Authors: Gerald L. Klein, MD; Roger E. Morgan, MD; Emilia Jones Amaowei, MD; Michael Fath, PhD; Freddy Byrth
Product Development
● Choosing the right contract manufacturer is critical for a biotech company to successfully bring new products to market. They must be able to provide a high-quality product on time, without unexpected cost increases and minimal delays.
o Small companies should understand that a poor choice can ruin or significantly delay company efforts. Engaging a specialized consultant with expertise in the specific type of manufacturing needed is often a crucial component of the team.
o Consideration should also be given to confirming the contract manufacturer has a back-up location for providing medication, in the event the primary facility becomes inoperable due to a catastrophic incident.
o The manufacturer should have a strong record of compliance with FDA, European Medicines Agency (EMA), and other regulatory bodies, supported by extensive quality systems (e.g., Good Manufacturing Practice) (GMP) certification, validated processes, audit readiness).
● AI may be a useful tool, but its outputs must be thoroughly reviewed. Some platforms have been known to generate inaccurate or even fictitious information (termed hallucinations). For example, when asked to supply scholarly references for a publication, some AI tools generate fabricated citations.
● One potential FDA reform that could significantly reduce the time and cost of pharmaceutical product development is the formal acceptance of approval based on a single, well-designed Phase 3 efficacy trial—on the condition that a confirmatory post-marketing effectiveness study, capable of generating statistically robust results, is conducted within a predefined timeframe. This approach would not only accelerate initial market access but also provide real-world evidence of the product’s pharmacoeconomic value. Failure to complete the post-marketing study as agreed would trigger automatic market withdrawal. While it is sometimes possible to negotiate approval based on a single Phase 3 trial under current regulations, formalizing this as a consistent regulatory pathway would eliminate the default expectation for two pivotal trials in most cases.
Medical Affairs
● It appears that when major journals publish articles on the results of drug clinical trials, they sometimes omit significant safety information.
o An article may compare the number of serious adverse events (SAEs) and adverse events (AEs) in both the placebo and investigational product groups but fail to specify which events were related to the active product. This omission is especially disconcerting as it may obscure the complete safety profile of the active product. This omission makes it difficult to assess the actual risk-to-benefit ratio.
o To enhance transparency without shifting focus from efficacy, journals should require brief, clear attribution of AEs or SAEs to the investigational product. This simple step strengthens credibility and ensures readers understand a more complete safety profile.
● When developing consumer information, it is critical to present accurate details without sugarcoating or concealing a product’s disadvantages. Although it is appropriate to emphasize the benefits of its use, the total story should be clearly articulated. Failure to do so may result in regulatory issues which may undermine trust with both healthcare professionals and the general public.
● Optimizing the Medical Affairs Role in Real-World Evidence Generation: Medical Affairs is essential in developing real-world evidence to complement data from controlled clinical trials and support the broader understanding of a product’s value and use.
o Stakeholder Engagement: Collaborates with healthcare professionals, payers, and patient advocacy groups to gather post-market insights.
o Real-World Insights: Tracks safety signals, uncovers new therapeutic benefits, and observes usage patterns across diverse populations.
o Data Utilization: Leverages real-world data sources such as electronic health records (EHRs), patient registries, and claims databases.
o Evidence Generation: Produces data that informs medical decision-making and supports peer-reviewed publications.
o Payer Communication: Provides critical input for pharmacoeconomic discussions and value-based reimbursement strategies.