The webinar is divided into several sections, either focusing on different aspects of orphan designation (OD) in key global markets or rare disease trials. The goal is to provide attendees with a comprehensive understanding of the benefits, application processes, and strategic implications of securing orphan designations globally.
European Orphan Designation
This section discusses the European Union’s OD process. The significant benefits were highlighted, including eligibility for research grants, increased investor interest, and market exclusivity which provides a ten-year period free from competition from similar medicines. Additional incentives include reduced regulatory fees, access to the centralized procedure for a single EU-wide market authorization, and scientific advice.
The eligibility criteria for EU OD are stringent. The condition must be rare and severe enough and there should be a lack of existing satisfactory treatments or potential significant benefit offered by the investigational product. Project management of EU orphan designations must consider existing data suitability, timelines, and if all relevant stakeholders are in place (e.g. EEA sponsor).
APAC Orphan Designation
The orphan drug landscape in the Asia-Pacific region and Australia was explored in this section. Australia has a significant population affected by rare diseases, and its orphan drug program, established in 1998, offers benefits such as fee waivers for Therapeutic Goods Administration (TGA) evaluation and Pharmaceutical Benefits Advisory Committee (PBAC) applications. The program’s eligibility criteria have evolved over time, now requiring a prevalence threshold of fewer than 5 in 10,000 people and excluding certain biological products.
The orphan designation criteria across APAC markets were compared, including the absence of specific orphan drug legislation in China and recent regulatory developments in Korea and Taiwan that support conditional approvals based on Phase 2 data and accelerated approval pathways, respectively.
U.S. Orphan Designation
The presentation also covers the U.S. orphan designation process. The benefits such as market exclusivity and financial (e.g. tax credits) were mentioned prior to outlining the eligibility criteria (i.e. prevalence, data requirements, and competitive medicine landscape). Like other OD projects, considerations regarding existing data sets, timeline, and procedural requirements are needed for successful delivery.
Rare Disease Clinical Trials in Australia
A section of the presentation is dedicated to the advantages of conducting rare disease clinical trials in Australia. Australia’s clinical trial landscape is highlighted for its cost-efficiency and the supportive regulatory environment provided by the TGA, which can be particularly beneficial for small to medium-sized companies looking to expedite their development timelines.
Strategic Considerations and Global Perspective
The webinar concludes with a global perspective, emphasizing the importance of a well-rounded strategy when seeking orphan designation across multiple regions. Companies are encouraged to align their regulatory strategies with the specific requirements and incentives of each target market to maximize their chances of success. The presenters also address post-approval considerations, such as maintaining orphan status and navigating related regulatory procedures like the Small and Medium-Sized Enterprise (SME) status and Advanced Therapy Medicinal Product (ATMP) classification.
Conclusion
The presentation underscores the critical importance of a global strategy for orphan drug designation, highlighting the varying requirements and benefits across different regions. By understanding and leveraging these differences, companies can optimize their development and regulatory approval processes for rare disease treatments, ultimately improving patient access to innovative therapies. The Q&A session at the end of the webinar offered attendees the opportunity to clarify specific concerns and gain further insights from the experts.
This post was written by Liam Spencer. Liam is Senior Project Manager, Regulatory Consulting and Services. His 8 years’ experience includes both pharmaceutical and medical device regulatory affairs. He has worked across different technologies, including; small chemical entities, ATMPs, biologics, vaccines, combination products, and class I-III devices. Working across all phases of development has allowed Liam to gather a broad understanding of regulatory affairs and, therefore, devise creative regulatory strategies.
This strategic and project management skillset was developed at his previous position as a global regulatory lead for a large CRO (Contract Research Organisation). Throughout his career he has also worked on complex international submissions and helped prepare a variety of regulatory documents such as IMPDs, protocols, investigator brochures, clinical evaluation reports, and device technical files. Liam has worked for companies of different sizes and backgrounds and, with his strong experience in a consultancy environment, he can tailor regulatory solutions to the client’s needs.