Regulatory Strategy in Pharma & Biotech Submissions Trial Design

September 14, 2020

Drug development can be a protracted and multifaceted process. This is often the case for startups and newer organizations, which may not have dedicated regulatory compliance departments and established standard operating procedures (SOPs). These organizations may seek regulatory consulting services to support submission strategies and develop the required procedures.

According to Professor Thomas Koperniak of Northeastern University’s Regulatory Affairs for Drugs, Biologics, and Medical Devices department, it’s quite common to take more than ten years to get a single drug to market.

The process requires broad cooperation across internal and external stakeholders, with cross-functional groups in the organization (clinical research, research and development, manufacturing, quality assurance, marketing, and sales) working to advance novel drugs from initial development to commercial production.

Forming a Regulatory Strategy

Regulatory strategy itself is a game plan calculated to obtain approval or clearance of a novel drug. As a matter of course, regulatory requirements are ever-changing, so a well-honed regulatory strategy can maximize the opportunity to achieve regulatory goals.

Based on the insights and any history of precedents, researchers will conduct the following steps. These will help verify the viability of the strategy, and certain elements will be ongoing throughout.

1. Engage Internal Stakeholders

The initial step in developing a sound regulatory strategy for pharma and biotech submission trial design is engaging internal stakeholders. This will sometimes include members of the organization’s resource management team.

Internal stakeholders will vary based on the organization’s nature and size, but can include regulatory affairs personnel, research scientists, legal advisors, key executives, or other decision-makers. Other internal stakeholders may consist of R&D/engineering personnel, medical/clinical affairs staff, and marketing departments.

2. Evaluate Product Attributes and Potential Regulatory Liabilities

Here, stakeholders will quantify the attributes of the novel compound to thoughtfully evaluate the regulatory implications of its use, design, and performance. Regulatory affairs managers and directors often spearhead the development of the regulatory strategy. Quite often, research scientists or biopharma executives will play a key role.

The goal of this step is for stakeholders to understand the novel compound comprehensively. This can be a tricky proposition, as some stakeholders involved in developing the regulatory strategy may not be well versed in drug development. The research scientists on the team may have to spend a substantial amount of time acquainting other team members with the pharmacology and pharmacokinetics of the proposed compound. The goal is to derive useful insights into the new drug’s efficacy and potential regulatory liabilities.

Stakeholders evaluating attributes will typically consider issues relating to the following:

  • Intended use (users, pathology or pathologies addressed, environment, patient populations)
  • Design (functionality and mechanism of action)
  • Claims of efficacy
  • Competing products
  • Target markets
  • Data to support claims and meet regulatory objectives (arising from existing or ongoing research studies)
  • Regulatory concerns
  • Jurisdictional concerns
  • Post-market issues (e.g., legal liability)
  • Post-approval studies

3. Perform Regulatory Intelligence

With product attributes that may have regulatory impact identified, researchers need to uncover precedents that may influence the emerging regulatory strategy. Even considering the most novel compound, there are generally analogues that can anticipate regulatory issues and impediments.

In the U.S., small biotech firms and pharmaceutical conglomerates alike often rely on the FDA’s website to perform research into conducting new submissions. Another useful source of information is the website, which may help identify clinical investigation products, inform clinical trial design strategy, and provide insight into study results.

4. Document Past Strategies

In this phase, researchers scrupulously analyze the novel compound in light of the aspects previously researched. The goal is to determine if any issues are likely to come up from a regulatory standpoint, and how to best address them.

Analyzing past submissions will provide insight into past strategies that have either succeeded or failed.

Risks, critical assumptions, and any barriers to approval should be documented. This documentation will provide insight into how previous or current requirements may impact the product development timeline.

5. Testing Effectiveness in the Clinical Study

With the strategy in draft form, the internal stakeholders will evaluate the strategy to ensure that it reflects the product attributes and goals identified at the beginning of the process. Researchers should anticipate revisions as new data is revealed and the implications of initial assessments become apparent. The organization’s tolerance for risk and overall business goals will also factor into this part of the process.

At this stage, it may be appropriate to consult with regulatory authorities to gain clarity regarding the chief key components of the regulatory strategy. This is often done in the form of briefing books and regulatory authority meetings.

6. Implementation and Continuous Improvement

The regulatory environment is persistently changing, so organizations involved in pharma and biotech submission trials must stay current with regulatory developments. Regulatory and governing bodies are prone to change as well, which can impact policy. Bear in mind that your organization’s regulatory strategy will remain a “work in progress” that will need to be revisited as the regulatory environment and your organization change.


With a well-reasoned and well-executed regulatory strategy, an organization can enjoy far more predictable product development and clearance efforts. When in doubt, enterprise resource planning solutions can also help fill in organizational strategy gaps. However, a good understanding of the product and factors that drive the regulatory review are the first steps in formulating a winning regulatory strategy.


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