By Joseph Lamendola Ph.D. and Tim Franson M.D.
Joseph Lamendola, PhD and Timothy Franson, MD walk through the requirements for kickstarting a new development program in a Biopharma organization.
As an entrepreneurial biopharmaceutical or MedTech company, your main focus is likely getting your product(s) to market as fast as possible. It’s also likely that your main challenge is ensuring the quality and robustness of your development plans – whether you know it or not.
Oftentimes, we find that startup companies are working with development plans seeking the wrong clinical end points, regulatory strategies targeting the wrong FDA divisions, and some teams too confident to outline an FDA engagement and approval strategy at all because they feel that their technology and product are too strong to fail. If a firm faces any of these challenges, they will invariably experience major setbacks in their development programs, which can lead to millions of dollars in reanalyzing and redoing studies or regulatory failures that could sink their company.
However, working alongside highly experienced regulatory strategists and development experts can greatly help remediate these issues, as these types of people are uniquely positioned to uncover their main development challenges within hours of their interactions. After identifying the critical challenges, they can provide very specific and pragmatic approaches to minimize these risks and accelerate development pathways. Ultimately, this helps avoid millions of dollars in unnecessary costs, save thousands of manhours, and realize more timely value from their assets.
We have found that companies which kickstart their development programs early by engaging external, unbiased experts at the beginning of the process have greater success and often get their assets to market months ahead of schedule. This is especially true for startups developing their initial product(s), OUS firms seeking to introduce new assets to the U.S. market, and firms with cutting-edge technologies whose regulatory pathways are ambiguous.
Launching Your First Product
It’s well-documented that, for every product that gets FDA approval, there are hundreds of compounds, molecules, or device concepts that never make it past the pre-clinical phase. Therefore, we understand the struggles that occur while trying to turn interesting research idea into highly valuable, clinical-ready assets; it’s even harder to get a product to receive FDA approval. Many companies who are moving through the development lifecycle for the first time are anticipating and navigating the complex inner workings of the FDA, which can lead to failures in submissions or never getting the product past the pre-clinical phase due to avoidable clinical and regulatory issues.
For example, a recent client with numerous pre-clinical assets engaged Dr. Tim Franson, former VP of Regulatory Affairs at Lilly, to review their current portfolio and recommend ways to accelerate development and increase the value of the assets. After just a few conversations, Dr. Franson helped guide the team down a development and regulatory pathway prior to pre-IND, which “helped them save $2M in potential development costs,” according to the client.
Launching Your First Asset with the FDA
We have also experienced multiple instances where organizations based outside the US were seeking to develop and commercialize assets with the FDA for the first time. Some of these firms even had extensive regulatory teams, but lacked the U.S. capacity and FDA knowledge required to formulate a development plan that met regulator expectations and best leveraged acceleration levers, such as Fast Track Designations. On the flipside, when companies engage with highly experienced FDA regulatory experts, they will likely discover ways to streamline the development and submission process, which can lead to a products’ launch months earlier than anticipated, driving considerable revenue increases.
One such instance involved a large, EU-based pharmaceutical company that was planning its pediatric clinical program for a marketed drug. The company knew that a global program with the same clinical studies, fulfilling both FDA and EU requirements, would conserve resources and lead to a shorter timeline. The planned indication was sickle cell disease, and with a greater number of patients with sickle cell disease based in the U.S., the company needed someone in the U.S. to lead the clinical trials. Despite having a U.S. facility, they lacked the clinical and regulatory bandwidth to accomplish the program. My team was able to engage a highly experienced, clinical development and regulatory subject matter expert who was able to successfully guide the development of the drug with their skills and background handling the U.S. drug development process, along with her extensive experience with the drug’s mechanism of action.
Groundbreaking Technologies with Ambiguous Regulatory Pathways
The pace of technical change across the life sciences industry continues to accelerate, especially as technology breakthroughs, including artificial intelligence, digital health, biomarkers, gene therapy, CAR-T, and personalized medicine start to gain traction. Furthermore, many companies are seeking to leverage original approaches, such as benefit-risk assessments, to gather real-world input from patients to enhance rare-disease treatments for the nearly 55 million patients (in just the U.S. and EU) suffering from one of the 7,000 rare diseases, 95% of which have no treatments to date. Many of these firms may not be able to fully grasp or execute upon the regulatory rigor and understanding required to effectively move these advancements through the FDA regulatory pathway to effectively commercialize their capabilities.
For example, Duchenne Muscular Dystrophy (Duchenne), until recently, did not have any approved treatment to stop or slow its fatal progression and did not have an established benefit-risk assessment approach that put patients first, similar to 95% of rare disorders. A specialized team of subject matter experts, spanning multiple expertise areas and even multiple companies, performed a preference-based benefit-risk study and subsequently developed an independent guidance for a number of process enhancements, including an innovative risk-benefit assessment approach for the rare disorder in FDA review. This was the first independent disease-specific guidance submitted to the FDA. The guidance detailed new clinical trial designs and improved benefit-risk assessment approaches, which is one of the FDA’s goals for its patient-focused drug development initiative.
Whether you are launching your first asset as a company, bringing your OUS asset to the U.S., or seeking innovative approaches to advance groundbreaking therapies with complex, ambiguous, and possibly undocumented regulatory pathways, the first step is to develop a team led by a seasoned regulatory executive to guide your way to success.
Be sure to visit our webinar to learn more about how smart biotechs scale quickly and maximize value.
About the Author: Dr. Franson an industry expert in regulatory affairs and drug development policy. Dr. Franson is the Chairman of the Board for the Critical Path Institute, the Immediate Past President of the USP, and former VP of Global Regulatory Affairs and Patient Safety for Eli Lilly & Company.
About the Author: Joe Lamendola, Principal of YourEncore’s Regulatory Center of Excellence, over a 30-year regulatory career, is responsible for 30+ approvals across 10+ therapeutic areas. With hundreds, if not thousands, of agency interactions under his belt, Joe provides clients with the insights, relationships, and credibility needed to bring drugs to patients quicker.