Life Science Compliance Update

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April 24, 2013

PhRMA Report: Over 5400 Medicines in Development and 70% are First in Class

Drugs in Development
According to a report released by PhRMA, companies have more than 5,400 medicines in development globally, and more than 70% of therapies in the pipeline are potentially first-in-class and could offer patients new treatment options, and a notable number of potential therapies target diseases with limited treatment options such as ALS and rare diseases.  A breakdown of their report offers insight into the various medicines in development for different diseases and populations.   

Older Americans 

America’s biopharmaceutical research companies are developing 465 new medicines that target the 10 leading chronic conditions affecting seniors.  These medicines – all in human clinical trials or under review the U.S. Food and Drug Administration (FDA) – are diverse in scope.  They include: 

  • 142 for diabetes, which affect 12.2 million Americans age 60 and older;
  • 92 for rheumatoid arthritis and osteoarthritis, which affect 1.3 million Americans and up to 80 percent of people over age 80, respectively;
  • 82 for Alzheimer’s disease, which could afflict 8 million people in the U.S. by 2030 unless a treatment or preventative measure is found;
  • 48 for heart failure (affecting 5.7 million Americans) and ischemic heart disease; and
  • 40 for chronic obstructive pulmonary disease, which impacts more than 13 million adults, with the highest prevalence rate in those over age 65. 

Similarly, many of the medicines in development for older Americans build on build on our growing knowledge and scientific progress to attack diseases in different ways. Among the potential medicines listed in the report are: 

  • A treatment that aims to prevent or reverse progression of Alzheimer’s disease by using a human monoclonal antibody specifically designed to draw beta amyloid protein away from the brain through the blood system.
  • A medication that combines two long-acting drugs, allowing for once-daily dosing in COPD.
  • A potential first-in-class medicine to treat type 2 diabetes that increases insulin secretion without causing insulin to significantly lower blood sugar.
  • A medicine that recruits a patients’ own neural stem cells to repair or protect against damage to the central nervous system from stress hormones, which can lead to depression 


There are more than five million Americans living with Alzheimer’s today, which costs the American health care system $200 billion a year. That number that could jump to 13.5 million by 2050, and the related costs of care would reach $447 billion.  A report released in December 2012, “Alzheimer’s Research: Setbacks and Stepping Stones,” found that between 1998 and 2011, 101 treatments have failed to reach patients.  In the same time period, three medicines have been approved to treat symptoms of the disease.  This 34 to one ratio of setbacks to successes underlines the difficulty of developing new medicines for Alzheimer’s. 

For patients with Alzheimer’s and other dementia, America’s biopharmaceutical research companies have 93 medicines in clinical trials or awaiting FDA approval.  The medicines in development – all in either clinical trials or under review by the Food and Drug Administration – include 81 for Alzheimer’s, 11 for cognition disorders and 2 for dementias.  Examples include: 

  • An intranasal medicine that is able to penetrate the blood-brain barrier for mild cognitive impairment, a precursor to Alzheimer’s disease.
  • A gene therapy for the treatment of Alzheimer’s disease.
  • A synthetic vaccine that induces antibody production without creating a systemic immune response 

PhRMA provided a useful list of clinical trials and links to their homepage.   


Arthritis is the most common cause of disability in the United States. Each year, it is responsible for 44 million outpatient doctor visits, nearly 1 million hospitalizations, and nearly 10,000 deaths. And it costs the American economy nearly $128 billion annually in direct medical costs and indirect costs, such as lost wages and productivity.  By 2030, it is estimated that the number of Americans with some form of diagnosed arthritis will increase to more than 67 million. 

The gains that have been made over the past 20 years in the treatment of patients with rheumatoid arthritis (RA), a devastating autoimmune disease that causes progressive joint deterioration and pain, show the impact treatment advances over time can play in improving the lives of millions of patients.  A PhRMA-sponsored white paper authored by Boston Healthcare Associates, Inc., Recognizing the Value of Innovation in the Treatment of Rheumatoid Arthritisexplores the various ways in which additional clinical value — including improved outcomes and quality of life — has been realized over time for RA patients as our understanding of the underlying disease expands. 

