Challenges in Rare Diseases
Making a pharmaceutical care development program for the treatment of a uncommon disease can, however, prove to be a monumental task. Poor understanding of the characteristic history of the proposed indication due to few observational studies considering disease progression, heterogeneous patient populations with variable phenotypes and clinical courses, geographic dispersion of patients and investigators, regulatory uncertainties, and lack of prior clinical studies to establish a format for study execution, can all prove challenging. While individually rare, orphan diseases are really collectively common, with an estimated 350 million sufferers around the world.
- Patient attrition
- Interface between clinical development and the regulatory process
- Less knowledge on pathophysiology of disease
- Lack of awareness
- Diagnosis
- Developing and using Orphan medicinal products
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Challenges in Rare Diseases Conference Speakers
Recommended Sessions
- Clinical Research on Rare Diseases
- Living with a Rare Diseases
- Rare Disease
- Rare Paediatric Diseases
- Treatment for Rare Diseases
- Challenges in Rare Diseases
- Future of Rare Diseases Research
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- POC Treatment in NTD - Tropical Diseases 2018 (UAE)
- Practical Guidelines - Infection Control 2019 (USA)
- Practices & Awareness - Infection Control 2019 (USA)
- Preventing Gastrointestinal Infection - Infection Prevention 2018 (Spain)
- Preventing Infectious Diseases - Euro Infectious Diseases 2018 (Italy)
- Preventing Mother-to-Child Transmission of HIV - STD-HIV AIDS 2018 (Switzerland)
- Prevention &Treatment - STD Asia Pacific 2018 (Japan)
- Prevention and Control - Global Infections 2018 (Australia)
- Prevention and control of Infectious diseases - Infectious Conference 2018 (South Africa)
- Prevention and Treatment of Infectious Diseases - Microbiology Meet 2018 (Romania)
- Prevention of Infectious Diseases - Infectious Disease Conference (Netherlands)
- Prevention of mother to child transmission - STD Asia Pacific 2018 (Japan)
- Problems in infectious disease practice - Infectious Diseases Meet 2018 (Japan)
- Programs in biological Retrovirology - STD-HIV AIDS 2018 (Switzerland)
- Protozoan Infection and Prevention - STD Asia Pacific 2018 (Japan)
- Protozoan Infectious diseases - Infectious Disease Conference (Netherlands)
- Public awareness - Emerging Diseases 2018 (Switzerland)
- Public Awareness - Influenza 2019 (Germany)
- Public Health Microbiology - Microbiology Meet 2018 (Romania)
- Public health practice - Emerging Diseases 2018 (Switzerland)
- Public Health practice - Euro Infectious Diseases 2018 (Italy)
- Pulmonary Infection Therapy - Infection Prevention 2018 (Spain)
- Pulmonary Infectious Diseases - Influenza Research 2019 (Canada)
- Pulmonary/Chest Infections - Infection Congress 2019 (UK)
- Rare Aging Diseases - Rare Diseases Meet 2019 (UAE)
- Rare Brain Disorders - Rare Diseases Meet 2019 (UAE)
- Rare Cancer Diseases - World Rare Diseases 2018 (UAE)
- Rare Cardiac Diseases - Rare Diseases Meet 2019 (UAE)
- Rare Disease - World Rare Diseases 2018 (UAE)
- Rare Diseases - Tropical Diseases 2018 (UAE)
- Rare Diseases in Aging - World Rare Diseases 2018 (UAE)
- Rare Diseases in Aging - Rare Diseases 2018 (USA)
- Rare Diseases in Anaesthesiology - Rare Diseases Meet 2019 (UAE)
- Rare diseases in Bloodstream - Rare Diseases Congress- 2019 (Germany)
- Rare Diseases in Brain - World Rare Diseases 2018 (UAE)
- Rare Diseases in Cancer - Rare Diseases 2018 (USA)
- Rare Diseases in Endocrine System - World Rare Diseases 2018 (UAE)
- Rare Diseases in Eye and Ear - World Rare Diseases 2018 (UAE)
- Rare Diseases in Nephrology - Rare Diseases Congress- 2019 (Germany)
- Rare Diseases in Neurology - Rare Diseases Congress- 2019 (Germany)
- Rare Diseases of Endocrine System - Rare Diseases Meet 2019 (UAE)
- Rare diseases of Endocrine System - Rare Diseases Congress- 2019 (Germany)
- Rare Diseases of Genitourinary System - Rare Diseases Meet 2019 (UAE)
