At NDA, we know how crucial a well-planned and executed regulatory strategy is for a collaborative and rapid review, approval and introduction of a new treatment to the patients that need it.
How to get ahead when things are moving fast
Most novel medicines that are introduced in clinical practice globally are first approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA). Over the past two decades, both regulatory agencies have established several programs, such as fast track and breakthrough designations in the US and the European PRIME pathway, intended to help shorten the drug development and approval timeline and make new therapies available more rapidly. These programs enable closer collaboration with the agencies, more detailed feedback and a smoother track to submitting a marketing application.
Any drug development process must generate evidence in several stages to produce a product that is safe and efficacious to pass all regulatory requirements. The intention of acceleration strategies is to speed the availability of new therapies to patients with serious conditions, while preserving appropriate standards for safety and efficacy. Which regulatory pathway to pursue depends on the characteristics of the future indication and the target patient population of your project. When a drug is accredited with an expedited drug development designation, it will typically involve more frequent interactions with the agency to conduct the trial and to submit the relevant data.
Need for speed
These expedited pathways are not by any means intended to lower the bar for approval. Instead they provide a platform where you as an applicant can partner with Health Authorities to optimise generation of the most relevant data. Drugs approved via an expedited pathway may however have less data available at the time of initial approval than a drug in a standard development program. As these programs aim to provide access to therapy for serious diseases that lack therapeutic options, the benefit/risk profile in expedited programs balances on a razor’s edge predicated on just the right data, robust enough to justify any decision. By asking the right questions, your designated expert from NDA will keep you steady making sure every high pace decision is in the best interest of your product and the patients you aim to treat.
The approval decision via expedited pathways will most often not be based on established clinical benefit but on surrogate endpoints or clinical endpoints measured early and is reasonably likely to predict a beneficial effect. Choosing the right endpoint and proving its value is anything but straightforward and there are a number of factors to consider. With the collective experience of our experts, NDA can offer reliable and detailed advice on how to translate your product into clinical use.
From confusion to clarity
Navigating these accelerated drug-development pathways requires an in-depth understanding of health authority expectations and the required documentation. Communication with the Agencies is a critical aspect of expedited programs. Partnering up with external expertise will be beneficial for the designation process, through all stages of development as well as for any high-stakes meetings with the authorities.
Creating and submitting a robust application that meets the rigorous regulatory requirements in an efficient and timely manner requires a strong dedication to science, effective quality control and a sense of urgency. At NDA, we share your passion and will help you make the most of your product’s potential.
Your drug development needs are unique
Contact us to learn how our experts can support you to meet all regulatory, quality and safety requirements and ensure the best path to approval.
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