How Scientific Affairs Can Assist Your Team with the FDA

 

By Joseph Pierro, M.D., chief medical officer of Biomedical Systems, and Kelley Atherton, M.A., medical writer for Biomedical Systems.

 

 

In its research report on regulatory science, the Center for Drug Evaluation and Research at the U.S. Food and Drug Administration proclaimed, “Scientific knowledge steers the drug approval decision-making process of the Center for Drug Evaluation and Research. Regulatory Science is the science of developing new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of all FDA-regulated products.”

 

The ultimate goal of any clinical research and development team is to receive regulatory approval, which is accomplished by assembling a science-based, data-driven development plan capable of producing an adequate amount of sufficient-quality data to meet regulatory requirements. Given that the intricacies of product development experience are the key to success, the role of a scientific affairs group, irrespective of that group’s organization (i.e., in a pharmaceutical, biologics, or device company, clinical research organization, or specialty vendor), is to work with the clinical and R&D teams to create a thorough and comprehensive development plan. By starting with an informed and inclusive strategy that anticipates critical product-related regulatory questions, incorporates recent regulatory decisions, and anticipates future regulatory directions, the probability of regulatory approval will be greatly improved.

 

While acknowledging the many complexities of product development, the sponsor teams should factor in these six guiding principles when planning programs for regulatory submission.

 

Deliver clear communication of the development plan and clinical studies. It is important to understand that the sponsor team has all the available information (at any stage of development) about the new drug, biologic, or device. The regulatory agency may have no prior experience with the innovation, but the reverse situation may also be true. In that case, regulators will utilize past review experience or knowledge that defines the principles of regulatory science and will present questions or requests for data that might seem disconnected from your program.

 

Understand relevant regulatory development history. Review, discuss, and understand all of the available regulatory guidance documents, and apply relevant study requirements and trends from past approval decisions within the appropriate therapeutic class or category. With this knowledge, the team can build a collection of these data points into studies (i.e., in vitro, nonclinical, and clinical studies) in terms of safety, efficacy, or risk minimization/mitigation to support approval.

 

Attend advisory committee meetings. Or review past advisory committee meeting transcripts held by the representative drug or device review division; these transcripts are readily available from the Freedom of Information Act Office.

 

Consider global requirements in planning regulatory interactions. Plan a process of asking the right questions that are appropriate for the stage of the product’s development. Keep in mind that regulatory decisions are based on data provided to the regulators; it’s important to keep to your regulatory plan and not get too far out in front of your development strategy, as this may lead to additional or unnecessary requests.

 

Experienced teams succeed. Product development can be complex, and experienced teams are more likely to succeed. Keep the team focused on the critical questions and aware of the product’s important or relevant benefits and risks rather than trying to know everything there is to know and conducting a constant stream of “interesting” experiments or clinical studies that complicate the submission plan and may add financial costs or timeline delays. Include in the team’s development plan a strategy for studies that should be conducted while the regulatory agency is reviewing the submission and post-approval.

 

Anticipating regulation requirements. Often, development teams will seek additional support from external groups, such as scientific affairs groups, regulatory and clinical professionals, and clinical research organizations, to provide additional resources that will complement the internal team’s knowledge and experience. Experienced SA groups can enhance the team’s development strategy and increase the likelihood of first-pass regulatory approval by providing a higher level of scientific and regulatory understanding based on their access to a wide range of product development programs from a more diverse group of sponsors, whereas an individual sponsor’s team may have more limited experience based on a fairly narrow R&D pipeline of new ideas or products.

 

Avoiding potential surprises or regulatory traps is one of the most frequently reported benefits of using external expertise. Having the right level of knowledge and experience allows the sponsor not only to select the best study designs, but also to be better prepared to respond to regulatory bodies. For example, using SA consultants with recent regulatory interactions can guide development plans in anticipation that the regulatory body may request a certain study design or request the sponsor to collect a certain amount or type of data. Having this insight, which may be available only from an external source, allows the sponsor to proactively perform a risk assessment as to whether the company will decide to build the anticipated request into the development plan. The company can also prepare a “position paper” in advance to explain its data in light of the request.

 

Additionally, the SA consultants can serve as independent reviewers of the development plan or study results, and, by nature of being independent of the clinical team, may be better positioned to serve as unbiased assessors, thereby mimicking the level of review that a regulatory body would perform. An independent assessment of the plan could provide valuable insight on the adequacy of data and provide objective observations on the strengths, deficiencies, and gaps in the submission package.

 

Utilizing the knowledge of SA groups can improve regulatory and development strategies and improve the chances for first-pass regulatory review and approval. Adequate knowledge and preparation are critical elements of a clinical trial, so to quote Benjamin Franklin: “By failing to prepare, you are preparing to fail.”

 

 

ABOUT THE AUTHORS

 

Joseph Pierro M.D. is chief medical officer of Biomedical Systems, a premier global provider of centralized diagnostic services. With more than 20 years of experience in clinical trials and regulatory affairs, he has served in global senior-level positions within the pharmaceutical industry and as a medical reviewer at the FDA Center for Drug Evaluation and Research. His thorough understanding of regulatory submission requirements and outstanding track record of success with regulatory approvals and responses to regulatory authorities make him an asset to Biomedical Systems’ Scientific Affairs team.

 

Kelley Atherton, M.A. is a medical writer for Biomedical Systems. She is a professional writer who has been published across multiple platforms on an array of topics.

 

 

References:

 

1. Center for Drug Evaluation and Research. Regulatory Science in Action: Enhancing Drug Safety and Manufacturing Quality at FDA with Research. FDA website. http://www.fda.gov/downloads/Drugs/DrugSafety/UCM300948.pdf. Accessed 28 June 2016.