Further implications for the pharmaceutical industry

Pharma will have to consider the most efficient way to tackle RWE generation and regulatory submission, given the case-by-case approach from the FDA. Companies will need to address the major concern from the FDA that real world results would be cherry picked for positive beneficial data. Some of the questions that the Pharma industry would need to address would include:

1. Does the pharma company’s current organization structure (decision ownership and stakeholder involvement) align to efficient execution of RWE generation for regulatory approval?

2. Have sufficient resources been allocated to handle RWE generation for regulatory approvals?

3. What is the decision-making process to evaluate trade-offs between RWE vs. trials to receive follow-on indication approvals for products?

4. Will the FDA’s acceptance of RWE open the door for more partnerships between pharma and academic centers and how this will be handled?

5. Is the company able to effectively choose between different types of RWD and patient reported outcomes?

6. How cost effective would it be to choose the RWE program vs. traditional clinical trials?

7. What discussions should be held with the FDA and when should they be held for RWE trial design and submissions—especially given the current case-by-case approach to review and approval?

“Pharma manufacturers will need to consider a new model of integrated decision making which evaluates traditional clinical trials, hybrid designs, and RWE in life cycle management planning. Roles, responsibilities, timelines, decision models and resourcing may change as a result”


The RWE Program opens opportunities for more resource efficient approaches to follow-on indications. Increased definition by the FDA is needed to guide resource investment and trial design approaches to ensure efficient investment. Pharmaceutical manufacturers will likely need to evolve their internal structure and systems, including decision models related to RWE trial design and regulatory interaction to accommodate this new route of approval. We look forward to reviewing and understanding the updated guidance expected from the FDA in the future.

Want to know more?

Herspiegel Consulting can help you evaluate the implications of the new guidelines on your organization including stage gate models, lifecycle management strategic planning, and organizational structure. Please contact Beth Schurman at Beth@Herspiegel.com or 267-759-2132

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