Conquering Rare Diseases

January 24, 2020 • Volume 30, Issue 4
Should taxpayers keep paying to develop drugs for unusual disorders?
By Sara Toth Stub


More than 30 million Americans suffer from one of 7,000 rare diseases — incurable, often genetic conditions affecting fewer than 200,000 people. There is no treatment for 95 percent of these diseases, in part because pharmaceutical companies have been reluctant to invest in developing drugs for such small potential markets. That began to change after Congress in 1983 created tax incentives and exclusive marketing rights for companies that develop treatments for rare diseases. Since then, more than 700 drugs targeting such conditions have been approved, and about half of all new pharmaceuticals approved in 2018 were for rare diseases. While these treatments, including expensive new gene therapies, save lives and offer patients hope, they often come with high price tags: One drug, a one-time treatment, costs more than $2 million. Some experts and policymakers question whether taxpayers need to continue subsidizing the development of such drugs. Meanwhile, patient advocacy organizations are investing in drug research, participating in clinical trials, sharing data and lobbying for continued government support and investment in treatments for rare diseases.

Dr. Michael Keller, an immunologist at Children's National Hospital in Washington (AFP/Getty Images/Eva Hambach)
Dr. Michael Keller, an immunologist at Children's National Hospital in Washington, examines a T-cell research sample as he explains a new treatment for a rare disorder, chronic granulomatous disease. The treatment is one of a new type of drug that is tailored for the individual patient. (AFP/Getty Images/Eva Hambach)
ISSUE TRACKER for Related Reports
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Jan. 08, 2021  Health and Society
Nov. 20, 2020  The Public Health System
Jul. 17, 2020  The Pandemic Economy
Jun. 26, 2020  Zoonotic Diseases
May 08, 2020  CTE and Athletes
Jan. 24, 2020  Conquering Rare Diseases
Sep. 13, 2019  Measles Resurgence
Nov. 30, 2018  Obesity Crisis
Jun. 15, 2018  Superbug Threat
Jun. 02, 2017  Pandemic Threat
Jul. 22, 2016  Mosquito-Borne Disease
Feb. 13, 2015  Emerging Infectious Diseases
Nov. 08, 2013  Lyme Disease
Jan. 06, 2012  Preventing Disease
Apr. 02, 2010  Breast Cancer
Sep. 12, 2008  Heart Health
Aug. 24, 2007  Fighting Superbugs
Jan. 13, 2006  Avian Flu Threat
Jun. 20, 2003  Fighting SARS
Apr. 05, 2002  Chronic Fatigue Syndrome
Mar. 09, 2001  Diabetes Epidemic
Mar. 02, 2001  Mad Cow Disease
Dec. 24, 1999  Asthma Epidemic
Aug. 05, 1983  Multiple Sclerosis
May 27, 1983  Chronic Pain: The Hidden Epidemic
Sep. 24, 1976  Influenza Control
Sep. 16, 1970  Virus Research
Mar. 14, 1956  Progress Against Polio
May 25, 1955  Degenerative Diseases
May 25, 1949  Chronic Disease
Mar. 01, 1924  The Foot and Mouth Disease
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