New Medicines are Transforming Patient Care

The U.S. healthcare system faces great challenges that must be addressed. At the same time, outcomes continue to improve, with the use of new medicines playing a large role in achieving better results. This fact sheet provides examples for a few major disease areas.

In the last 10 years, more than 300 new medicines have been approved by the Food and Drug Administration. These medicines are helping patients live longer, healthier lives. They 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. (To download this document, click here.) 

"Many examples exist of major therapeutic gains achieved by the industry in recent years. ... Anecdotal and statistical evidence suggests that the rapid increases that have been observed in drug‐related R&D spending have been accompanied by major therapeutic gains in available drug treatments.”
—Congressional Budget Office, 2006

Increasing life expectancy

• Since 1950 life expectancy for men and women in the United States has increased by a full decade, from 68.2 to 78.2. In 1950 men could expect to live to 65.6 years and women, 71.1. In 2009 life expectancy was up to 75.7 for men and 80.6 for women. Life expectancy is continuing to rise steadily; just since 2000 we have seen an increase of 1.4 years.

Decreasing disability

• Disability among seniors sharply down. A 2008 study by Harvard University researchers found that between 1984 and 2004-05, disability in the elderly population fell by one-fifth. For cardiovascular disease the researchers report that medicines and other treatments increased the chances an elderly patient would survive a cardiovascular event without becoming disabled by 50 percent.

Cancer

• Life expectancy is increasing for cancer patients. Medicines are an important part of life expectancy gains. Since 1980 life expectancy for cancer patients has increased about 3 years, and 83 percent of those gains are attributable to new treatments, including medicines. Another study found that medicines specifically account for 50 percent to 60 percent of increases in survival rates since 1975.

  "I think we really are in the midst of a revolution in the treatment of cancer.”
  —Dr. Len Lichtenfeld, American Cancer Society, 2006

• Cancer death rates began to fall for the first time in the 1990s and are continuing to decline. According to a new report from the National Cancer Institute, between 2003 and 2007 death rates fell an average of 1.6 percent per year. According to the report, “These decreases indicate real progress in cancer control, reflecting a combination of primary prevention, early detection and treatment.”
   
• Decline in the cancer death rate is tremendously valuable. Research findings by University of Chicago economists Kevin Murphy (a MacArthur fellow) and Robert Topel show that reducing cancer death rates by 10 percent would be worth roughly $4.4 trillion in economic value to current and future generations.
   
• Five‐year survival is rising. The chances that a cancer patient will live at least five years has increased by 36 percent across cancers. In 1975 the five‐year survival rate was 49 percent. By 2003 (the most recent data available), survival rose to 67 percent.
   
• Survival is increasing dramatically for many forms of cancer. Between 1975 and 2003 (the most recent data available) five‐year survival went up 19 percent for women with breast cancer (fron 75.5 percent to 89.9 percent); 50 percent for men with prostate cancer (from 66.4 percent to 99.4 percent); 35 percent for patients with colon and rectum cancer (from 48.7 percent to 65.6 percent), and 39 percent for lung and bronchus cancer (from 11.5 percent to 16 percent).
   
• The chance of survival for children with cancer is up 40 percent. For all childhood cancers combined, the number of children surviving five years after diagnosis has grown from 58 percent in 1975 to 81 percent today due in part to new and improved treatments.
   
• New cancer medicines significantly advanced treatment in 2010. A report by the American Society of Clinical Oncology (ASCO) identified 12 major advances in the treatment of cancer in 2010 and, among those, eight related to new medicines, better ways to use existing medicines, or newly discovered benefits or approved medicines.

"Scientifically, we have never been in a better position to advance cancer treatment. ... We now understand many of the cellular pathways that can lead to cancer. We have learned how to develop drugs that block these pathways. And increasingly, we know how to personalize therapy to the unique genetics of the tumor, and the patient.”
—Richard L. Schilsky, MD, professor, University of Chicago, and former president, ASCO, 2008

Cardiovascular disease

• Deaths from heart disease and stroke are falling. According to a 2010 statistics update by the American Heart Association (AHA), death rates for cardiovascular disease fell a dramatic 28 percent between 1997 and 2007. According to the lead researcher of another AHA report, there would have been an additional 190,000 deaths in 2006 if death rates had remained at 1999 levels.
   
• According to the Centers for Disease Control and Prevention, new medicines contribute to improving trends in cardiovascular disease: “Factors contributing to the decline in heart disease and stroke mortality include better control of risk factors, improved access to early detection, and better treatment and care, including new drugs and expanded uses for existing drugs.”
   
• Heart failure and heart attack death rate fell by nearly half from 1999 to 2005. A 2007 study published in the Journal of the American Medical Association reports that the death rate for heart-attack patients in hospitals decreased from 8.4 percent in 1999 to 4.6 percent in 2005. The study also showed that congestive heart failure developed in 11 percent of heart-attack patients in 2005, down from 19.5 percent in 1999. The researchers point to the increased use of cholesterol drugs, blood thinners and angioplasties as a direct cause of lowered rates of death and heart failure.
   
