Orphan medicinal products
Orphan medicinal products are intended for the analysis, prevention or treatment of dangerous or very serious conditions that affect no more than 5 in 10,000 people in the European Union. Patients suffering from rare diseases deserve the same quality of treatment as other patients within the European Union. Given the small numbers of patients affected by rare diseases, the pharmaceutical industry has been unwilling in the past to invest in the research and development of medicinal products to treat them. The EU introduced new legislation in 2000 with the aim of providing incentives for the development of medicines for rare diseases (so-called orphan medicinal products).
- Orphan designation
- Medicines for rare diseases
Related Conference of Orphan medicinal products
January 28-29, 2026
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Orphan medicinal products Conference Speakers
Recommended Sessions
- Allergic and immunologic disorders
- Challenges in rare disease development
- Clinical trials and treatment
- Gene therapy for rare diseases
- Infectious diseases
- Orphan Diseases and public health
- Orphan drugs potentiality
- Orphan medicinal products
- Rare diseases in blood stream
- Rare infectious diseases and immune deficiencies
- Rare mental and behavioral diseases
- Rare neurological diseases
- Rare nutritional and metabolic diseases
- Rare pediatric diseases
- Treatment and advanced therapies for rare diseases
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- Pharmaceutical Industry - Pharmatech 2026 (USA)
- Pharmaceutical Industry - Pharmatech 2026 (USA)
- Pharmaceutical Manufacturing - PHARMACEUTICA 2026 (UK)
- Pharmaceutical Manufacturing 4.0 - Pharmaceuticalindustry-2026 (Netherlands)
- Pharmaceutical Manufacturing and Pharmacoeconomics - MedTech-2026 (Switzerland)
- Pharmaceutical Manufacturing, Scale Up and Tech transfer - MedTech-2026 (Switzerland)
- Pharmaceutical Microbiology - Pharma Europe 2026 (UK)
- Pharmaceutical Nanotechnology - Pharma Europe 2026 (UK)
- Pharmaceutical Nanotechnology - PHARMACEUTICAL SCIENCES 2026 (Italy)
- Pharmaceutical Nanotechnology - PHARMACEUTICA 2026 (UK)
- Pharmaceutical Packaging & Smart Labeling - Pharmaceuticalindustry-2026 (Netherlands)
- Pharmaceutical Packaging and Logistics - MedTech-2026 (Switzerland)
- Pharmaceutical Preformulation Studies - Drug-Delivery-2026 (France)
- Pharmaceutical Process Chemistry - PHARAMACEUTICAL CONF 2026 (France)
- Pharmaceutical Process Validation - PHARMACEUTICAL SCIENCES 2026 (Italy)
- Pharmaceutical Quality Assurance and GMP - Pharma Expo-2026 (Thailand)
- Pharmaceutical Research and Development - Pharmatech 2026 (USA)
- Pharmaceutical Sciences - Pharma Europe 2026 (UK)
- Pharmaceutical sciences - PHARMACEUTICAL SCIENCES 2026 (Italy)
- Pharmaceutical Sciences - MedTech-2026 (Switzerland)
- Pharmaceutical Supply Chain & Logistics - Pharma Expo-2026 (Thailand)
- Pharmaceutical Supply Chain Optimization - PHARMACEUTICAL SCIENCES 2026 (Italy)
- Pharmaceutical Supply Chain Resilience & Risk Management - Pharmaceuticalindustry-2026 (Netherlands)
- Pharmaceutics - Pharma Europe 2026 (UK)
- Pharmaceutics and Pharmacotherapeutics - Pharmatech 2026 (USA)
- Pharmacists Role in Patient Safety - Pharmatech 2026 (USA)
- Pharmacists Role in Patient Safety - Formulations 2026 (UK)
- Pharmacoeconomics - MedTech-2026 (Switzerland)
- Pharmacoepidemiology & Real-World Evidence - Euro Pharmacology 2026 (France)
- Pharmacogenetics - Personalizedmedicine-2026 (Switzerland)
