Medicines in Development

Explore the Latest Progress on Medicines in Development

Each patient battling with a disease or managing a condition lives in the hope that tomorrow will bring a new medicine that delivers better health. America’s biopharmaceutical research companies share the same goal of developing new medicines that can prevent diseases, improve patients’ health, and save lives. Beyond each individual patient, new drugs can help society overall by reducing sick days and helping cut the cost of healthcare.

Subscribe ButtonThe economic impact of new medications and adherence could be significant. For example, one study found that annual costs for diabetes care could drop as much as 48 percent for patients who take their diabetes medicines properly. With 24 million people with diabetes in the U.S., and only one-quarter of them controlling the disease, the savings could add up quickly as millions of Americans improved their health.

Since 2000, more than 500 new medicines have been approved by the FDA, helping patients live longer, healthier lives. Medications are transforming many cancers into treatable conditions, reducing the impact of cardiovascular disease, offering new options for patients with hard-to‐treat diseases like Alzheimer’s and Parkinson’s, and fighting even the rarest conditions.  

Featured Report

Rare Diseases

The National Institutes of Health estimates that there are approximately 7,000 rare diseases affecting 30 million Americans. In the past, treatment options for such conditions have been nonexistent or limited, but since the passage of the Orphan Drug Act of 1983, more than 500 medicines have been approved to treat rare diseases. These include the first treatments for Crohn’s disease and Lou Gehrig’s disease (also called ALS) and five treatments for pulmonary hypertension.


There are more than five million Americans living with Alzheimer’s today. For patients with Alzheimer’s and other dementia, America’s biopharmaceutical research companies are developing medicines to bring us closer to our goal of tackling and eventually conquering this debilitating disease. 


Arthritis is part of a group of related musculoskeletal diseases consisting of more than 100 different conditions that affect more than 52 million people in the United States. Prevalence of musculoskeletal diseases could increase to 67 million by 2030 if current trends continue. America’s biopharmaceutical research companies are currently developing 92 medicines to help the millions of Americans affected by arthritis. All of the medicines are either in clinical trials or awaiting review by the U.S. Food and Drug Administration.


Asthma is a narrowing of the airways to the lungs caused by inflammation in the air passages, resulting from both genetic and environmental influences. Today, more than 24 million American adults and children suffer from asthma, with the prevalence increasing 12 percent in the last decade, according to the Centers for Disease Control and Prevention. Each day, 40,000 Americans miss school or work due to asthma, costing the U.S. economy an estimated $56 billion each year in direct and indirect costs.

Biotechnology and Biologics

Millions of people already have benefited from medicines and vaccines developed through biotechnology. America’s biopharmaceutical research companies are using biological processes to develop 907 medicines and vaccines targeting more than 100 diseases—including 352 for cancer, 188 for infectious diseases, 69 for autoimmune diseases, and 39 for AIDS/HIV and related conditions. Biologics are made from a variety of natural sources—human, animal or microorganisms. Like small-molecule drugs, some biologics are intended to treat diseases and medical conditions. Other biologics are used to prevent or diagnose disease. These medicines represent the next exciting frontier of biopharmaceutical research.


Research has expanded our knowledge of how cancer develops and how to target medicines for specific cancer types, which has resulted in more effective therapies for patients. America’s biopharmaceutical research companies are currently developing 836 new medicines and vaccines for cancer, all of which are in clinical trials or awaiting review by the U.S. Food and Drug Administration.


America’s biopharmaceutical companies are researching 316 medicines to help meet the unique health care needs of children and adolescents. Considerable progress in the fight against diseases that impact children has been achieved in recent decades, with medicines playing a key role

Chronic Obstructive Pulmonary Disease (COPD)

Chronic obstructive pulmonary disease (COPD) is a progressive lower respiratory disease that encompasses two main conditions–chronic bronchitis and emphysema. The condition is characterized by obstructed airflow to the lungs that interferes with normal breathing. For the more than 13 million American adults that suffer from COPD, hope lies ahead as biopharmaceutical companies are working on 54 medicines to treat the disease.