• Blood-pressure medicines save lives and prevent hospitalizations, but even more could be saved if all patients received recommended care. A 2007 study in Health Affairs found that use of antihypertensive medicines prevented 86,000 premature deaths from cardiovascular disease in 2001 and 833,000 hospitalizations for heart attack and stroke in 2002. If all untreated patients received recommended care, an additional 89,000 deaths and 420,000 hospitalizations could be prevented each year.
   
• Despite rising obesity levels, Americans have reached a milestone in controlling high cholesterol. The Centers for Disease Control and Prevention reported at the end of 2007 that U.S. adults reached an average cholesterol level in the ideal range (below 200) for the first time in 50 years. Authors of the report attribute the drop to the increased use of cholesterol‐lowering medicine in the over‐60 population, but warn that increasing obesity rates could wipe out the gains.

"Protein enzymes, receptors or channels identified by the pharmaceutical industry as 'drugable targets' have led to striking, remarkable and repeated achievement.”
—Drs. Myron Weisfeldt and Susan Zieman, Johns Hopkins University, “Advances in the Prevention and Treatment of Cardiovascular Disease,” Health Affairs, 2007

HIV/AIDS

• Life with HIV/AIDS has changed dramatically. Patients diagnosed with AIDS in 1990 could expect to live only months, during which time they would be likely to contract a number of opportunistic infections. The only treatment available had to be taken every four hours -- around the clock -- and had serious side effects. Since the approval of anti‐retroviral treatments (ART) in 1995, the AIDS death rate has dropped by 78 percent. If diagnosed today, a range of treatment options, including different combinations of drugs, often keep patients symptom‐free for years.
   
• HIV is a rapidly mutating virus, but new treatments are keeping ahead. Researchers are always in a race against time with HIV because the virus adapts to existing treatments, making them less effective. In recent years new HIV treatments have continued to be approved, including a new personalized HIV treatment that represents a new class of medicines. New treatments such as this one represent important options for patients whose infections are not responding to available medicines.
   
• Thanks in part to continued treatment advances, HIV death rates are continuing to fall. The most dramatic drops in HIV death rates occurred following the introduction of ART, with decreases averaging 33 percent per year between 1995 and 1998. From 1999 to 2008, death rates continued to drop by 5 percent per year. The most recent data has shown continued decline in death rates: Between 2008 and 2009, death rates fell another 9 percent. Among people aged 25‐44 years, death rates fell by more than one‐half in 2007 alone (the most recent age group‐specific data).
   
• Hospitalizations have dropped. Since ART became available, the number of people with HIV increased by 28 percent between 1996 and 2000, primarily because of rising survival rates. Hospital rates, however, fell by 32 percent over the same period.
   
• University of Chicago economists report that each patient with HIV now lives 15 years longer than they would have in the 1980s.
   
• Earlier use of medicines is recommended. Based on growing evidence that unchecked HIV virus is a greater risk than adverse reactions to the medicine, new guidelines issued by the International AIDS Society are recommending earlier initiation of ART even in many cases when the patient is asymptomatic. These guidelines recommend treatment when the CD4 cell count drops below 500/microlitre and should even be considered before blood count drops to the level. Previous guidelines had recommended treatment when the CD4 count fell to 350/microlitre.
   
• Use of HIV medicines helps prevent transmission. A recent study reported in The Lancet and carried out in Africa found that initiation of ART reduces the risk of transmission from infected individuals to their sexual partners by 92 percent. A large new study sponsored by the National Institute of Allergy and Infectious Disease echoes this result, finding that early initiation of ART reduced transmission by 96 percent. The international study was stopped and unblinded four years early because the findings were so robust.
   
• Medicines have become easier to use, adding to their effectiveness. The first once‐daily one‐pill combination tablet was approved for treatment of HIV in 2006. This medicine combines the active ingredients of three widely used antiretroviral drugs into a single dose. The single‐pill treatment regimen is a marked improvement over previous HIV/AIDS treatments, which often require patients to take multiple pills every day. Missing doses can lead the virus to mutate and become resistant to medicines. Because this new pill will make it easier for patients to remember to take their medicines, it is believed that “widespread use of the combination drug could potentially even slow the spread and evolution of the AIDS epidemic itself."

