By Lisa Jenkins VanLuvanee, Ph.D.
I hear all the time from life sciences CEOs and innovators, “I don’t know what I don’t know, and that keeps me up at night.” One of the best ways to address this situation is to obtain a gap analysis for your product. Drug, biologic, medical device, and combination product developers can use gap analyses to find out what they don’t know, where their product falls short (if anywhere), and if they are ready to progress to the next stage in regulated development.
What is a Gap Analysis & Why is it Important for Life Sciences?
A gap analysis is an evaluation tool that helps life sciences companies identify where they might be falling short in their development process. Whether it’s regulatory, clinical, or commercial, conducting a gap analysis helps companies understand their current standing and how to move forward efficiently. These analyses are essential for ensuring that all aspects of a product’s development comply with industry standards and regulatory requirements, minimizing the risk of delays or costly mistakes.
Types of Gap Analyses in Life Sciences
There are a variety of different types of gap analyses in the R&D space:
Regulatory Gap Analysis
The focus of this type of gap analysis is to identify gaps in your program, data, or documentation that are needed to support a regulatory submission (e.g., IND/NDA/BLA). For example, at Facet, we are often asked to evaluate whether a company is ready for a pre-IND meeting or an IND submission. Using a regulatory gap analysis, we review the available nonclinical studies, chemistry, manufacturing, and controls information, and the IND-enabling study protocol to determine if the information complies with FDA regulations and guidances and if there is adequate safety information to support the safety of the subjects in the proposed clinical trial.
In the case of a gap analysis with a focus on a pre-IND meeting, we determine whether a pre-IND meeting is the right type of meeting to be requested, based on the stage in development and whether the questions the company has for the FDA are focused and well-written. We also evaluate whether the company has the necessary data for FDA to be willing to grant a meeting. For an IND submission, the development program and IND submission content are reviewed against FDA’s IND requirements. If gaps are identified in a regulatory gap analysis, we make recommendations on whether those gaps must be addressed or if it’s possible to move to the next regulatory milestone and address the gap later.
Clinical Gap Analysis
The purpose of a clinical gap analysis is to evaluate the adequacy of one or more clinical trial protocols, final study reports, or the overarching clinical program to satisfy moving forward. Emphasis in protocol and study report review is placed on determining whether the patient population, inclusion/exclusion criteria, schedule of assessments, trial endpoints, and analytic methods are appropriate, especially to support a desired indication. Gaps can be identified in a number of areas including overall trial design, study procedures, analytical methods, efficacy and safety endpoints, and adverse event reporting.
Clinical trials are evaluated against GCP and specific FDA requirements for the product, product class, specific indication, and industry standards. Trials that do not meet FDA standards because of departures from GCP or other gaps may not pass regulatory scrutiny and are unlikely to satisfy regulatory requirements. Knowing these gaps and fixing issues prior to trial execution will save both time and money.
Nonclinical Gap Analysis
The purpose of a nonclinical gap analysis is to determine if there are any gaps or risks in the nonclinical data package that would prevent the Sponsor from progressing to the next stage in development. The evaluation begins with a comparison of what has been completed (e.g., pharmacology, pharmacokinetics, and toxicology) compared to what nonclinical studies are required for the product at the specific stage in development. Early in development, a nonclinical gap analysis evaluates whether animal studies (and possibly data from the literature) adequately support the proposed IND-enabling trial. Later in development, a nonclinical gap analysis evaluates whether the nonclinical package and data are adequate to support an FDA approval.
In both cases, the goal of a small company should be to run only the required nonclinical studies to support development and nothing more. An early gap analysis can help accomplish this goal!
CMC Gap Analysis
The focus of a CMC gap analysis early in development is to ensure that the current manufacturing processes and information submitted to an IND support first-in-human trials. Later in development, the focus of a CMC gap analysis is directed towards evaluating whether the manufacturing processes and data support commercial-scale production and the stability requirements cover the desired shelf life of the product. A CMC gap analysis at the NDA/BLA stage will also verify that any previous issues or questions raised by the Agency have been addressed prior to submitting the NDA/BLA. This can be a particularly helpful gap analysis because FDA has reported that the most common reason why marketing applications do not receive first-round approval is because there are CMC gaps that must be mitigated prior to approval.
Commercial / Market Access Gap Analysis
A commercial or market access gap analysis elucidates gaps that could affect the successful development and launch of a product in the US market. The commercial gap analysis marries development strategy with commercial objectives so that the product is ultimately valuable to payers, physicians, and patients. Failing to understand gaps in market research and payer requirements early in development can lead to inefficiencies in early research and development efforts and may not support cost-effectiveness claims for payers.
For example, payers might require real-world evidence to set pricing, but if the Sponsor has not collected this information, pricing and reimbursement discussions may be less effective or delayed. A commercial gap analysis is most commonly forgotten by small companies, but it is often the difference between a company that succeeds and one that fails to secure investment during development or one that fails to achieve US market success upon approval.
Why Gap Analyses Are Critical at Multiple Stages of Development
Each type of gap analysis targets a specific aspect of research and development in the regulated space. No single gap analysis is more important than any other. Gap analyses should be performed at several stages in development because gaps can arise at any time when new information about the product comes to light. In general, it is critical that there is alignment with scientific, regulatory, commercial, and corporate objectives and gap analyses can elucidate this alignment. Knowing the gaps and limitations of your product’s regulatory, nonclinical, CMC, clinical, and commercial position can give peace of mind when progressing to the next stage in development. It can also help ensure that product development stays focused, goal-driven, and positioned for success.
How Facet Life Sciences Can Help with Gap Analyses
At Facet Life Sciences, we specialize in providing strategic gap analyses to identify gaps in your product development and regulatory pathways. With our extensive experience in life sciences, we tailor our services to ensure your product is prepared for the next regulatory milestone. Whether it’s preparing for a pre-IND meeting, addressing clinical trial design issues, or ensuring compliance with FDA regulations, we help you move forward with confidence.
Why Choose Facet Life Sciences for Gap Analysis Consulting Services?
Facet Life Sciences has helped over 250 life sciences companies successfully navigate the complex FDA approval process. Our gap analysis services are specifically designed for small to mid-sized companies aiming to streamline their product development process. Contact us today to identify and close any necessary regulatory, nonclinical, CMC, clinical, and/or commercial gaps in your development program to ensure smoother progress toward FDA approval.
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