Diabetes affects nearly 26 million Americans—8.3 percent of the U.S. population—and about one-quarter are unaware they have the disease. A diagnosis and appropriate medication can help patients avoid serious complications and death, and can reduce overall healthcare spending. There are currently 180 medicines in the pipeline to treat diabetes and related conditions, either in clinical trials or awaiting approval from the FDA. These medicines represent an exciting new chapter in the ongoing quest to better treat this debilitating disease.

Heart Disease and Stroke

According to the American Heart Association, someone in the United States dies from cardiovascular disease every 40 seconds, and more than 85 million Americans have at least one form of the disease. Heart disease has been the leading cause of death in the United States since 1921, but these numbers are declining, thanks in large part to new medicines.  America’s biopharmaceutical research companies are developing 190 medicines for heart disease and stroke. 


Since the approval of anti‐retroviral treatments (ART) in 1995, the AIDS death rate has dropped by more than 80 percent. Testing for the disease also has advanced dramatically, enabling earlier treatment. Instead of being a death sentence, HIV is now often a manageable chronic disease. Biopharmaceutical research companies are developing 44 medicines and vaccines, focusing on improved treatment regimens, more effective therapies and promising new preventative solutions. 

Infectious Diseases

Throughout history, infectious diseases have taken a devastating toll on the lives and well-being of people around the world. Today, America’s biopharmaceutical research companies are developing 394 new medicines to help fight the continuing threat from infectious diseases.

Leukemia and Lymphoma

Each year more than 162,000 Americans are diagnosed with a blood cancer—accounting for more than 9 percent of all new cancer diagnoses. The major types of blood cancer include leukemia, lymphoma and myeloma. Biopharmaceutical research companies are developing 247 medicines targeting leukemia, lymphoma, myeloma and other cancers of the blood.

Mental Health

Mental health conditions exact a heavy human and economic toll in the United States. According to the National Institute of Mental Health (NIMH) serious mental illnesses cost the United States more than $317 billion annually in lost wages, health care expenditures and disability benefits.

Neurological Disorders

Neurological disorders—such as epilepsy, multiple sclerosis and Parkinson’s disease—inflict great pain and suffering on patients and their families, and every year costs the U.S. economy billions of dollars. Biopharmaceutical research companies are developing 444 new medicines to prevent and treat neurological disorders.

New Drug Approvals

In 2013 the U.S. Food and Drug Administration approved 34 new medicines, 27 of which were approved by the Center for Drug Evaluation and Research (CDER) and seven by the Center for Biologics Evaluation and Research (CBER). Among the drugs approved were eight medicines to treat cancer, including the first treatments for the first two medicines approved with “Breakthrough Therapy” designation, the first antibody-drug conjugate approved for breast cancer, and two for melanoma.

Older Americans

With the population of Americans over 65 on the rise and life expectancy climbing, chronic diseases remain a principal threat to the health and productivity of older Americans, as well as to rising health care costs. Innovative medicines have led to major advances against many chronic diseases – and the robust discovery pipeline of new medicines portends continued progress for seniors and our health care system. America’s biopharmaceutical research companies are developing 435 new medicines that target 15 leading chronic conditions affecting seniors.


Nearly 54 million adults in the United States have either osteoporosis or low bone density, according to the National Osteoporosis Foundation. Today, biopharmaceutical research companies are developing nine medicines for the treatment of osteoporosis, all of which are in clinical trials or undergoing regulatory review by the U.S. Food and Drug Administration.

Parkinson's Disease

America’s biopharmaceutical research companies are currently developing 37 medicines to help the estimated 1.5 million Americans living with Parkinson’s disease, a motor system disorder resulting from the loss of dopamine-producing brain cells. All of the medicines are either in clinical trials or awaiting review by the U.S. Food and Drug Administration.

Skin Diseases

America’s biopharmaceutical research companies currently are developing 277 medicines to help the more than 100 million Americans, one third of the U.S. population, that suffer from at least one skin disease. Skin diseases, ranging from acne to psoriasis and from melanoma to infections, are more common than most people know and they come with not only a medical but also a financial burden.


For many years, vaccines have been used to successfully prevent devastating infectious diseases such as smallpox, measles and polio. America’s biopharmaceutical companies are currently developing 250 vaccines to prevent – and in some cases treat – a variety of conditions, including infectious diseases, various forms of cancer and neurological disorders.


More On PhRMA — powered by PhRMApedia


Cost in Context