The past decade has seen more Orphan Drug (OD) development and approvals than any other time period in history with EMA and FDA receiving record breaking numbers for orphan drug applications year on year. Obtaining OD designation is one of the first and fundamental steps in developing a drug for a rare disease. An OD designation greatly strengthens the business case and investment basis for early stage drugs, providing the momentum to enter the clinic in larger scale.
There are increasingly high barriers for OD designation in respect of proving medical plausibility, prevalence of the disease, and the significant benefit (the latter applicable for EU only) over existing therapies. Getting strong independent scientific advice and operational support can significantly improve the chances of success of an OD designation and save valuable time and money.
NDA experts have built significant experience in Orphan Drugs and we offer our global services to help clients to successfully overcome many of the common obstacles in this field. Our dedicated team comprise regulatory, clinical as well as statistical experts with practical experience and deep knowledge in this challenging area. The NDA team has extensive experience with EMA, FDA and National EU Health authorities and in depth knowledge on how to develop successful proposals and applications to regulators. Many of NDA’s experts are former regulators themselves.
NDA support includes setting a robust orphan drug development strategy. The NDA Advisory Board and experienced regulatory consultants work together to provide strategic regulatory and scientific insights to help define the best development path for your product.
On an operational level, NDA can manages the OD designation application process and write the application. In addition, NDA has considerable experience in preparing and conducting Health Authority meetings and oral explanations.
How we help
NDA can support you in all aspects of applying for and maintaining OD designation in Europe and the US from early development to marketing approval. These include:
- Regulatory advice on orphan drug requirements, incentives and benefits
- Scientific advice on data requirements
- Search, analysis and presentation of prevalence data
- Authoring of combined or region-specific OD designation applications
- Procedural advice and full support (e.g. preparation of submission package, translations, health authority liaison)
- Preparation of response documents
- Preparation of and participation in the dialogue and meetings with authorities
- Authoring of annual reports and maintenance reports
- Provision of an EU-based Orphan Drug sponsor service enabling non-European companies to benefit from European OD incentives.
- Provision of a US-based entity facilitating OD designations for companies outside US.
We offer the full support package, and are very open to define a custom package that fits the needs of the individual client.