Infectious Diseases and HIV Conference

Rare diseases affect fewer than 200,000 individuals in the United States: they are a large, diverse group of health challenges. Diseases-gene disorders, metabolic disorders, some infectious diseases-are often under- or misdiagnosed, thereby causing poor patient outcomes and great healthcare burdens.

This session summarizes the scope of rare diseases and the place of orphan drugs for their treatment. Challenges in Diagnosis: Rare diseases pose significant diagnostic challenges because they are rare, manifestations are diverse, and in some cases, the health providers are unaware of them.

Session on Rare Diseases- The Issues in Diagnosis and Diagnostic Odyssey Using Interdisciplinary Approaches. Session focusing on the inherent complexities of rare disease diagnosis, the role that genetic testing can play in helping to diagnose these conditions, and the promise of interdisciplinary approaches in advancing proper diagnosis. Case studies will focus on the diagnostic odyssey experienced by patients, implications of delay, and resulting outcomes for the patient. Orphan

Drugs: Orphan drugs are specifically made for rare diseases and are regularly given special regulatory status to encourage orphan drug development. Participants in this module learn about orphan drug regulatory frameworks, incentives, such as tax credits, grants, and market exclusivity that different health authorities in various locations around the world provide. Problems associated with developing orphan drugs include market potential, research and development costs, as well as complexity in carrying out clinical studies in small patient groups.

Treatment Strategies and Innovations: Access to therapies: This would also touch on the profile of treatment options for rare diseases, such as orphan drugs, gene therapies, and approaches to personalized medicine. New ways of finding new treatments will be discussed, such as the use of artificial intelligence and data platforms to identify potential drug candidates. The availability of orphan drugs is the big concern for the patients and health care systems, and the session is going to focus on the major issues associated with access which includes unreasonable prices, problems with reimbursement, and sustainable policies for health care well too in order to assure equal and equitable treatment for the treatment of rare diseases.

Discussions will highlight any advocacy work connected with achieving improvements in necessary therapy.

Let us have an interactive session with leading experts in a discussion on unpacking the dilemmas of rare diseases and orphan drugs to further our knowledge and progress in improving outcomes of patient care.