{"id":100989,"date":"2023-01-04T09:35:10","date_gmt":"2023-01-04T14:35:10","guid":{"rendered":"https:\/\/rarediseases.org\/?p=100989"},"modified":"2023-02-09T14:57:31","modified_gmt":"2023-02-09T19:57:31","slug":"the-orphan-drug-act-turns-40-nord-celebrates-its-impact-on-rare-diseases","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/the-orphan-drug-act-turns-40-nord-celebrates-its-impact-on-rare-diseases\/","title":{"rendered":"The Orphan Drug Act Turns 40: NORD Celebrates Its Impact on Rare Diseases"},"content":{"rendered":"

Individuals suffering with rare diseases had very few therapeutic options 40 years ago.<\/span>\u00a0<\/span><\/p>\n

But that began to change with the stroke of a pen by then-President Ronald Reagan in January 1983 when he signed the bipartisan Orphan Drug Act into law.<\/span>\u00a0<\/span><\/p>\n

On Jan. 4, the rare disease community will celebrate the 40<\/span>th<\/span> anniversary of the Orphan Drug Act (ODA) \u2014 legislation that incentivized drug companies to put more resources toward the research, development, and distribution of therapeutics for people with rare diseases, who until then had been \u2018orphaned\u2019 by the medical and scientific community.<\/span>\u00a0<\/span><\/p>\n

\"\"<\/p>\n

The creation of the ODA and the founding of the National Organization for Rare Disorders (NORD) are forever intertwined. The rare patients and caregivers who started the rare advocacy movement \u2014 which convinced lawmakers to introduce orphan drug legislation \u2014 knew their work was not done after the bill became law. They formed NORD to unify the voices of the unheard within the rare disease community under the premise that a rising tide could lift all boats.\u00a0<\/span>\u00a0<\/span><\/p>\n

We are forever thankful to NORD\u2019s founders \u2014 Abbey Meyers, Ruby Horansky, Niss Ryan, Jung Chung, Sharon Dobkin, Melvin Van Woert, M.D., George Brewer, M.D., Ann Kone, Judy Rosner and Richard Vodra, among others \u2014 for their unwavering, tenacious activism and for all those that joined in with their voices to start the rare disease advocacy movement. It was their devotion to their loved ones, their courage to step outside of their comfort zones, and their determination to move mountains so that others could see hope ahead that have brought us here today.<\/span>\u00a0<\/span><\/p>\n

As we celebrate both NORD and the ODA\u2019s 40<\/span>th<\/span> anniversaries, it\u2019s fitting to recognize their impact on the rare disease community. <\/span>\u00a0<\/span><\/p>\n

The Origins of the Orphan Drug Act<\/span><\/b>\u00a0<\/span><\/p>\n

The impetus for what would become the Orphan Drug Act started in 1979 with a Food and Drug Administration (FDA) Task Force report calling for measures to address the \u201corphan drug problem,\u201d which the task force labeled a significant public health issue \u2014 namely that drugs for small patient populations were not being developed.<\/span>\u00a0<\/span><\/p>\n

The report coincided with a bipartisan effort by lawmakers \u2014 led by U.S. Rep. Henry Waxman of California \u2014 to address this issue after learning about it from a constituent.<\/span>\u00a0<\/span><\/p>\n

Waxman, then the chair of the Subcommittee on Health and the Environment of the House Energy and Commerce Committee, held hearings and gathered evidence of the problem before drafting legislation \u2014the Orphan Drug Act \u2014 to create financial incentives designed to encourage the development of treatments for those with rare diseases.\u00a0<\/span>\u00a0<\/span><\/p>\n

Waxman served as the bill\u2019s primary House sponsor. Kansas Sen. Nancy Landon Kassebaum introduced it in the Senate.<\/span>\u00a0<\/span><\/p>\n

\u201cFor those with rare diseases, this is a tragic, and sometimes hopeless, situation,\u201d Rep. Waxman said in his opening statement for the Orphan Drug Act hearings in 1982. \u201cFor the rest of us who believe that people with rare diseases suffer no less and are no less deserving because they are so few, this situation is intolerable. It must be changed.\u201d<\/span><\/i><\/b>\u00a0<\/span><\/p>\n

Adding to the noise surrounding the Orphan Drug Act were the voices of Maurice and Jack Klugman, brothers who respectively starred in and produced a then-popular television show called Quincy, M.E. Maurice learned of the orphan drug problem after reading an article in the L.A. Times from a reporter who attended one of the hearings. He too had a rare condition and was compelled to help shine a light nationally. The show aired multiple episodes featuring the plight of people searching for unknown answers and calling for Congress to intervene and help solve the orphan drug problem. Jack Klugman later testified in front of Congress, which at the time was unheard of for a celebrity. The recognition it brought to the issue, along with the tireless efforts of parents, patients and lawmakers, helped to push the Orphan Drug Act over the finish line.<\/span>\u00a0<\/span><\/p>\n

\"Rows<\/a>Actor Jack Klugman and his brother, Maurice, assisted rare disease patient advocates in focusing national attention on the issues through a 1980 episode of the television show, Quincy, M.E.<\/span><\/div>\n

The bill was approved by the House on Dec. 14, 1982, and by the Senate three days later. President Reagan signed the bill into law on Jan. 4, 1983.<\/span>\u00a0<\/span><\/p>\n

40 Years of Action and Activism<\/span><\/b>\u00a0<\/span><\/p>\n

Since 1983, the Orphan Drug Act has paved the way for four decades of innovation and change and NORD has helped strengthen the rare disease community, which today numbers more than 25 million Americans.<\/span>\u00a0<\/span><\/p>\n

In 1989, the National Commission on Orphan Diseases published a pivotal report, based on a NORD-led study conducted in 1988, documenting the continued need for a national effort to address issues such as the delay in obtaining an accurate diagnosis, the financial hardship of having a rare disease on patients and their families, and the lack of access to therapies.\u202f<\/span>\u00a0<\/span><\/p>\n

That report, in turn, helped spur the creation of the National Institutes of Health (NIH) Office of Rare Diseases Research (ORDR), which has played a pivotal role in connecting patient advocacy organizations with the NIH \u2014 the primary government funder of medical research in the U.S.\u00a0<\/span>\u00a0<\/span><\/p>\n

The NIH Office of Rare Diseases Research has also provided funding for numerous disease-specific conferences, in partnership with patient advocacy organizations, and played a key role in assuring that patient advocacy groups would be included as partners with researchers in the NIH Rare Diseases Clinical Research Network.<\/span>\u00a0<\/span><\/p>\n

The unrelenting advocacy of NORD and\u00a0 incredible rare disease community has been instrumental to the gains and milestones made possible over the last 40 years.\u00a0 A few of our community\u2019s many notable wins:<\/span>\u00a0<\/span><\/p>\n