Orphan Drug Designation Support

Orphan Drug Designation is a status granted by regulatory authorities to encourage the development of drugs and biologics for rare diseases or conditions, affecting fewer than 5 in 10,000 people in the European Union (EU), fewer than 200,000 people in the United States (U.S.) or a similarly small population in other regions. The benefits of obtaining this designation are significant and include:

tax credits, fee waivers, market exclusivity after approval, and increased assistance from regulatory bodies, including protocol assistance and scientific advice.

To qualify for Orphan Drug Designation, the drug must be intended to treat a rare disease or condition that meets the rarity threshold set by the relevant regulatory authority. There must be a plausible hypothesis explaining how the drug could potentially benefit patients with rare conditions. If treatments already exist, the new drug must offer significant advantages over current therapies.

The application must include detailed information such as description of the disease, including prevalence and incidence rates, data on the drug’s development, mechanism of action, and preliminary efficacy and safety data, scientific data supporting the drug’s potential effectiveness in treating the rare condition and a justification for why the drug should receive orphan status, emphasizing the rarity and unmet medical need.

A regulatory affairs consultancy provides invaluable support throughout the Orphan Drug Designation process, offering expertise and resources to ensure a smooth and successful application. Key services include:

• Application Preparation: crafting and compiling a comprehensive and compelling application, including the necessary scientific rationale.
• Regulatory Navigation: guiding you through the regulatory requirements and processes, ensuring that all documentation meets the standards set by authorities such as the EMA in the EU or the FDA in the U.S.
• Strategic Advice: offering strategic advice on positioning your drug for Orphan Drug Designation, including identifying and articulating the unmet medical need and potential benefits of your drug.
• Ongoing Support: acting as a liaison between your company and regulatory agencies, responding to queries, and providing updates on the application status to help facilitate timely approval.