40 Years of Impact Driven by You
Before the Orphan Drug Act became law, fewer than 40 drugs were developed to treat rare diseases. Today, more than 600 treatments have been approved by the Federal Drug Administration (FDA) for rare diseases. The rare community has also made great strides in many other areas, including education, public policy, and patient/family services. As the community celebrates 40 years of progress, these milestones reflect the dedication and passion of the patients, caregivers, researchers, clinicians, and government and industry partners who have made this journey together.
Scroll to explore NORD’s 40-year history and major milestones that shaped the rare disease community:
1979
FDA/NIH Task Force Issues Report
Task Force, chaired by Marion Finkel, MD of FDA, issues report on need to address lack of treatments for diseases affecting small patient populations.
1979 - 1980
Patient Advocates Form Ad Hoc Coalition
Leaders of rare disease patient organizations form ad hoc coalition to advocate for orphan drug legislation.
1979 - 1980
House Subcommittee Gathers Evidence
House Subcommittee on Health and the Environment, chaired by Rep. Henry Waxman, holds hearings on the “orphan drug problem.”
1980
Popular TV Show Features the Issue
Actor Jack Klugman assists the patient advocates in focusing national attention on the problem with an episode of the TV show, Quincy, M.E.
1980
Pre-Orphan Drug Act
Only 10 new drugs were developed by the pharmaceutical industry for rare diseases in the decade before 1983.
January 4, 1983
Orphan Drug Act Enacted
The Orphan Drug Act is signed by President Reagan after being approved by the House and Senate in December 1982.
February 1983
First Orphan Drug Approved
FDA grants first marketing approval to an orphan drug for acute intermittent porphyria and other acute porphyrias.
May 4, 1983 NORD Milestone
NORD is Established
The ad hoc coalition of patient advocates launches NORD to continue their collaboration and Abbey Meyers is named president.
1984
Orphan Drug Act Amendment
The Orphan Drug Act is amended to define “rare” and the diseases that would qualify for orphan designation.
1984NORD Milestone
NORD Provides Expert-Reviewed Information to Patients & Families
NORD begins developing its Rare Disease Database, still widely used today, to provide expert-reviewed information in patient-friendly language.
1987NORD Milestone
NORD Establishes Patient Assistance Program
NORD launches its first patient assistance program to help patients access the care their physicians feel they should have.
1989
National Commission Issues Report
The National Commission on Orphan Diseases, chaired by NORD Medical Advisor Jess Thoene, MD, conducts major study of patient/family experience.
1989NORD Milestone
NORD Establishes Research Program
NORD launches Research Program to provide grants for study of rare diseases with little or no other funding.
1999
EU Adopts Orphan Law
European Union adopts rare disease legislation and NORD shares its experiences with patient leaders in Europe.
2000
NIH Establishes ClinicalTrials.gov
NIH launches a website (www.clinicaltrials.gov) to provide a central source of information on clinical trials.
2000NORD Milestone
NORD Leads Advocacy for Rare Diseases Act
Senators Ted Kennedy and Orrin Hatch introduce the Rare Diseases Act, advocated by NORD, to advance rare disease research and development.
2002
Rare Diseases Act Signed into Law
Rare Diseases Act is enacted, codifying NIH Office of Rare Diseases Research and establishing NIH Rare Diseases Clinical Research Network.
2003NORD Milestone
NORD Publishes Medical Textbook for Physicians
With Lippincott, Williams & Wilkins, NORD publishes The NORD Guide to Rare Disorders, with entries by 600+ leading medical experts.
2005
First Meeting of ICORD
The first meeting of ICORD takes place in Stockholm, with representatives of NIH, FDA, and NORD participating from the US.
2006
Rare Diseases Clinical Research Network Founded
NIH launches Rare Diseases Clinical Research Network with funding made possible by the Rare Diseases Act of 2002.
2007
FDA and EMA Collaboration
FDA and its counterpart in Europe, the European Medicines Agency, adopt a shared orphan designation application.
2008NORD Milestone
New NORD Leadership
NORD President Abbey Meyers retires after leading the organization for 25 years and is succeeded by Peter L. Saltonstall.
2008NORD Milestone
NIH Establishes Undiagnosed Diseases Program
NIH launches a program for undiagnosed patients and NORD later establishes a program to pay for diagnostic testing for applicants.
2008
Compassionate Allowances Program Established
Social Security Commissioner Michael Astrue announces the “Compassionate Allowances Program” at a NORD patient/ family conference.
2009NORD Milestone
NORD and EURORDIS Formalize Partnership
NORD and EURORDIS (Rare Diseases Europe) form a strategic partnership to collaborate in shared initiatives.
2009NORD Milestone
NORD Launches Rare Disease Day in US
Through its partnership with EURORDIS, NORD introduces Rare Disease Day in the US.
2010NORD Milestone
Rare Disease Office Established in FDA CDER
NORD successfully advocates for a new FDA position, Associate Director for Rare Diseases, in the CDER Office of New Drugs.
