StemCell Therapy

By Kate Cook, Catherine M. Cook Group, LLC

In the past two years, the pandemic has kept sponsors developing drugs, biological products, and medical devices from meeting in person with FDA.Advisory committee meetings, workshops, and conferences have gone virtual, too. While some sponsors have hopes that changing pandemic conditions will open the doors to in-person meetings, this seems unlikely.

In-person meetings with FDA officials are likely to remain the rare exception, not the norm.The FDA commitment letter developed in the latest round of prescription drug user fee negotiations sets out FDAs commitments to meet face to face with the sponsor at specified stages in a product development program, but defines face-to-face to include... both in-person meetings and virtual meetings on IT platforms that allow for both audio and visual communication.

This means that FDA can opt to meet virtually instead of in person, and it has strong reasons to do so. Meeting virtually avoids the administrative work needed to bring outsiders onto the secure FDA campus, and the format more easily accommodates individual employee schedules.There are very clear scheduling advantages to virtual advisory committee meetings, which are easier to schedule when busy experts are not required to spend a half day or more traveling to and from an in-person meeting.

Moreover, it can seem impossible to bring FDA meeting participants together in one conference room. FDA staff are increasingly likely to be working remotely at locations nowhere near the FDAs White Oak campus in Maryland. FDA officials have made recent statements about the value in authorizing remote work as a recruitment tool, and it is a tool FDA is likely to be using.

FDA has a lot of recruiting to do.In the latest round of user fee agreements, the agency committed to significant numbers of targeted hires to support the prescription drug user fee program, including, for Fiscal Year 2023, 132 CBER staffers and 77 CDER staffers.Recruiting qualified scientific employees is always a challenge for FDA, but it will also need to address recent staff departures.Exhausted by the work of developing and supporting the FDAs pandemic response, experienced staff members are retiring or seeking outside employment in significant numbers.This exacerbates existing understaffing and can lead to significant losses of expertise and institutional memory.

Once these hundreds of new employees are hired, FDA will have to develop employees who work remotely and do not have the benefit of daily in-person interactions with their managers and mentors who may also be working remotely. This will present management and supervisory challenges for the agency and may change its work culture significantly. One thing for sponsors to look out for is that new reviewers sometimes default to an overly conservative position. Sometimes, it may be necessary for sponsors to ensure that supervisors are involved in giving FDA advice, particularly on novel issues.

The primary mechanism for sponsors to obtain advice from FDA on their development programs is by filing a request for a meeting. When FDA grants the meeting, it may be a face-to-face meeting (defined to include a virtual meeting), a teleconference, or a written response only. FDA and industry have described different meeting types in user fee commitment letters for prescription drugs and biological products (PDUFA), biosimilars (BsUFA), generic drugs (GDUFA), and medical devices (MDUFA) as well as in related guidance documents addressing meetings under PDUFA, BsUFA, GDUFA, and MDUFA.

These guidance documents are likely to be revised to incorporate new user fee commitments under the recently concluded negotiations, once Congress has enacted the legislation necessary to reauthorize these user fee programs. For example, the new PDUFA commitment letter addresses two types of meetings for the first time: INTERACT meetings and Type D meetings. An INTERACT meeting provides an opportunity for sponsors to discuss novel questions and unique challenges in early development, prior to filing an IND.A Type D meeting is focused on a narrow set of issues (typically not more than two issues and associated questions). Requests could include a follow-up question that raises a new issue after a formal meeting, a narrow issue on which the sponsor is seeking agency input with only a few associated questions, or a general question about an innovative development approach that does not require extensive, detailed advice.

FDAs commitment letters and guidance documents describe the criteria the sponsor must meet to be granted a specific type of meeting. Although FDAs user fee commitments recognize a right to a face-to-face meeting at certain stages in a product development program, under other circumstances FDA may decline to schedule a face-to-face meeting and will provide a written response only.The commitment letters and guidance documents address the timeframes for the sponsor to submit their briefing document and for FDA to respond to requests and to schedule meetings.

FDA has committed to send preliminary written responses to a sponsors questions contained in their background package.When a meeting has been scheduled, FDA will send the response in advance of the meeting, giving the sponsor the opportunity to focus the meeting on discussing certain FDA responses, or even to determine that the written response is adequate and a meeting is no longer necessary.