America’s biopharmaceutical research companies are currently developing 198 medicines to help the more than 50 million Americans afflicted with at least one of the 100 different musculoskeletal disorders, including arthritis.  All of the medicines are either in clinical trials or awaiting review by the U.S. Food and Drug Administration.  They include: 

  • 67 for rheumatoid arthritis that affects about 1.3 million American adults.
  • 23 for osteoporosis that affects 10 million people, 80 percent of whom are women.
  • 19 for lupus which affects an estimated 1.5 million Americans.
  • 19 for osteoarthritis, the most common form of arthritis, affecting nearly 27 million Americans.
  • 15 for musculoskeletal pain that affects the muscles, ligaments, tendons, and bones.
  • 9 for fibromyalgia which affects 3 million to 6 million Americans. 

Examples of some medicines now being tested to treat musculoskeletal disorders include: 

  • A new monoclonal antibody in development for lupus modulates B-cells that produce antibodies against the body’s own cells and tissue, causing the immune system to turn on itself.
  • A medicine in development for rheumatoid arthritis (RA) that inhibits two types of an enzyme that are key components in signaling activation of cytokines and growth factors that are elevated in patients with RA.
  • A potential first-in-class medicine in development for pain associated with osteoarthritis that is an inhibitor of a gene-encoding protein that plays a role in inflammatory pain. 


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. 

America’s biopharmaceutical research companies are working on 74 medicines to treat asthma, and PhRMA gave several examples 

Biotechnology and Biologics 

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.  The medicines and vaccines – all in human clinical trials or under review the U.S. Food and Drug Administration (FDA) – include 338 monoclonal antibodies, 250 vaccines, 93 recombinant proteins, 60 cell therapies, 46 gene therapies and 30 antisense medicines.  on biologics.   


America’s biopharmaceutical research companies are testing 981 medicines and vaccines to help in the fight against cancer.  These potential medicines, which are either in clinical trials or under review by the Food and Drug Administration, include 121 for lung cancer, 117 for lymphoma and 111 for breast cancer. 

More than 1.6 million new cases of cancer are expected in 2012. Researchers’ deep commitment to patients and advancing science is at the core of the remarkable progress made in fighting cancer.  Since 1980, life expectancy for cancer patients has increased by about three years, and 83 percent of those gains are attributable to new treatments.  According to the American Cancer Society, the cancer death rate fell 22% for men and 14% for women between 1990 and 2007, which translated to 898,000 fewer deaths from the disease in this period. 

In addition to the benefits for patients and their families, declines in cancer death rates have a tremendous economic impact.  The National Institutes of Health estimates overall costs for cancer in 2007 at $226.8 billion: $103.8 billion for direct medical costs (all health expenditures) and $123 billion for indirect mortality costs (cost of lost productivity due to premature death). According to recent research from the University of Chicago, reducing cancer death rates by 10 percent would be worth roughly $4.4 trillion in economic value to current and future generations. 


America’s biopharmaceutical companies are researching 316 medicines to help meet the unique health care needs of children and adolescents.  Thanks in part to major treatment advances, 82 percent of children diagnosed with cancer will survive five years or longer today, compared to 58 percent three decades ago, according to the American Cancer Society. 

Chronic Obstructive Pulmonary Disease (COPD) 

Approximately 13 million American adults suffer from COPD, which is a progressive lower respiratory disease that encompasses two main conditions–chronic bronchitis and emphysema.  In addition to those who have been diagnosed with the disease, 12 million Americans likely have COPD without knowing it.  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.  One in 10 American adults has diabetes now and as many as one in three could face the disease by 2050 if current trends continue, according to the Centers for Disease Control and Prevention. In addition to the terrible human toll associated with these outcomes, the economic consequences of diabetes are enormous.  In 2007, the cost of diabetes in the United States was $174 billion. If the additional costs of undiagnosed diabetes ($18 billion), pre-diabetes ($25 billion) and gestational diabetes ($623 million) are factored, the total annual cost of diabetes in the U.S. amounts to $218 billion. 

There are currently 221 medicines in the pipeline to treat diabetes and related conditions, in either clinical trials or awaiting approval from the FDA.  These medicines in development – all in either clinical trials or under review by FDA – include 32 for type 1 diabetes, 130 for type 2 and 64 for diabetes-related conditions.  There are 439 clinical trials listed here include 298 that have not yet started recruiting patients or are just now seeking volunteers to participate and another 141 that are active, but not recruiting new  patients.  

