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National Academies of Sciences, Engineering, and Medicine; Health and Medicine Division; Board on Health Sciences Policy; Committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union; Shore CK, Worku TL, Smith CW, et al., editors. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington (DC): National Academies Press (US); 2024 Oct 30.
Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities.
Show details- AACC
Accelerated Approval Coordinating Council
- AAV
adeno-associated virus
- ACD
alternative and confirmatory data
- AIDS
acquired immunodeficiency syndrome
- ARC
Accelerating Rare Disease Cures program
- ATMP
Advanced Therapy Medical Products
- BLA
biologics license application
- BPCA
Best Pharmaceuticals for Children Act
- CBER
Center for Biologics Evaluation and Research
- CDER
Center for Drug Evaluation and Research
- CDRH
Center for Devices and Radiological Health
- CHMP
Committee for Medicinal Products for Human Use
- CID
Complex Innovative Trial Design
- CIRS
Centre for Innovation in Regulatory Science
- CMS
congenital myasthenic syndrome
- CoGenT
Collaboration on Gene Therapies Global Pilot
- COMP
Committee for Orphan Medicinal Products
- CTIS
Clinical Trial Information System
- CTTI
Clinical Trials Transformation Initiative
- DMD
Duchenne muscular dystrophy
- EC
European Commission
- ECD
Erdheim-Chester disease
- eCTD
electronic common technical document
- EMA
European Medicines Agency
- EPAR
European public assessment report
- ERG
Eastern Research Group
- EU
European Union
- EUA
emergency use authorization
- EU-IN
EU Innovation Network
- FA
Friedreich’s ataxia
- FD&C Act
Federal Food, Drug and Cosmetic Act
- FDA
U.S. Food and Drug Administration
- FDARA
Food and Drug Administration Reauthorization Act
- FDASIA
Food and Drug Administration Safety and Innovation Act
- FDORA
Food and Drug Omnibus Reform Act
- GAO
U.S. Government Accountability Office
- GD1
Gaucher disease type 1
- HC
Health Canada
- HHS
U.S. Department of Health and Human Services
- HIPAA
Health Insurance Portability and Accountability Act
- HIV
human immunodeficiency virus
- ICD
International Classification of Diseases
- ICH
International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use
- IND
investigational new drug
- iPSP
initial pediatric study plan
- IRB
institutional review board
- ITF
Innovation Task Force
- LADDER
Linking Angelman and Dup15q Data for Expanded Research
- LEADER 3D
Learning and Education to Advance and Empower Rare Disease Drug Developers
- MAA
Marketing Authorization Application
- MDRI
multi-domain responder index
- MHLW
Ministry of Health, Labour and Welfare of Japan
- MIDD
Model-Informed Drug Development Paired Meeting Program
- NAS
new active substance
- NCATS
National Center for Advancing Translational Sciences
- NDA
new drug application
- NIH
National Institutes of Health
- NMA
network meta-analysis
- NME
new molecular entity
- NORD
National Organization for Rare Disorders
- OBRR
Office of Blood Research and Review
- OCE
Oncology Center of Excellence
- ODD
orphan drug designation
- OMP
orphan medicinal product
- OND
Office of New Drugs
- OOPD
Office of Orphan Products Development
- OTP
Office of Therapeutic Products
- OVRR
Office of Vaccines Research and Review
- PCWP
Patients’ and Consumers’ Working Party
- PDCO
Paediatric Committee
- PDUFA
Prescription Drug User Fee Act
- PEC
Patient Engagement Collaborative
- PED
patient experience data
- PFDD
patient-focused drug development
- PIP
paediatric investigational plan
- PMDA
Pharmaceuticals and Medical Devices Agency
- PREA
Pediatric Research Equity Act
- PRIME
Priority Medicines program
- PRO
patient-reported outcome
- PRV
priority review voucher
- PSA
Parallel Scientific Advice
- QSP
quantitative systems pharmacology
- RACE Act
Research to Accelerate Cures and Equity for children Act
- RBI
randomization-based inference
- RCT
randomized controlled trial
- RDCA-DAP®
Rare Disease Cures Accelerator-Data and Analytics Platform
- RDEA
Rare Disease Endpoint Advancement Pilot Program
- RMAT
regenerative medicine advanced therapy
- RPM
regulatory project manager
- RTOR
real-time oncology review
- RWD
real-world data
- RWE
real-world evidence
- SMA
spinal muscular atrophy
- SME
small or medium-sized enterprise
- SMN
survival motor neuron 1 (gene)
- START
Support for clinical Trials Advancing Rare disease Therapeutics
- TGA
Therapeutic Goods Administration
- Acronyms and Abbreviations - Regulatory Processes for Rare Disease Drugs in the ...Acronyms and Abbreviations - Regulatory Processes for Rare Disease Drugs in the United States and European Union
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