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Jan. 3, 2018

TOPIC: Press Releases

NORD and the Orphan Drug Act Celebrate 35th Anniversaries

Posted by Jennifer Huron

January 4th Marks 35 Years of Helping People with Rare Diseases and the Beginning of a New, Year-Long Education and Awareness Campaign

national organization for rare disorders 35th anniversary logoThis year marks the 35th anniversaries of both the Orphan Drug Act, a law that provided incentives for the development of drugs for patients with rare diseases, and the formation of the National Organization for Rare Disorders (NORD), the organization that is the leading advocate for patients with rare diseases.

NORD today launched a year-long commemoration of the two anniversaries along with a renewal of the national commitment to medical research and programs to advance health care for the 30 million Americans with a rare disease.

“The advances in diagnosis, treatments and care for patients with rare diseases have been remarkable, but we still have many challenges ahead of us,” said Peter L. Saltonstall, President and CEO of NORD.  “The Orphan Drug Act and the creation of NORD brought national attention to rare diseases. The joint anniversaries are an appropriate time for us to recognize the progress made and rededicate ourselves to the needs of the patient community.”

“There are 30 million Americans with rare diseases—one-tenth of the population—making it an issue that touches nearly every U.S. household in one way or another,” Saltonstall added. “But of the 7,000 diseases that are defined as rare, less than 500 have an approved treatment.  We have a long way to go.”

To get the law passed, an ad hoc coalition of parents of children with rare diseases worked with the Congress and the medical/pharmaceutical communities to call attention to rare diseases.  The parents organized and gave themselves a name, the National Organization for Rare Disorders (NORD).  They taught themselves how to be advocates for the desperate patients and families they represented.  When passed, the new law represented a triumph of patient advocates working with government partners, the media, and other supporters to address a critically important unmet need.

Exactly four months after President Reagan signed the Orphan Drug Act into law, on May 4, 1983, the coalition of patient advocates formally established NORD as a nonprofit organization to provide advocacy, education, research and patient/family services for all Americans affected by rare diseases.  For the first time, people with a medical condition that had been “orphaned” by the medical community had a home and place to connect with others.

NORD’s year-long observance of the 35th anniversary will include a special section on its website with information and stories about rare disease milestones and achievements. Rare Disease Day® 2018 (Feb. 28th), which NORD sponsors nationally, will offer special activities to honor the history of the rare disease community.  There will be additional announcements for new activities, including white papers and other publications, throughout the year.

In addition, NORD will host its annual Rare Impact Awards ceremony on May 17 at the Andrew Mellon Auditorium in Washington, D.C. The event will be a celebration of NORD’s 35th anniversary, as well as the 35th anniversary of the Orphan Drug Act. The event will highlight more than three decades of work to fulfill NORD’s mission.
Saltonstall continued, “The Orphan Drug Act is just as important today as it was in 1983. This is particularly pertinent since scientific and medical momentum is spurring the development of innovative, safe, and effective treatments for children and adults with very challenging medical conditions. This year, we will be saluting the progress made to date, we will also be focusing on the fact that much remains to be done.”

From 1967 to 1983, only 34 drugs approved by the Food and Drug Administration (FDA) were for rare diseases, and only 10 of the products brought to market by the pharmaceutical industry in the decade before 1983 would have qualified under today’s Orphan Drug Act as orphan drugs. Since the Orphan Drug Act has been law, thousands more potential treatments have entered the research pipeline as orphan products, more than 600 have been approved by FDA, and one-third of all new treatments approved in recent years are to treat rare diseases.

“Many people are alive today because of these treatments and people are able to live with their rare diseases because of the drugs that have been developed,” Saltonstall said.

Highlights since the Orphan Drug Act was enacted include:

  • As of December 2017, FDA had approved more than 600 orphan products and 4,000 orphan drug designations since 1983.
  • The Orphan Drug Act has been credited with helping to drive innovation in cancer treatment, gene therapies, and has resulted in life-saving enzyme replacement therapies for children and adults with metabolic diseases for which there was previously no treatment.
  • People with rare diseases have new hope that medical researchers one day will develop a treatment for their disease, and hundreds of patient organizations have come into existence to advocate on behalf of the patient communities.

The Orphan Drug Act works by providing incentives to the pharmaceutical industry to invest in developing therapies to help small patient populations.  The incentives include:

  • Market exclusivity: 7-year market exclusivity for approved orphan drugs or products for the orphan indication;
  • Tax incentives: Companies have a tax incentive to invest in research as they can benefit from tax credits (Orphan Drug Tax Credit) for expenses occurred prior to the orphan designation;
  • Clinical research subsidies: Orphan Product Grant program provides funding for clinical testing of new therapies to treat and/or diagnose rare diseases;
  • User fee exemptions:  Orphan drugs and products are exempt from the usual new drug application or “user” fees charged by FDA.

Recent milestones from NORD’s 35-year history include:

  • Named a 4-star charity by Charity Navigator, one of the 100 Best Charities by Worth Magazine, and “Charity of the Week” by The Week Magazine;
  • In at least two instances, NORD’s rare disease research grant program has resulted in FDA-approved orphan therapies;
  • NORD established a Natural History Study program and entered into a cooperative agreement with FDA to further rare disease research;
  • NORD established a Rare Action Network™ to promote state advocacy;
  • Rarediseases.org, NORD’s main website, reached the milestone of 1 million monthly visitors.

Read all of NORD’s news here.

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