Since 1990, six new classes of diabetes type 2 medicines have been approved by FDA, giving patients and providers powerful new tools to treat the condition. America’s biopharmaceutical research companies continue to explore many different approaches to battle diabetes. Examples of the potential innovations outlined in the report include: 

  • A once-daily medicine that selectively inhibits the protein associated with glucose metabolism.
  • A medicine designed to inhibit an enzyme linked to diabetic neuropathy.
  • A medicine to treat type 2 diabetes that may allow for once-weekly dosing. 

A recent study in Health Affairs projected that improved adherence to diabetes medications could result in a reduction of more than one million emergency room visits and close to 620,000 hospitalizations annually, for a total potential savings of $8.3 billion annually. 

Heart Disease and Stroke 

The momentum of drug discovery helped cut deaths from heart disease and stroke by 31 percent between 1998 and 2008.  In 2008, stroke dropped to the fourth leading cause of death after being the third for more than 50 years.  According to the National Heart, Lung and Blood Institute (NHLBI), if death rates were the same as those of 30 years ago, 815,000 more Americans would die of heart disease annually and 250,000 more would die of stroke.  The cost of these diseases to American society is more than $503 billion a year

Much of the progress is due to the development of effective medicines to control both blood pressure and cholesterol, according to officials at the NHLBI. In addition, treatment of heart attacks has vastly improved. Twenty-five years ago, the treatment for heart attacks was simply bed rest. Today, doctors have medicines that can stop a heart attack in mid-stream, as well as other effective treatments.   

Maintaining that momentum, biopharmaceutical companies have developed 299 medicines for these diseases that are in clinical trials or awaiting approval by the FDA.  The medicines in development include 43 for lipid disorders, such as high cholesterol, 36 for heart failure, 27 for high blood pressure, 17 for heart attacks, and 27 for stroke. Many of the potential medicines use cutting-edge technologies and new scientific approaches. 


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 73 medicines and vaccines, focusing on improved treatment regimens, more effective therapies and promising new preventative solutions.  The medicines in development – all in either clinical trials or under review by the FDA – include 40 antivirals, 25 vaccines, four cell or gene therapies, and four immunomodulators. Examples of potential innovations included in the report include: 

  • An antisense gene therapy that uses genetic material derived from HIV-1 itself to remove disease-causing aspects of the virus.
  • A transdermal vaccine comprised of DNA plasmids that helps suppress virus replication and destroys HIV-infected cells.
  • A first-in-class medicine that is intended to prevent the HIV virus from attaching to new cells and breaking through the cell membrane. 

PhRMA provided a useful list of 137 clinical trials, including 51 that had not started recruiting or ar just now seeking volunteers, and another 86 that are active but not seeking new patients.  

Mental Illness 

The National Institute of Mental Health (NIMH) estimates that 1 in 4 American adults suffer from a diagnosable mental disorder.  According to the NIMH, serious mental illnesses cost the United States more than $317 billion annually in lost wages, health care expenditures, and disability benefits. 

Pharmaceutical research is targeting mental illnesses with nearly 200 medicines in clinical trials or awaiting approval by FDA.  This includes 26 medicines for addictive disorders; 20 for ADHD; 52 for depression; 36 for schizophrenia; 22 for sleep disorders; and 26 for anxiety disorders.  These medications are targeted at helping the more than 300 million people worldwide who suffer from some form of mental illness—from anxiety to depression and from schizophrenia to addictive disorders, such as dependence on alcohol or drugs.  Examples of some medicines now being tested to treat mental illnesses include: 

  • A medicine to potentially treat the various symptoms associated with schizophrenia, with diminished side effects.
  • An intranasal medicine for anxiety which has been shown to improve symptoms within several minutes of administration.
  • A potential first-in-class medicine for the treatment of major depression that recruits the patients’ own neural stem cells to protect  the central nervous system against damage from chronic exposure to stress. 

Parkinson's Disease 

Each year, approximately 60,000 Americans are diagnosed with Parkinson’s disease, a motor system disorder resulting from the loss of dopamine-producing brain cells, and incidence increases with age.  The combined cost to the U.S. economy in direct and indirect expenses is nearly $25 billion a year, according to the Parkinson’s Disease Foundation. 