- Rare Diseases of Lymphatic System - Rare Diseases Congress- 2019 (Germany)
- Rare Gastrointestinal Diseases - World Rare Diseases 2018 (UAE)
- Rare Genetic Diseases - Rare Diseases Meet 2019 (UAE)
- Rare Gynecological and Obstetrical diseases - Rare Diseases Congress- 2019 (Germany)
- Rare Haematological Diseases - Rare Diseases Meet 2019 (UAE)
- Rare Haematology Diseases - World Rare Diseases 2018 (UAE)
- Rare Hereditary diseases - Rare Diseases Congress- 2019 (Germany)
- Rare Immune System Diseases - World Rare Diseases 2018 (UAE)
- Rare infectious diseases - Global Infections 2018 (Australia)
- Rare infectious diseases - Emerging Diseases 2018 (Switzerland)
- Rare Infectious Diseases and Immune Deficiencies - Rare Diseases 2018 (USA)
- Rare Mental and Behavioral Disorders - Rare Diseases Congress- 2019 (Germany)
- Rare Metabolic Diseases - Rare Diseases Meet 2019 (UAE)
- Rare Musculoskeletal Diseases - Rare Diseases Meet 2019 (UAE)
- Rare Musculoskeletal Diseases - Rare Diseases Congress- 2019 (Germany)
- Rare Nutritional and Metabolic diseases - Rare Diseases Congress- 2019 (Germany)
- Rare Obstetrical Diseases - Rare Diseases Meet 2019 (UAE)
- Rare Oncology - Rare Diseases Congress- 2019 (Germany)
- Rare Oncology Diseases - Rare Diseases Meet 2019 (UAE)
- Rare Oral Diseases - World Rare Diseases 2018 (UAE)
- Rare Oral Diseases - Rare Diseases Meet 2019 (UAE)
- Rare Paediatric Diseases - World Rare Diseases 2018 (UAE)
- Rare Paediatric Diseases - Rare Diseases Meet 2019 (UAE)
- Rare Pediatric Diseases - Rare Diseases Congress- 2019 (Germany)
- Rare Pulmonary Diseases - Rare Diseases Meet 2019 (UAE)
- Rare Skin Diseases - Rare Diseases Congress- 2019 (Germany)
- Rare Skin Diseases - Rare Diseases Meet 2019 (UAE)
- Rare, Neglected & Tropical Infectious Diseases - Microbiology Meet 2018 (Romania)
- Re-emergence of neglected infectious diseases - Emerging Diseases 2018 (Switzerland)
- Recent emerging diseases - Emerging Diseases 2018 (Switzerland)
- Recent Outbreaks and their Control - Infection Congress 2019 (UK)
- Research on virology and Infectious diseases - Virology Asia 2019 (Singapore)
- Respiratory diseases - Emerging Diseases 2018 (Switzerland)
- Respiratory Infections - Global Infections 2018 (Australia)
- Respiratory Infectious Diseases - Euro Infectious Diseases 2018 (Italy)
- Respiratory Tract and Pulmonary Infectious Diseases - Infectious Diseases 2018 (Portugal)
- Rheumatic Heart Disease - Infectious Diseases 2018 (Portugal)
- Rise of Multiomics: Integrative approach - Neglected Tropical Diseases 2018 (USA)
- Risk Factors & Prevention - Infection Control 2019 (USA)
- Sepsis/ Septicaemia - Infectious Diseases 2018 (USA)
- Sexually transmitted diseases - STD-HIV AIDS 2018 (Switzerland)
- Sexually Transmitted Diseases - STD Asia Pacific 2018 (Japan)
- Sexually Transmitted Diseases/ Sexually Transmitted Infections - Infectious Disease Conference (Netherlands)
- Sexually Transmitted Diseases/ Sexually Transmitted Infections - Infectious Diseases 2018 (USA)
- Sexually Transmitted Infectious Diseases - Influenza Research 2019 (Canada)
- Sexually transmitted infectious diseases - Emerging Diseases 2018 (Switzerland)
- Skin Infections - Global Infections 2018 (Australia)
- Socio-economic impact of NTD - Tropical Diseases 2018 (UAE)
- Stature of Influenza Vaccine - Influenza 2019 (Germany)
- STD and Contact Diseases - Infection Congress 2019 (UK)
- STD And HIV infection - Euro Infectious Diseases 2018 (Italy)
- STD and HIV Infection - STD-AIDS-2018 (Newzealand)
- STD and HIV infections - Infectious Diseases Meet 2018 (Japan)
- Super and Co-Infections - STD-HIV AIDS 2018 (Switzerland)
- Surgical site infections - Emerging Diseases 2018 (Switzerland)
- Surgical Site Infections - Infectious Diseases 2018 (USA)
- Terrifying Viral Infections - Immune Response-2018 (UAE)
- Tracking and Preventing Zoonotic Disease - Influenza 2019 (Germany)
- Translational Strategies to Prevent NTD’s & Infectious Diseases - Neglected Tropical Diseases 2018 (USA)
- Treatment and Advanced Therapies options for Rare Diseases - Rare Diseases Congress- 2019 (Germany)
