Policy and
Public Affairs

Resources

Articles
The FDA could help save my son from a rare disease
Articles
Too many treatable diseases go unnoticed. This could change that.
Articles
"Bootcamp" helps rare disease advocates learn the ropes of drug development
Articles
The FDA is at a crossroads on cell and gene therapies
Articles
Aduhelm's accelerated approval offers a promising roadmap for rare neurological diseases
Articles
Three misconceptions about the accelerated drug approval pathway
Articles
My son’s time is running out due to a rare disease. The FDA needs to add more clinical trial flexibility
Articles
Gene therapy is in crisis. For nine hours, the field’s leading minds looked for a solution
Articles
‘We’ve exhausted all avenues’: A small biotech may give up on its ultra-rare disease drug over frustration with FDA
Articles
Rare-disease doctors support expanded newborn genomic screening, survey finds
Articles
"No magic bullet": For drugmakers and the FDA, clinical trials on ultra-rare diseases pose thorny challenges
Articles
My granddaughter’s devastating rare disease deserves attention, too
Articles
The FDA is failing children with a rare, neglected disease sometimes called "childhood dementia"
Articles
Compassion or Cop-out?
Articles
Does the orphan drug act need a tune-up?
Articles
Accelerated approval in the US: It really is rare outside of oncology
Articles
Impasse at US FDA could mean stealth abandons Barth syndrome treatment
Articles
Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints
Articles
The future of medicine is unfolding before us. Are we nurturing It?
Articles
Not too rare to care: The Barth syndrome foundation delivers petition to FDA advocating for a fair, equitable and appropriate review of the only potential treatment for Barth syndrome
Articles
Sheffield family plead for help after drug trial for boy, 11, is abruptly stopped
Articles
Patient group pushes FDA to take action on drug
Articles
Accelerated approval will be 'the norm' for gene therapies, FDA's Peter Marks says
Articles
The transformation of drug development for the 21st century: Time for a change
Articles
It takes an average of 8 years for a rare disease patient to get diagnosed. Why is it so hard to get life-altering genetic testing in the U.S.?
Articles
Rare disease trials can't find enough patients. It's forcing the FDA to rethink its approach.
Articles
The burden of rare diseases: An economic evaluation
Articles
Accelerating a path to new treatments for rare neuronopathic MPS diseases
Articles
Tiny biotech says its troubles make case for ultra-rare FDA pathway
Articles
Hopes, fears for orphan drug act
Articles
A new paradigm for ultra-rare diseases
Articles
Drug providing hope for Arizona toddler with rare genetic disorder
Articles
Ultrarare inflection point: stakeholders argue for biomarker-based approvals
Patient Advocacy Groups
Global genes
Patient Advocacy Groups
NORD
Patient Advocacy Groups
Everylife foundation for rare diseases
Industry and trade associations
Institute for gene therapies
Industry and trade associations
ASGCT
Industry and trade associations
Alliance for regenerative medicine
Industry and trade associations
Rare disease company coalition
Industry and trade associations
Mass BIO
Industry and trade associations
California life sciences
Industry and trade associations
BIO