Some sponsors want to initiate meetings with FDA as early in development as possible. FDA has recognized that emerging technologies raising cross-cutting issues may warrant very early discussion and has created forums for such discussions in the Critical Path Innovation Meeting, CDERs Emerging Technology Team Meeting, and the CBER Advanced Technology Team Meeting. However, a sponsor that obtains advice too early in preclinical development of a specific product may find that significant pre-IND questions arise a short time after a pre-IND meeting because they decided to modify the product in important ways.Since FDA generally limits a sponsor to one pre-IND meeting, the sponsor may have to ask for advice on their new questions using a different meeting request format, such as a Type C meeting, which has longer timelines for agency response, or the new Type D meeting if FDA agrees that the scope is narrow enough for such a meeting.FDA has advised that for products regulated by CBERs Office of Tissues and Advanced Therapies (responsible for regulating cell and gene therapies, among other biological products), a pre-IND meeting is appropriate if:

A sponsor should assure that all sponsor attendees can access the video conference platform successfully, and that audio is clear and bug-free.The sponsors meeting plans should address not only who is responsible for the sponsors presentation, but also who is the critical note taker and who is authorized to call for a short internal conference if needed during the meeting.The sponsor should be able to mute the sound to FDA in case there is a need to hold a sidebar with sponsor attendees only.

If possible, all sponsor attendees should assemble at the same location.This can be challenging, given that sponsor employees often work at different locations and that a sponsors external consultants may be in attendance, but having everyone in the same room can allow a sponsor to adjust strategy in real time after an internal sidebar discussion.If it is necessary for sponsor representatives to participate from multiple locations, participants should have a plan in place to address the problems that can occur when one site loses connectivity.This may include identifying and preparing back-up presenters for critical presentations.

In addition, the sponsor should think about how their representatives are likely to present themselves to FDA and be on the lookout for presenters who may come across as dismissive of FDA questions and concerns.

A sponsors briefing book should make its case in support of the sponsors position on the issues they are asking FDA to advise on. This is especially important for a sponsor seeking FDA support for an innovative or less burdensome approach.If specific subject matter expertise is important to understanding the issues, a sponsor should request the participation of specific FDA employees or representatives of specific offices.For example, if an issue requires CMC or other manufacturing expertise, the sponsor may request the involvement of a representative from CDERs Office of Pharmaceutical Quality or CBERs Office of Compliance and Biologics Quality.

A sponsors primary point of contact with FDA is the project manager assigned to the development program.Sponsors regulatory affairs personnel generally understand the value of a good relationship with the project manager, who can sometimes work magic in facilitating informal communications between the sponsor and regulators.

FDAs written response to a briefing package may identify issues of concern to FDA.The sponsor should take every opportunity to probe the scope and significance of these concerns in the meeting and in future product development discussions.It sometimes happens that FDA raises an issue in early correspondence with a sponsor whose application for approval, filed years later, is not successful due to the same issue raised in early communications. Sometimes the early notice has prepared the sponsor for this possibility, and the sponsor is ready to make its case for approval on appeal or has already initiated studies to develop data to satisfy FDAs concern.However, sometimes the sponsor will have discounted the significance of FDAs early comments and is unprepared to address them.

A sponsor should also pay attention to the FDA staff attending the meeting.If you are unfamiliar with someone and their FDA role, do a little research.You may learn from an attendees slide presentations that they specialize in a specific issue, such as the distribution of a drug under a Risk Evaluation and Mitigation Strategy (REMS) program.This may signal that the parameters of a REMS distribution program for your drug are the subject of discussion within FDA. Similarly, the involvement of other individuals or offices may signal that FDA is focusing on a drug exclusivity or other type of issue.

Conclusion

The processes for using meeting requests to interact with FDA may seem overly formal, but a sponsor who follows them carefully, submits high-quality briefing materials, and maximizes their meeting opportunities will maximize their chances of receiving high-quality agency feedback. In addition to the assigned reviewers, a sponsors briefing book will also be read by FDA supervisors, whose supervisory and oversight roles may become more important and more challenging as they are called upon to train, mentor, and develop remote workers.

About The Author:

Kate Cook is an FDA veteran who has worked in legal and policy positions in the Office of Chief Counsel, as well as in the FDA's CBER and CDRH, where she served as associate director for regulations and policy.For more than 20 years, she was involved in every aspect of policy development, authoring or reviewing many regulations and guidances. Since she left the agency in 2016, she has worked as a regulatory attorney and consultant, helping her clients in their interactions with the FDAs medical product centers and on appeals to the Office of Commissioner. Cook graduated from Swarthmore College and the New York University School of Law, and was a New York County Assistant District Attorney and an attorney in private practice before joining the FDA. She founded the Catherine M. Cook Group, LLC, in 2022, and can be reached at Kate.Cook@CatherineMCook.com.

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Tips For Your Virtual Meetings With The FDA - Med Device Online

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