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Saving & Improving Lives

New Medicines Transforming Patient Care
Extending Lives

Promoting Productive and Healthy Lives

New Approvals in 2011

Bringing Hope to Patients with Rare Diseases


New Medicines Transforming Patient Care
In the past 10 years, 340 new medicines have been approved by the U.S. Food and Drug Administration (FDA). Prescription medicines developed as a result of biopharmaceutical research have contributed to significant reductions in deaths from many diseases. These medicines bring great value, allowing people to live productive and healthy lives and offering new hope and improved quality of life to millions of patients.

Extending Lives
New medicines and better prevention have made significant contributions to reducing death and disability from many diseases. For example:


  • Cardiovascular disease (CVD). According to the American Heart Association, death rates for CVD fell a dramatic 28% between 1997 and 2007[i], due in large part to improved treatments. Similarly, heart failure and heart attack death rates following hospital discharge fell by half between 1999 and 2005. [ii
  • Diabetes. Eight new classes of diabetes medicines have been developed in recent years, providing powerful new treatment tools to fight the disease. People recently diagnosed with diabetes can now expect to live longer than those diagnosed 10 or 20 years ago. And while heart disease is a frequent complication of diabetes, today people with diabetes who take medicines are 31% less likely to develop lipid disorders such as high cholesterol and 13% less likely to develop high blood pressure—two major risk factors for premature death from heart disease—than those not taking medicines. [iii] 
  • HIV/AIDS. The development of highly active antiretroviral therapy (HAART), a combination of medicines, in 1995 completely changed the face of HIV treatment. Since then, the HIV/AIDS death rate has fallen by 83% in the United States.[iv] The death rate has continued to fall in recent years: between 2009 and 2010, death rates fell 13%.[v]  Among people aged 25 to 44 years, death rates from HIV/AIDS fell by more than one-half in 2007 alone (the most recent age group-specific data). [vi] 
  • Cancer. For people diagnosed between 1975 and 1979, the five-year cancer survival rate was 49%. For those diagnosed in 2003 (the most recent year for which five-year survival rates are available), it was 67%.[vii]  For children, the five-year survival rate has grown from 58% for those diagnosed between 1975 and 1977 to more than 80% today.[viii]

Promoting Productive and Healthy Lives
Prescription medicines can prevent disease progression and serious complications, allowing patients to live productive and active lives.


  • Rheumatoid arthritis (RA). New disease-modifying therapies, in combination with older medicines, can dramatically slow disease progression, transforming the lives of people with this crippling condition. One study showed that patients using combined treatment had a 50% chance of complete remission, compared with a 28% chance among those taking only the older medicine.[ix] Another study found a 26% decrease in lost productivity among RA patients who were more adherent to their medicines.[x]  
  • Osteoporosis. Osteoporosis medicines significantly reduce fracture risk, and people who consistently take their osteoporosis medicine have a 25% lower rate of fracture compared with people who are less adherent. [xi] Preventing fractures is important in elderly populations; for example, one in five people who experience a hip fracture move to a nursing home within a year.[xii]

New Approvals in 2011
In the past 10 years, 340 new medicines have been approved by the U.S. Food and Drug Administration (FDA). In 2011, 35 new molecular entities were approved, one of the highest totals in the last decade. Here are just a few examples:

  • Cancer: Two new personalized medicines for lung cancer and melanoma now provide effective options for patients with tumors expressing certain genetic markers. [xiii] The personalized melanoma treatment and another new melanoma medicine became the first new approvals for the disease in 13 years.
  • Rare Diseases and Orphan Indications: Eleven new medicines were made available to patients for rare diseases such as the genetic defect congenital factor XIII deficiency, several cancers, and scorpion poisoning.[xiv] 
  • Lupus: The first new medicine for lupus since 1955 takes a new approach to treating this serious and potentially fatal autoimmune disease.[xv
  • Hepatitis C: Two new medicines approved last year are the first in a new class and offer a greater chance of cure for some patients, compared with existing therapies.[xvi

Bringing Hope to Patients with Rare Diseases
Much attention is focused on the most common, well-known diseases, but scientific advances have also led to innovative medicines for rare conditions. Although each rare disease affects a relatively small number of people—200,000 or fewer in the United States—their cumulative effect is significant.[xvii]  More than 6,000 rare diseases are known, and they affect 25 million Americans.[xviii] Many of these diseases are serious or life threatening, and often no treatment options exist, so development of new medicines for these diseases is particularly important.