- Pharmacogenomics - Personalizedmedicine-2026 (Switzerland)
- Pharmacogenomics & Personalized Medicine - Biotech-2026 (France)
- Pharmacogenomics & Precision Medicine - Euro Pharmacology 2026 (France)
- Pharmacogenomics and Pharmacoepidemiology - MedTech-2026 (Switzerland)
- Pharmacogenomics in Cardiovascular Medicine - Cardiovascular-2026 (France)
- Pharmacognosy - Pharma Europe 2026 (UK)
- Pharmacokinetics & Pharmacodynamics (PK/PD) - Euro Pharmacology 2026 (France)
- Pharmacokinetics and Drug Absorption Optimization - Drug Devilvery 2026 (France)
- Pharmacokinetics and Pharmacodynamics - Drug-Delivery-2026 (France)
- Pharmacokinetics and Pharmacodynamics in Cardiology - Cardiovascular-2026 (France)
- Pharmacokinetics and Pharmacodynamics in Drugs - PHARMACEUTICA 2026 (UK)
- Pharmacological Approaches to Myocardial Ischemia - Cardiovascular-2026 (France)
- Pharmacological Management of Cardiovascular Risk Factors - Cardiovascular-2026 (France)
- Pharmacological Sciences - Pharma Europe 2026 (UK)
- Pharmacology - Pharmacology 2026 (Italy)
- Pharmacology - Pharmatech 2026 (USA)
- Pharmacology and Toxicology - MedTech-2026 (Switzerland)
- Pharmacovigilance & Biologic Drug Safety - PHARMA BIOTECH 2026 (UK)
- Pharmacovigilance & Drug Safety - Pharma Expo-2026 (Thailand)
- Pharmacovigilance & Drug Safety - Euro Pharmacology 2026 (France)
- Pharmacovigilance & Drug Safety Innovation - Pharmaceuticalindustry-2026 (Netherlands)
- Pharmacovigilance & Post-Marketing Safety - Biologics Meet 2026 (UK)
- Pharmacovigilance and Drug Safety - Clinical Research 2026 (UK)
- Pharmacovigilance and Risk Management - PHARMACOVIGILANCE 2026 (Italy)
- Pharmacovigilance in Clinical Trials - Clinical Trials Congress-2026 (France)
- Pharmacovigilance in Personalized and Precision Medicine - Clinical Trials-2026 (UK)
- Pharmacovigilance in Rare and Orphan Drugs - Clinical Trials-2026 (UK)
- Pharmacovigilance in Vaccines and Biologics - Clinical Trials-2026 (UK)
- Pharmacovigilance Significance & Scope - PHARMACOVIGILANCE 2026 (Italy)
- Pharmacovigilance Significance and Scope - Pharmacology 2026 (Italy)
- Pharmacy Development Services - Pharma Europe 2026 (UK)
- Pharmacy Education and Practice - PHARMACEUTICAL SCIENCES 2026 (Italy)
- Pharmacy Practices and its Challenges - PHARMACOVIGILANCE 2026 (Italy)
- Phases of Clinical Trials - Clinical Trials Congress-2026 (France)
- Phytochemicals in Drug Discovery - PHARAMACEUTICAL CONF 2026 (France)
- Phytochemistry - Naturalproducts 2026 (UAE)
- Placebo-Controlled Studies - Clinical Trials Congress-2026 (France)
- Polymer & Material Chemistry - PHARAMACEUTICAL CONF 2026 (France)
- Population Health - Personalizedmedicine-2026 (Switzerland)
- Post-Marketing Surveillance and Phase IV Trials - Clinical Trials Congress-2026 (France)
- Post-Marketing Surveillance and Real-World Data - Clinical Trials-2026 (UK)
- Pre-clinical and Clinical Trails - Pharmacology 2026 (Italy)
- Pre-Clinical and Clinical Trials - PHARMACOVIGILANCE 2026 (Italy)
- Precision Medicine - Personalizedmedicine-2026 (Switzerland)
- Precision Medicine and Biomarker Discovery - Clinical Trials Congress-2026 (France)
- Precision-Driven Chemical Innovation in Drug Development - PHARAMACEUTICAL CONF 2026 (France)
- Psychological and Behavioural Pain Interventions - Pain Management 2026 (France)
- Psychopharmacology & Behavioral Science - Euro Pharmacology 2026 (France)
- Public Health Approaches to Pain Management - Pain Management 2026 (France)
- Purpose and Principles of GMP - PHARMACEUTICAL SCIENCES 2026 (Italy)
