FDA Marks Orphan Drug Act Milestone:

30 Year RecognitionJanuary 2013

One of Many Rare Disease Heroes…

Henry Waxman and Orrin Hatch

Author and cosponsors of the Orphan Drug Act (ODA)

One of Many Rare Disease Heroes…

Abbey MeyersChampion for ODA and founder of

National Organization for Rare Disorders

One of Many Rare Disease Heroes…

Media/Entertainment

IndustryRaising awareness about rare diseases

and the need for medical products

One of Many Rare Disease Heroes…

Rare Disease Advocacy Groups

Organization, education, support, research, advocacy, and action

One of Many Rare Disease Heroes…

FDA Orphan Incentive Programs

Advancing orphan products through orphan designations, grants, and

facilitation

One of Many Rare Disease Heroes…

NIH Office of Rare Diseases

ResearchAdvancing rare disease information,

collaboration, and research

One of Many Rare Disease Heroes…

Robert Campbell Jr, MD

representing Clinician/product developers and advocates;Developed the artificial rib humanitarian use

device

One of Many Rare Disease Heroes…

Hemin and Desiree Lyon

representing1st approved orphan designated drug

and patient advocacy

One of Many Rare Disease Heroes…

Jana Monacorepresenting

Parent advocacy for newborn screening and medical foods

One of Many Rare Disease Heroes…

Industry-Investors-Academia

Developing products in the spirit of the ODA

One of Many Rare Disease Heroes…

Sharon Terryrepresenting

Advocacy for innovative and collaborative approaches for rare disease products

One of Many Rare Disease Heroes…

NIH Undiagnosed Diseases Program

Cross-cutting efforts to understand and treat rare diseases

One of Many Rare Disease Heroes…

Nancy Wexler, PhD

representingPatient advocacy and gene

discovery/research

One of Many Rare Disease Heroes…

Ashley Appellrepresenting

Patients living and thriving with a rare disease

One of Many Rare Disease Heroes…

FDA Offices/Centers

Communication, diagnostic and therapeutic product evaluation, safety, and access

One of Many Rare Disease Heroes…

Myozyme and Tiffany

Houserepresenting

Contributions of patient representatives to the FDA orphan approval process

One of Many Rare Disease Heroes…

Ronald J. Bartekrepresenting

Family member founder of a rare disease advocacy group

One of Many Rare Disease Heroes…

Pediatric Ventricular Assist Device-Berlin

Heartrepresenting

ODA Grants and Humanitarian Use Devices for pediatric rare diseases

One of Many Rare Disease Heroes…

Denise Ney, PhDrepresenting

Clinical dietitian/medical foods researcher for metabolic disorders

One of Many Rare Disease Heroes…

Hal Dietz, MDrepresenting

Clinical research in rare diseases

One of Many Rare Disease Heroes…

FDA/CDER Rare Diseases Program

Focused regulatory science and education efforts for rare disease drug

development

One of Many Rare Disease Heroes…

NIH TRND Program

Stimulating drug discovery and collaborative preclinical development for rare disease

drugs

One of Many Rare Disease Heroes…

EMA/EURORDIS/IRDIRC/ICORD

representingCollaborative international rare disease

efforts

One of Many Rare Disease Heroes…

Pearl O’Rourke, MD

representingIRB efforts on behalf of rare diseases

One of Many Rare Disease Heroes…

Jouni Uitto, MD, PhD

representingBasic research in rare diseases

One of Many Rare Disease Heroes…

George Harris Carterrepresenting

Patient advocacy for drug development and clinical trials

One of Many Rare Disease Heroes…

Kalydecorepresenting

Personalized medicines for rare diseases

One of Many Rare Disease Heroes…

Leslie Boyer, MDrepresenting

Academic grantees successfully pursuing biological marketing approvals

One of Many Rare Disease Heroes…

Rodney Howell, MD

representingAdvocacy for newborn screening/early detection and treatment of congenital

disorders

FDA Marks Orphan Drug Act Milestone:

30 Year RecognitionJanuary 2013