Expedited pathways & designations

Supporting the development and evaluation of new treatments for rare diseases is a key priority for the FDA. As you conduct your IND preparations, it is important to consider and take advantage of the FDA’s special programs/designations to speed up your approval. You can submit your application for a special designation at any stage of the drug development process. However, it is typically recommended that you apply early in the development process to maximize the benefits and incentives associated with the programs.

Pro tip
The FDA encourages sponsors to discuss their plans for special programs before they submit an Investigational New Drug (IND) application. This allows time for the FDA to review and grant the designation early on, providing important incentives and regulatory advantages throughout the drug development process.

What are special designations/programs?
These are specific regulatory pathways and designations established by the U.S. Food and Drug Administration (FDA) to expedite the development, review, and approval of certain products. These designations are important because they provide incentives and regulatory advantages to encourage innovation in areas where treatment options are limited. By so doing, the FDA helps to advance treatments for serious or life-threatening conditions, expediting access to potentially life-saving treatments for patients in need.

Special designations are meant to address 21 CFR part 312 (intended to speed up the availability of new therapies to patients with serious conditions, especially when there are no satisfactory alternative therapies, while preserving appropriate standards for safety and effectiveness). See figure below for important definitions to note when it comes to special programs. [5]

If you are using this playbook, then chances are you are addressing an unmet need and qualify for at least one of the special designations – take advantage.

Special designation	Description
Serious condition	A disease or condition associated with morbidity that has substantial impact on day-to-day functioning - short-lived and self-limiting morbidity will usually not be sufficient, but the morbidity need not be irreversible if it is persistent or recurrent. All conditions meeting the definition of life-threatening as set forth in the CFR would also be serious conditions.
Unmet medical need	A condition whose treatment or diagnosis is not addressed adequately by available therapy. An unmet medical need includes an immediate need for a defined population (i.e., to treat a serious condition with no or limited treatment) or a longer-term need for society.
Available therapy	The FDA generally considers “available therapy” (and the terms “existing treatment” and “existing therapy”) as a therapy that: Is approved or licensed in the United States for the same indication being considered for the new drug. Is relevant to current U.S. standard of care (SOC) for the indication.

Now that we have established the definitions and baseline, let us get into the details of each of the special programs and how you can take advantage of them.

Documentation Index

Expedited pathways & designations