Regulatory Pathways for Orphan Drug Designation

by Michael Firgens

To obtain orphan drug designation, drug developers need to understand how to manoeuvre through the regulatory frameworks and to prepare a sound justification for their drug candidate. The regulatory requirements and evaluation process may vary from country to country and also depend on the policy status and experience of regulators. The individual elements for an orphan drug designation are illustrated by the example of the EU regulatory framework.

Approximately 6,000 to 9,000 distinct rare diseases have been identified worldwide affecting together millions of people, many of whom are children. Additionally, around 250 new rare diseases are described annually. However, only 5 percent of rare diseases have treatments representing a significant unmet medical need. Historically the development of medicines for rare disease has been unattractive for pharmaceutical companies. Therefore, governments put incentives in place to encourage development of medicines for rare diseases, so-called “orphan drugs” (Table 1).

Table 1: Country overview of legal frameworks for orphan drug designation

To obtain orphan drug designation, drug developers need to understand how to manoeuvre through the regulatory frameworks and to prepare a sound justification for their drug candidate. The regulatory requirements and evaluation process may vary from country to country and also depend on the policy status and experience of regulators.

Generally, an application for orphan drug designation addresses the following key areas:

  • Description of the disease/ condition
  • Is the disease rare (prevalence/ incidence)?
  • Sufficient scientific rationale supporting that the drug will treat, diagnose or prevent the disease/ condition at issue?
  • Which other therapies exist? And how is the new drug better than existing therapies?

Figure 1: Key elements for orphan drug designation in the EU

As an example, an overview on the individual elements for orphan drug designation in the EU is presented in Figure 1. Each element needs to be elaborated concisely in meetings with the authorities and in the application for orphan drug designation. Due to the nature of rare diseases, unique challenges have to be resolved, e.g. with regards to availability of only limited experimental data and the conduct of clinical trials.

Biopharma Excellence is experienced in developing orphan drugs and obtaining orphan drug designation. Please do not hesitate to contact us if you need support in areas such as:

  • Establishment and assessment of scenarios for orphan drug designation (globally or nationally)
  • Preparation and conduct of pre-submission meetings at EMA, FDA or other regulatory agencies
  • Preparation and filing of application for orphan drug designation
  • Development of integrated development strategies considering all regulatory requirements for biopharmaceuticals