"The development of [antiretroviral therapy (ART)] has been the one of the greatest accomplishments of basic and translational research: approximately 30 anti‐HIV agents are licensed and evidence‐based guidelines have been developed for their optimal use. Combination ART with at least three drugs has resulted in substantial reductions in morbidity and mortality in both rich and poor countries. Antiretroviral therapy has been simplified to the point where treatment with a single, multidrug pill once a day is possible with generally manageable adverse effects. With improvements in ART, the estimated life expectancy of certain HIV‐infected patients now approaches that of uninfected individuals. Antiretroviral therapy also has proven efficacious in HIV prevention, reducing the risk of mother‐to‐child transmission and serving as post-exposure prophylaxis for individuals exposed to HIV.”
—Gregory K. Folkers, MS, MPH; Anthony S. Fauci, MD, National Institute of Allergy and Infectious Diseases, 2010

"Antiretroviral therapy of HIV infection has changed a uniformly fatal into a potentially chronic disease. ... Patients who can access and adhere to combination therapy should be able to achieve durable, potential lifelong suppression of HIV replication.”
—Paul Volberding, MD and Steven Deeks, MD, University of San Francisco, The Lancet, 2010

Diabetes

• Although diabetes patients are undertreated and the disease is becoming more prevalent, many effective treatments are available and the diabetes death rate fell 4 percent in 2009. However, without greater prevention of obesity and other risk factors and consistent treatment, this progress could be at risk.
   
• Many new treatments exist for diabetes. In recent years, eight new classes of diabetes medicines have been approved, giving patients and providers powerful new tools to treat the condition.
   
• Taking diabetes medicines as directed reduces the risk of hospitalizations.
   
• Improved use of diabetes medicines cuts risk of hospitalization by half. Patients who are less than 80 percent adherent to their diabetes medicines are 2‐3 times more likely to be hospitalized in the next year compared with patients who are more adherent. The less often a patient took medication as directed, the more likely the patient was to be hospitalized.
   
• Adherent diabetes patients are less likely to use hospital and ER care. A recent study found that emergency-room visits among patients who took their diabetes medicines as directed was 46 percent lower than for patients who took their medicines less than 50 percent of the time. Similarly, the hospitalization rate and the number of days spent in the hospital were 23 percent and 24 percent lower, respectively, for adherent patients.
   
• Diabetes patients treated with medicines are less likely to develop other health problems. Diabetes patients who are treated with diabetes medicines are 31 percent less likely to develop lipid disorders and 13 percent less likely to develop high blood pressure than untreated patients.

Rheumatoid arthritis

• Clinical remission is now possible for patients with severe rheumatoid arthritis (RA). A recent study found that patients treated with combination therapy consisting of both a new and older medicine had a 50 percent chance of complete clinical remission after 52 weeks of treatment, compared with 28 percent taking only the older medicine. These results would have been “unthinkable” before new disease‐modifying biological medicines.
   
• In 2008 the American College of Rheumatology rewrote rheumatoid arthritis guidelines to account for biopharmaceutical advances in treating RA. These guidelines recongnized the new treatment options represented by biologic medicines.
   
• Treatment of rheumatoid arthritis has changed significantly in recent years:

"Only 20 years ago, rheumatoid arthritis (RA) was regarded as a relentlessly progressive disease. Treatments provided little hope of significantly modifying long‐term disease outcome. The literature painted “a grim picture” suggesting “that both premature death and marked functional morbidity occur even in population‐based analyses” and that “the long‐term prognosis of rheumatoid arthritis is bad”. ... “During the ensuing 20 years, we have witnessed significant advances. In clinical trial design and daily practice, widespread use is made of a variety of disease activity measures and response criteria that have been developed and validated. ... In parallel, unique advances were made in the therapeutic arena: recognition of the importance of early treatment, emergence of MTX [methotrexate] as a recognized DMARD [disease‐modifying anti‐rheumatic drug] with far greater effectiveness and safety than previously available DMARDs, and new biologic agents successfully expanded the results of treatment of RA.”
—Drs. Joseph Smolen and Daniel Aletaha, Clinical and Experimental Rheumatology, 2006

"Today a new class of anti‐rheumatic drugs dramatically slows disease progression.”
—Dr. Elias Zerhouni, former director, National Institutes of Health, 2008 Osteoporosis

Osteoporosis

• Osteoporosis medicines greatly reduce the risk of fractures when taken consistently. Patients who are over 80 percent adherent to their osteoporosis medicines have a 25 percent lower rate of fractures compared with those who are less adherent. Those who are the least adherent have a 40 percent increased risk.

Rare diseases

• New treatment options for rare diseases give new hope to patients with few existing treatment options. Since the passage of the Orphan Drug Act in 1983, 369 medicines have been approved to treat rare diseases. Examples include:
  
  • The first treatment for ALS or Lou Gehrig’s disease.
  • Five new treatments for pulmonary hypertension.
  • A genetically engineered antibody that is the first treatment for Crohn’s disease.
  • The first medicine that treats the cause of Fabry disease rather than its symptoms.
  • The first in a new class of medicines to treat acromegaly, a disorder in which excess growth hormone causes enlarged hands feet and facial features.

To see a full list of sources cited, click here.