Patients living with rare diseases are often persistently misdiagnosed or undiagnosed, potentially resulting in no treatment at all or treatments that are ineffective or unsafe. Due to the lack of definitive diagnostics or efficacious and safe therapies, foundations and research centers have been raising funds for re-purposing existing therapeutics or developing new drugs (typically classified with an orphan drug status*). As the development of biomarkers and genetic signatures continues to progress, techniques and technologies are anticipated to become more precise and comprehensive, thereby reducing the time to rare disease diagnoses (see related PMLS on May 11).
Such innovations at universities and medium to smaller companies has subsequently led to hundreds of clinical trials to test biomarkers, gene signatures, and new treatments.** Recruitment for these trials is complicated by identifying and making those eligible aware of enrollment. Therefore, design and structure of orphan drug trials for small, dispersed populations for efficient trial recruitment and execution are areas of great interest. Due to the nature of these diseases, data submission and review of successful trials are often allowed expedited review to speed access of drugs to patients and relief for families.
If you work in rare diseases or rare disorders, this conference is one you cannot miss. Come and hear talks and discussions by leaders in academia, biotech, pharmaceuticals, diagnostics, clinical research, informatics, healthcare and regulatory bodies at the world’s most informative virtual conference on the following topics and more:
- Experiences from all parties – patient journeys, doctors’ consultations, payer perspectives
- Recruiting rare disease patients for clinical trials
- Interfaces and graphics that convey information through remote access to providers and patients
- Tools that allow online access for patients, doctors, data sets, multi-participant meetings
- Challenges of treating rare disease patients and families during a pandemic
- Online support groups for sharing information among families and patients
- How large, medium, and small companies in pharma, clinical, and diagnostic groups are addressing rare disease challenges, including translational medicine, data analysis, economic modeling, real-world data capture
- Partnerships among diagnostics companies, pharmas, payers, and CROs for connected online resources
- Positioning rare disease services for coverage by payers
Who should attend:
- Executives and Decision Makers – CEOs, CTOs, CMOs, and CSOs
- Research, Development, and Practicing Clinicians
- Biomarker and Diagnostics Developers
- Clinicians and practitioners who use telehealth
- Translation Medicine
- Fee and Payer Models
- Diagnostics Purchasers, Sales, and Planning
- Regulatory Leaders in Government and Business Affairs groups
- Marketing Leaders
- Payers, Medical and Pharmacy Directors, Actuaries, CFO
- Data analytics and informatics specialists
Websites of interest
Rare Disease Day, https://www.rarediseaseday.org/
*Designating an Orphan Product: Drugs and Biological Products, https://www.fda.gov/industry/developing-products-rare-diseases-conditions/designating-orphan-product-drugs-and-biological-products
**See rare disease, rare disorder, orphan drug on ClinicalTrials.gov at https://clinicaltrials.gov/ct2/home