- Treatment for Infectious Diseases - Infectious Diseases 2018 (USA)
- Treatment for Infectious Diseases - STD-AIDS-2018 (Newzealand)
- Treatment for Rare Diseases - World Rare Diseases 2018 (UAE)
- Treatment for Tuberculosis - Infection Control 2019 (USA)
- Tropical Bacterial Diseases - Tropical Diseases 2018 (UAE)
- Tropical infectious diseases - Infectious Diseases Meet 2018 (Japan)
- Tropical Infectious Diseases - Euro Infectious Diseases 2018 (Italy)
- Tropical Parasitic Diseases - Tropical Diseases 2018 (UAE)
- Tropical Protozoan Diseases - Tropical Diseases 2018 (UAE)
- Tropical Viral Diseases - Tropical Diseases 2018 (UAE)
- Tropical Zoonotic Diseases - Tropical Diseases 2018 (UAE)
- Tuberculosis - Infectious Diseases 2018 (USA)
- Tuberculosis and Hepatitis - Global Infections 2018 (Australia)
- Types and causes of Infectious diseases - Virology Asia 2019 (Singapore)
- Types of STDs and Infectious Diseases - STD-AIDS-2018 (Newzealand)
- Urinary tract infection - Emerging Diseases 2018 (Switzerland)
- Urinary Tract Infections - Infectious Diseases 2018 (USA)
- Vaccination - Global Infections 2018 (Australia)
- Vaccination and Infectious diseases - Infectious Diseases 2018 (Portugal)
- Vaccine Research& Development - Infection Control 2019 (USA)
- Vaccines - Euro Infectious Diseases 2018 (Italy)
- Vaccines and Immunotherapy - Immune Response-2018 (UAE)
- Vaccines and Vaccination - Infectious Diseases 2018 (USA)
- Vaccines and Vaccination - STD-AIDS-2018 (Newzealand)
- Vaccines and Vaccination - Infectious Diseases Meet 2018 (Japan)
- Vaccines for Tropical Diseases - Tropical Diseases 2018 (UAE)
- Vector Borne and Zoonotic Infectious Diseases - Microbiology Meet 2018 (Romania)
- Vector borne infections - Emerging Diseases 2018 (Switzerland)
- Vector borne infectious diseases - Infectious Conference 2018 (South Africa)
- Veterinary bacteriology - Infectious Conference 2018 (South Africa)
- Veterinary Infection Control - Infection Control 2019 (USA)
- Veterinary Infectious Diseases - Infectious Disease Conference (Netherlands)
- Veterinary Infectious Diseases - Infectious Diseases 2018 (Portugal)
- Veterinary infectious diseases - Infectious Diseases Meet 2018 (Japan)
- Veterinary Infectious Diseases - Euro Infectious Diseases 2018 (Italy)
- Veterinary infectious diseases - Emerging Diseases 2018 (Switzerland)
- Veterinary Science - Influenza 2019 (Germany)
- Veterinary Virology - Virology Asia 2019 (Singapore)
- Viral Genomics - Immune Response-2018 (UAE)
- Viral Hepatitis - Infectious Diseases 2018 (Portugal)
- Viral immunology - Virology Asia 2019 (Singapore)
- Viral Immunology - Immune Response-2018 (UAE)
- Viral Infections - Global Infections 2018 (Australia)
- Viral Infectious diseases - Infectious Disease Conference (Netherlands)
- Viral infectious diseases - Infectious Diseases Meet 2018 (Japan)
- Viral Infectious Diseases - Euro Infectious Diseases 2018 (Italy)
- Viral Oncology - Microbiology Meet 2018 (Romania)
- Viral Pathogenesis - Immune Response-2018 (UAE)
- Viral Proteomics - Immune Response-2018 (UAE)
- Viral STD - STD Asia Pacific 2018 (Japan)
- Viral Virulence and Latency - Immune Response-2018 (UAE)
- Viral, Bacterial and Fungal Infections - Neglected Tropical Diseases 2018 (USA)
- Virology - Bacterial Diseases 2018 (UAE)
- Virology and infectious diseases - Infectious Conference 2018 (South Africa)
- Viruses and Cancer - Immune Response-2018 (UAE)
- Viruses and Epidemiology - Immune Response-2018 (UAE)
- Viruses and Tumours - Virology Asia 2019 (Singapore)
- Water, Sanitation and Hygiene - Tropical Diseases 2018 (UAE)
- Zika Virus - Infectious Diseases 2018 (Portugal)
- Zika Virus: The Next Emerging Threat? - Neglected Tropical Diseases 2018 (USA)
- Zoonotic diseases - Influenza 2019 (Germany)
- Zoonotic diseases - Infectious Conference 2018 (South Africa)
- Zoonotic Diseases - STD Asia Pacific 2018 (Japan)
- Zoonotic Diseases - Influenza Research 2019 (Canada)
- Zoonotic Infectious Diseases - Infection Congress 2019 (UK)
- Zoonotic Infectious Diseases - Infectious Diseases Congress 2018 (Turkey)