Accelerating development and managing risk
April 23-26, 2017
and industrial biopharmaceutical communities. While the benefits to patients and to sponsors of accelerating development are obvious, the risks of accelerating development are less clear. This talk will attempt to address some of the CMC risks associated with accelerating development and how they might be managed.
FDA (and analogous regulatory bodies outside the USA) have announced the availability of rapid regulatory paradigms for products which meet currently unmet medical needs. The details of these programs vary from country to country, but they are similar in that most may permit the product to be marketed based upon submission of Phase 2 clinical data. This may reduce the time required for clinical development from 8 - 12 years to as little as 3 – 4.5 years, and may reduce the number of patients needed from thousands to hundreds. While this has clear benefits in terms of saving time and expense for large clinical trials, there is relatively less benefit for the CMC package, as in general, the standards and expectations for the CMC package for an accelerated approval remain largely the same as for a market authorization application from a 8 – 12 year program. This creates a significant effort to complete what is normally an 8 – 12 year CMC effort in 3 – 4.5 years. While effective planning and management can achieve much of this, the time required to complete certain long term studies cannot be easily condensed. This requires realization of these needs early, and spending at risk in order to complete the needed work in time for the filing. Examples of these situations will be discussed.
There may also be technical requirements or target product profile requirements which can change the typical time and/or investment required to complete CMC studies in support of the filing. Examples of these might include a delivery device, a co-diagnostic, a special administration need (ultralow doses or ultrahigh volumes of administration), or an improved manufacturing process. The development timelines and/or investments need to take such considerations into the planning and budgeting for the project, and well as the risk-reward scenarios. These will inevitably lead to discussions with senior management about whether the requirements are absolutely required or nice-to-have, and will translate to what CMC studies are required for the market authorization and what can be accomplished as a post approval change. There are multiple ways to make these decisions, and several will be illustrated during the presentation.
Anthony S. Lubiniecki, "Accelerating development and managing risk" in "Regulatory Sciences for Biologics and Vaccines: Accelerating Development and Enabling Manufacturing Innovation", Prof. Antonio Moreira (University of Maryland, Baltimore County, USA) Dr. David Robinson (Robinson Vaccines and Biologics LLC, USA) Eds, ECI Symposium Series, (2017). https://dc.engconfintl.org/biologics_vaccines/12