America’s biopharmaceutical research companies are currently developing 36 medicines to help the nearly 1 million Americans suffering from Parkinson’s.  All of the medicines are either in clinical trials or awaiting review by FDA.  They include: 

  • Gene therapies that target specific areas in the brain.
  • Cell therapy that uses a patient’s own cells to reverse effects of the disease.
  • New delivery mechanisms of currently approved treatments, including a transdermal patch and an intranasal formulation.
  • Medicines to treat a motor function disorder associated with Parkinson’s disease treatment 

Rare Diseases 

The National Institutes of Health estimates that there are approximately 7,000 rare diseases affecting 25-30 million Americans.  They are officially deemed “rare” because alone they each affect fewer than 200,000 people.  Sometimes, only a few hundred Americans are known to have a particular rare disease.  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 400 medicines have been approved to treat rare diseases compared to fewer than 10 in the 1970s. These include the first treatments for Crohn’s disease and Lou Gehrig’s disease (also called ALS) and five treatments for pulmonary hypertension.   

The 460 medicines for rare diseases currently in development are all in later stages of the pipeline, meaning in clinical trials or under review by the FDA. A major area of research involves rare cancers. Solid tumors of the liver and thyroid, cancer of the blood and melanoma, or skin cancer, account for more than one-third of all rare disease drugs under development. 

Other major areas of research include: genetic disorders, such as cystic fibrosis, with 67 medicines in development; neurologic disorders, such as multiple sclerosis and muscular dystrophy, with 37 medicines in development; infectious diseases, such as anthrax and West Nile virus, with 31 medicines in development. 

Skin Diseases 

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.  According to a study by the Lewin Group, the total annual cost of skin diseases was estimated at $39.3 billion in 2005. 

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.  They include: 

  • 74 for skin cancers, including 63 for melanoma, which affects more than 68,000 Americans each year and is the most common form of cancer in the United States.
  • 60 for skin and soft tissue infections, which account for nearly 14 million outpatient visits each year.  
  • 41 for psoriasis, which affects about 7.5 million people in the United States, and is the most prevalent autoimmune disease in this country.
  • 14 for dermatitis (eczema), where 90% of suffers get the disorder before the age of 5. Worldwide 10-20% of children have dermatitis.
  • 9 for rosacea, which affects more than 14 million Americans. 


For many years, vaccines have been used to successfully prevent diseases such as smallpox, measles, polio and other infectious diseases. But vaccines are not only for preventing infectious diseases, newer vaccines are proving protections against a wide array of other diseases, including cancer prevention.  America’s biopharmaceutical research companies are working on 295 vaccines for the prevention and treatment of a wide variety of diseases.  They include 170 for infectious diseases, 102 for cancers and eight for neurological disorders.  Vaccines currently in development include: 

  • A genetically-modified vaccine for the treatment of pancreatic cancer.
  • A therapeutic vaccine that increases the immune response against the HIV virus.
  • A vaccine that protects infants against meningococcal disease, a leading cause of meningitis.
  • An immunotherapeutic vaccine for the treatment of Alzheimer’s disease.
  • A recombinant vaccine to prevent malaria. 

According to a recent study by the Centers for Disease Control and Prevention (CDC) in the U.S. there has been a greater than 90 percent decrease in incidence of nine infectious diseases, including smallpox and measles, for which vaccines have been recommended for decades. Reduction in mortality and morbidity by vaccination means less doctor’s visits and hospitalizations, which translates into healthcare cost savings, as well as fewer lost days at work, leading to improved productivity. 


Pharmaceutical researchers are developing more than 851 medicines for diseases that disproportionately affect women.  The medicines in the pipeline for women include: 

  • 139 for cancers affecting women, including 91 for breast cancer, 49 for ovarian cancer and nine for cervical cancer
  • 114 for arthritis/musculoskeletal disorders. Approximately 46 million Americans have some type of arthritis or related condition, and 60 percent of them are female.
  • 64 for obstetric/gynecologic conditions.
  • 110 for autoimmune diseases, which strike women three times more than men.
  • 72 for depression and anxiety. Almost twice as many women as men suffer from these disorders.
  • 83 for Alzheimer’s disease. Two-thirds (3.4 million) of the 5.4 million Americans living with Alzheimer’s today are women

Among the potential new medicines in development for women:  

  • A medicine that uses nanotechnology to target a cytokine that plays a key role in the inflammatory process associated with rheumatoid arthritis.
  • A first-in-class medicine in development for ovarian cancer that induces cell death and reduces cancer growth by inhibiting an enzyme responsible for cell division.
  • A first-in-class medicine in development for migraine that selectively blocks transmission of pain signals to the brain.
  • A new monoclonal antibody in development for lupus modulates B-cells that produce antibodies against the body’s own cells and tissue, causing the immune system to turn on itself.



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