- QC & QA: Quality control and Quality assurance - MedTech-2026 (Switzerland)
- Quality Assurance (QA) in Clinical Trials - Clinical Trials Congress-2026 (France)
- Quality Assurance, GMP & Biotech Manufacturing Excellence - PHARMA BIOTECH 2026 (UK)
- Quality Assurance, GMP & Data Integrity - Pharmaceuticalindustry-2026 (Netherlands)
- Quality by design (QbD) approach - MedTech-2026 (Switzerland)
- Quantum Chemistry and Molecular Simulations - Euro MedChem and CADD 2026 (France)
- Quantum Chemistry in Drug Design - PHARAMACEUTICAL CONF 2026 (France)
- R&D Advancement: Road to New Medicines - PHARMACEUTICAL SCIENCES 2026 (Italy)
- Radiopharmaceutical Chemistry - PHARAMACEUTICAL CONF 2026 (France)
- Randomization in Trials - Clinical Trials Congress-2026 (France)
- Rare Diseases & Orphan Drug Development - Pharmaceuticalindustry-2026 (Netherlands)
- Real-Time Safety Data Monitoring Strategies - Clinical Trials-2026 (UK)
- Real-World Evidence (RWE) and Real-World Data (RWD) - Clinical Trials Congress-2026 (France)
- Real-World Evidence in Clinical Trials - Pharma Europe 2026 (UK)
- Real-World Evidence in Clinical Trials - Clinical Research 2026 (UK)
- Recent Advances in Pharmacology and Toxicology - Pharmacology 2026 (Italy)
- Recent Innovations in Pharmaceutical Technology - Clinical Trials Congress-2026 (France)
- Regenerative Medicine & Stem Cell Applications - Biotech-2026 (France)
- Regenerative Therapies and Stem Cells - Pain Management 2026 (France)
- Regulatory Affairs & Drug Approval Pathways - Euro Pharmacology 2026 (France)
- Regulatory Affairs & Global Approval - Biologics Meet 2026 (UK)
- Regulatory Affairs and Quality Assurance - Drug-Delivery-2026 (France)
- Regulatory Affairs in a Rapidly Evolving Global Market - Pharmaceuticalindustry-2026 (Netherlands)
- Regulatory Authority Compliance - MedTech-2026 (Switzerland)
- Regulatory Compliance and Ethics - Clinical Trials Congress-2026 (France)
- Regulatory Harmonization & Compliance - Biologics Meet 2026 (UK)
- Regulatory Requirements for Pharmaceuticals - PHARMACEUTICAL SCIENCES 2026 (Italy)
- Regulatory Strategies & Quality Control in Biopharma - Biotech-2026 (France)
- Regulatory updates on Biosimilars - Eurobiosimilars 2026 (Italy)
- Regulatory, Quality Control, and Translational Aspects - Drug Devilvery 2026 (France)
- Rehabilitation and Physical Therapy in Pain - Pain Management 2026 (France)
- Risk-Based Monitoring (RBM) - Clinical Trials Congress-2026 (France)
- Risk-Based Monitoring and Trial Optimization - Clinical Trials-2026 (UK)
- Role of Gut Microbiome in Cardiovascular Health - Cardiovascular-2026 (France)
- Role of Inflammation in Cardiovascular Diseases - Cardiovascular-2026 (France)
- Safety Pharmacology - Pharmacology 2026 (Italy)
- Safety Reporting in Early Phase Trials - Clinical Trials-2026 (UK)
- Semi-Solid Dosage Forms - Formulations 2026 (UK)
- Signal Prioritization and Management Techniques - Clinical Trials-2026 (UK)
- Single-Use Technologies in Production - Biologics Meet 2026 (UK)
- Site Management Innovation - Clinical Research 2026 (UK)
- Site Selection and Management - Clinical Trials Congress-2026 (France)
- Smart Drug Delivery Systems - Pharmatech 2026 (USA)
- Smart Drug Delivery Systems - PHARMACEUTICA 2026 (UK)
- Smart Drug Delivery Systems - Formulations 2026 (UK)
- Smart Polymers and Biomaterials - Drug-Delivery-2026 (France)
- Solid Dosage Forms - Formulations 2026 (UK)
- Start-ups, Scale-ups & Pharma Entrepreneurship - Pharmaceuticalindustry-2026 (Netherlands)