A recent study by researchers at the FDA’s Office of Orphan Products Development found that between 1983—when the U.S. Orphan Drug Act was passed—and 2008, 326 orphan drugs received marketing approval, representing new treatment options for more than 200 rare diseases.[xix] Since the mid-1990s there has been a near tripling in the annual number of orphan drug designations for drugs in development, from 57 in 1996 to 165 in 2008. Orphan drugs also represent a growing proportion of FDA approvals, accounting for 30% in the most recent five-year period. There has been great progress in the fight against rare diseases, but many patients still lack treatment options. Today researchers are working to meet those needs, with 460 medicines for orphan diseases currently in the development pipeline. [xx] 

[i] V.L. Roger, et al., “Heart Disease and Stroke Statistics—2011 Update: A Report from the American Heart Association,” Circulation 123, no. 4 (2011): e18-e209.


[ii] K.A. Fox, et al., “Decline in Rates of Death and Heart Failure in Acute Coronary Syndromes, 1999–2006,” Journal of the American Medical Association 297, no. 17 (2007): 1892–1900.


[iii] PharMetrics, Examination of Treatment Patterns and Effects of Medication-Taking Behaviors Among Patients with Diabetes, (Watertown, MA: PharMetrics, 2004) (research supported by PhRMA).

[iv] U.S. Department of Health and Human Services, Centers for Disease Control and Prevention, National Center for Health Statistics, Health, United States, 2003: With Chartbook on Trends in the Health of Americans (Hyattsville, MD: HHS, 2003); S.L. Murphy, J. Xu, and K.D. Kochanek, “Deaths: Preliminary Data for 2010,” National Vital Statistics Reports 60, no. 4 (Hyattsville, MD: National Center for Health Statistics, January 2012): 17 (accessed 10 March 2012).

[v] S.L. Murphy, J. Xu, and K.D. Kochanek, “Deaths: Preliminary Data for 2010,” National Vital Statistics Reports 60, no. 4 (Hyattsville, MD: National Center for Health Statistics, January 2012): 17 (accessed 10 March 2012).

[vi] U.S. Department of Health and Human Services, Centers for Disease Control and Prevention, National Center for Health Statistics, Health United States, 2010: With Special Feature on Death and Dying, (Hyattsville, MD: NCHS, 2011)

[vii] National Cancer Institute, Surveillance Epidemiology and End Results, (accessed 16 December 2011).

[viii] A. Jemal, et al., “Cancer Statistics, 2010,” CA: A Cancer Journal for Clinicians 60, no. 5 (2010): 277–300, (accessed 6 December 2011).


[ix] J.M. Kremer, “COMET’s Path, and the New Biologicals in Rheumatoid Arthritis,” The Lancet 372, No. 9636 (2008): 347–348.


[x] Integrated Benefits Institute, A Broader Reach for Pharmacy Plan Design (San Francisco: IBI, May 2007).


[xi] J.J. Caro, et al., “The Impact of Compliance with Osteoporosis Therapy on Fracture Rates in Actual Practice,” Osteoporosis International 15, no. 12 (2004): 1003–1008.


[xii] U.S. Department of Health and Human Services, Office of the Surgeon General, The 2004 Surgeon General’s Report on Bone Health and Osteoporosis: What It Means To You (Washington, DC: HHS, 2004), (accessed 5 December 2011).


[xiii] U.S. Food and Drug Administration, FY2011 Innovative Drug Approvals (Washington, DC: FDA, November 2011),


[xiv] J.K. Jenkins, “CDER New Drug Review: 2011 Update,” presentation at the FDA/CMS Summit (Washington, DC), 8 December 2011,



[xv] Food and Drug Administration, “FDA Approves Benlysta to Treat Lupus,” press release, 9 March 2011, (accessed 29 January 2012).


[xvi] U.S. Food and Drug Administration, “Notable FY 2011 Approvals,” 15 November 2011, (accessed 29 January 2012).


[xvii] National Institutes of Health, Office of Rare Diseases Research, Office of Rare Diseases Research (ORDR) Brochure, online brochure, 11 March 2009, (accessed 5 December 2011).


[xviii] Ibid.

[xix] M.M. Braun, et al., “Emergence of Orphan Drugs in the United States: a Quantitative Assessment of the First 25 Years.” Nature Reviews Drug Discovery 9, no. 7 (2010) 519–522.


[xx] Pharmaceutical Research and Manufacturers of America, Orphan Drugs in Development for Rare Diseases 2011 (Washington, DC: PhRMA, 2011),


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