Pharma, Pharmaceutics and Biotechnology Conference

Orphan drugs are an important area of innovation in pharmaceuticals, oriented towards developing therapies for rare diseases involving small patient numbers.

It is in this scope that the session ""Orphan Drugs"" was so well focused - what are specific challenges and opportunities in developing an orphan drug, what regulatory frameworks, incentives, and patient access issues shape a specialty field.

The session will start with a definition of an orphan drug and criteria for an orphan designation, usually based on an illness or disease being diagnosed that affects fewer than 200,000 patients in the United States or comparable numbers in other parts of the world.

Attendees will learn about the trend for orphan drugs as treatment solutions often are lacking for many rare diseases and patients have few places to turn. One of the main themes will be the regulatory incentives for orphan drugs. They will hear about benefits from orphan drug designation, such as tax credits for clinical trials and market exclusivity and fee waivers-helping to offset the risk in developing for small patient populations.

The session will cover the role of the regulatory agencies, FDA and EMA, in granting orphan drug status and the specific pathways that expedite the approval process, including Fast Track and Priority Review designations.

The session will focus on a few specific challenges drug companies face in creating orphan drugs. These include the extremely high research and development costs, the practical difficulty of conducting large scale clinical trials, and so on.

Participants would then be able to identify some of these challenges and how adaptive trial designs, patient registries, and collaboration with rare disease advocacy groups can help address those challenges and move forward orphan drug research.

Another very important issue is the access to orphan drugs for the patient. During the session, strategies for improving access will be discussed, including patient support programs and managed access schemes as well as international collaboration in treatment of smaller populations.

The participants will be made familiar with appropriate models for regulatory frameworks that can ensure patients with rare diseases benefit from timely access to new therapies. Discussions regarding orphan drug development would involve emerging trends.

Advances in gene and cell therapies are promising to unlock new options for the treatment of rare genetic disorders. Presentations of successful cases in orphan drug development will be made, providing evidence of how targeted therapies have reframed the lives of patients suffering from previously intractable diseases. Participants will be informed on the orphan drug landscape, regulatory, financial, and clinical challenges the market-making process poses to such critical therapies.

From drug development to regulatory affairs and patient advocacy, this session will make you informed on what orphan drugs mean for the future of rare disease treatment.

The delegates will then leave here with practical knowledge about navigating the complex pathways of orphan drug development and be equipped to help accelerate innovation for patients with rare diseases.