2010
FDA Office of Orphan Products Development Offers Orphan Designation Workshops
FDA establishes one-day workshops for orphan designation applicants and NORD promotes awareness and participation.
2011NORD Milestone
NORD Hosts Medical Foods Conference
NORD hosts a conference on challenges patients encounter in access to medical foods and formulas.
2010
Affordable Care Act Signed
The Affordable Care Act, signed by President Obama, provides insurance reforms advocated by NORD and its policy partners.
2011NORD Milestone
NORD Releases Report on Regulatory Flexibility
NORD releases major new report written by board member Frank Sasinowski on flexibility in review of orphan drugs.
2011NORD Milestone
NORD Summit Connects Stakeholders
NORD and DIA co-sponsor the first US Conference on Rare Diseases and Orphan Products, later to become the annual NORD Summit.
2011NORD Milestone
NORD Hosts Forum with FDA Commissioner
NORD hosts the first-ever forum at which rare disease patient leaders sit down one-on-one with the FDA Commissioner.
2012
FDA Safety and Innovation Act Approved
The FDA Safety & Innovation Act is enacted with many key provisions for the rare disease community.
2014NORD Milestone
NORD Launches Natural History/Patient Registry Platform
NORD establishes a new platform for patient registries and natural history studies to advance knowledge, encourage research and support data-sharing.
2014NORD Milestone
With Rare Action Network® NORD Promotes State-Based Advocacy
NORD establishes the Rare Action Network to connect individuals within states which results in the engagement of more than 18,000 advocates nationwide.
2015
Rare Diseases International is Launched
NORD joins EURORDIS and other patient organizations around the world in this initiative to coordinate advocacy on a global level.
2015NORD Milestone
NORD Establishes Student Membership
NORD establishes a free membership for students that results in a network of clubs and chapters.
2015NORD Milestone
NORD Launches the Rare Disease State Report Card
The State Report Card is launched to measure effectiveness in key areas such as newborn screening across the country.
2015NORD Milestone
First Rare Disease Advisory Council is Established
The first Rare Disease Advisory Council is established in North Carolina to advise state officials on topics related to rare diseases.
2016NORD Milestone
NORD & FDA Enter Cooperative Agreement for Natural History Studies
NORD and FDA enter a cooperative agreement for natural history studies to be developed on NORD’s registry platform.
2017NORD Milestone
Rare Cancer Coalition is Launched
NORD’s Rare Cancer Coalition is launched to unite advocacy organizations serving those affected by rare cancers.
2017NORD Milestone
NORD Launches Continuing Medical Education Program
In partnership with PlatformQ, an online CME provider, NORD launches a Continuing Medical Education program.
2018
FDA Announces MOU with NORD to Co-Host Listening Sessions
FDA Commissioner Scott Gottlieb MD announces an MOU with NORD to co-host Listening Sessions for FDA staff and rare patients/caregivers.
October 1, 2018NORD Milestone
First Rare Cancer Day is Observed
NORD and the Rare Cancer Coalition establish and promote first Rare Cancer Day.
2019NORD Milestone
NORD Hosts First “Living Rare, Living Stronger Family Forum”
NORD hosts first “Living Rare, Living Stronger Family Forum” to continue its tradition of patient/family conferences.
2019NORD Milestone
NORD Joins FDA & C-Path in Accelerating Cures Initiative
NORD joins FDA & C-Path in the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) to encourage data collection, analysis and sharing.
2019NORD Milestone
NORD Launches Caregiver Respite Program
NORD establishes first-of-its-kind Caregiver Respite Program to support needs of rare caregivers.
2019NORD Milestone
NORD Launches Rare Disease Video Library
In partnership with Osmosis, a leading provider of video medical education, NORD launches Rare Disease Video Library.
2020NORD Milestone
NORD Provides COVID Assistance
Through two programs, one for patients and one for patient organizations, NORD provides financial assistance in coping with COVID-19.
2020NORD Milestone
NORD Issues Barriers to Diagnosis & Care Analysis
NORD releases a major report, “Barriers to Rare Disease Diagnosis, Care and Treatment in the US: A Comparative Analysis.”
2020NORD Milestone
NORD Website & Rare Disease Database Hit Record High
NORD web traffic hits record high – 2 million visits a month – and most visitors go first to the Rare Disease Database.
2021NORD Milestone
NORD Joins the Bespoke Gene Therapy Consortium
NORD joins NIH and other partners in the Bespoke Gene Therapy Consortium to advance development of gene therapies.
2021NORD Milestone
NORD Announces 31 Rare Disease Centers of Excellence
To encourage collaboration, improve patient care and advance research, NORD establishes a network of centers of excellence.
October 2021NORD Milestone
NORD and Rare Disease Diversity Coalition Announce Partnership
NORD and Rare Disease Diversity Coalition conduct national survey of underrepresented rare disease patients and caregivers.
2022NORD Milestone
NORD Launches Its 50th Student Chapter
NORD announces its 50th student chapter in its program for students at universities and medical schools.
