims the intellectual property of more than half of all new medicines invented. In terms of academic contributions, the United States also leads in peer-reviewed publications. Likewise, it is not surprising that more than two-thirds of worldwide venture capital investments in biopharmaceutical startups are made in the United States where the biopharmaceutical research and development enterprise thrives.

We need to continue to focus on where the United States has a competitive advantage and shore up areas where other countries are catching up and where the United States is falling behind — including science, technology, engineering and math (STEM) education. Looking ahead, we need to strengthen U.S. economic foundations and compete for biopharmaceutical development.

Today, a more intensive and globalized competition for the biopharmaceutical industry is taking root, with the developing world joining European competitors in seeking to challenge the U.S. global leadership in innovation. The United States is now facing increasing competition to attract and grow a biopharmaceutical presence not just from developed countries, but also from emerging nations such as Brazil, China and Singapore that are laying the groundwork for future growth."
TEConomy Partners

Profile Toolkit: R&D

The Dynamic U.S. Research and Development Ecosystem


On average it takes 10 to 15 years for a medicine to make its way through the entire R&D process to approval by the U.S. Food and Drug Administration (FDA)

America's Biopharmaceutical Companies Play Central Role in R&D Ecosystem

U.S. biopharmaceutical companies play a central role in the biomedical research ecosystem. The system is complex and marked by collaborations across industry, academic institutions, government agencies, venture capital firms, nonprofit foundations and others. This diverse group of stakeholders works in concert to advance basic science that forms the foundation for research and development of new medicines, which is led by biopharmaceutical companies.

Download Results from the 2017 PhRMA Annual Member Survey
The Complex Biopharmaceutical R&D Process

The drug development process typically begins with the screening of an enormous number of potential medicines. And after an average of 10-15 years, only 12 percent of investigative medicines entering clinical trials are ultimately approved by the FDA.

The average cost to develop a new medicine is estimated at $2.6 billion dollars, including the cost of failures. The cost of development has more than doubled over the past decade. Over this same period, the regulatory burdens have steadily increased resulting in more complex clinical trials. Despite these challenges, biopharmaceutical research companies remain committed to bringing new and important new treatment options to patients. PhRMA members alone have invested more than half a trillion dollars in R&D since 2000.

The Evolving R&D Process

Biopharmaceutical companies are using the latest tools such as innovative clinical trial designs, real world evidence and biomarkers to make development as efficient as possible. They also incorporate use of patient-reported outcomes measures.

New scientific advances bring greater promise and complexity, as well as a higher degree of scientific and regulatory uncertainty for biopharmaceutical companies.

Providing Hope to Patients

We are in the midst of an unprecedented period of medical discovery, one that decades from now will be credited as resulting in new cures for many of our most costly and debilitating diseases and providing treatment options that allow many to live longer, healthier lives.

It is critical that we have a policy and regulatory environment that fosters innovation. To continue to advance medical discovery, we need to ensure a well-functioning, science-based regulatory system that keeps pace with the latest advances and also ensures the timely review, approval and introduction of new medicines. We also need to foster the development of a highly trained STEM workforce to support the discovery, development and delivery of new treatments. Finally, it is important that we have robust intellectual property (IP) rights and regulatory incentives to foster investment in R&D and commercialization of new technologies.