- Stem Cell and Genetic Clinical Research - Clinical Research 2026 (UK)
- Stereochemical Drug Design - PHARAMACEUTICAL CONF 2026 (France)
- Structure-Based Drug Design and Molecular Docking - Euro MedChem and CADD 2026 (France)
- Sustainable & Green Pharmaceutical Development - Pharmaceuticalindustry-2026 (Netherlands)
- Sustainable Biologics Manufacturing - Biologics Meet 2026 (UK)
- Synthetic and Green Medicinal Chemistry - Euro MedChem and CADD 2026 (France)
- Synthetic Biology & Bioengineering Approaches - Biotech-2026 (France)
- Target Identification and Validation - Euro MedChem and CADD 2026 (France)
- Targeted Oral and Gastro-Resistant Formulations - Drug Devilvery 2026 (France)
- Targeted Therapies - Personalizedmedicine-2026 (Switzerland)
- Targeted Therapies in Cardiovascular Regeneration - Cardiovascular-2026 (France)
- Targeting the Renin-Angiotensin-Aldosterone System (RAAS) - Cardiovascular-2026 (France)
- Technology in Pharma Sciences - MedTech-2026 (Switzerland)
- Telemedicine - Personalizedmedicine-2026 (Switzerland)
- The Role of CROs in Clinical Trial Execution - Clinical Trials-2026 (UK)
- Theranostics and Personalized Medicine - Drug Devilvery 2026 (France)
- Think Tank: Solving Real-World Pharma Challenges - Pharmaceuticalindustry-2026 (Netherlands)
- Toxicology - Pharmacology 2026 (Italy)
- Toxicology & Safety Pharmacology - Euro Pharmacology 2026 (France)
- Track 01: Digital Marketing in Pharma Industry - Future Pharma 2026 (Netherlands)
- Track 02: The Future of Pharma R&D - Future Pharma 2026 (Netherlands)
- Track 03: Pharmaceutical Chemistry - Future Pharma 2026 (Netherlands)
- Track 04: Pharmaceutical Analysis - Future Pharma 2026 (Netherlands)
- Track 05: Pharmaceutics & Pharmacotherapeutics - Future Pharma 2026 (Netherlands)
- Track 06: Industrial Pharmacy and Physical pharmacy - Future Pharma 2026 (Netherlands)
- Track 07: Clinical and Hospital Pharmacy - Future Pharma 2026 (Netherlands)
- Track 08: Pharmaceutical and healthcare Industry - Future Pharma 2026 (Netherlands)
- Track 09: Regulatory Affairs and Intellectual Property Rights - Future Pharma 2026 (Netherlands)
- Track 10: Pharma and Biotech Financial Outlook - Future Pharma 2026 (Netherlands)
- Track 11: Novel Drug Delivery Systems - Future Pharma 2026 (Netherlands)
- Track 12: Drug Discovery and Development - Future Pharma 2026 (Netherlands)
- Track 13: Green and Community Pharmacy - Future Pharma 2026 (Netherlands)
- Track 14: Big data analytics and Pharma - Future Pharma 2026 (Netherlands)
- Training, Talent Development & Future Workforce - Pharmaceuticalindustry-2026 (Netherlands)
- Transdermal and Intradermal Delivery Systems - Drug-Delivery-2026 (France)
- Transdermal and Topical Drug Delivery - Drug Devilvery 2026 (France)
- Translational Research - Personalizedmedicine-2026 (Switzerland)
- Translational Research & Clinical Applications - Biotech-2026 (France)
- Types of Formulations - Formulations 2026 (UK)
- USFDA Approved Biosimilars - Eurobiosimilars 2026 (Italy)
- Vaccine Delivery Technologies - Drug-Delivery-2026 (France)
- Vaccine Drug Delivery Systems - PHARMACEUTICA 2026 (UK)
- Vaccine Technologies & Immunotherapeutics - PHARMA BIOTECH 2026 (UK)
- Vaccines & Immunotherapy - Pharma Expo-2026 (Thailand)
- Vascular Pharmacology: Focus on Endothelial Dysfunction - Cardiovascular-2026 (France)
- Veterinary & Comparative Pharmacology - Euro Pharmacology 2026 (France)
- Wearable and Implantable Drug Delivery Devices - Drug Devilvery 2026 (France)