An Executive Roundtable was held Oct 10 with 10 attendees from a range of startup pharma. The topics covered a wide range of drug development challenges and strategies such as clinical trial design and execution, patient engagement and registries, data infrastructure and management, and funding strategies for early-stage biotech. The discussion started with participants sharing their current pain points, which include issues around clinical trial design, getting through IND and early clinical development, and challenges in demonstrating meaningful efficacy.  Then the group delved deeper into specific challenges, such as the importance of building the right team with experienced clinical and regulatory experts, the need to carefully plan and design clinical trials with the right endpoints and statistical analysis, and the critical role of patient registries and natural history data to support rare disease drug development. There was also discussion around the complexities of global clinical trial expansion and access, as well as the importance of data infrastructure and management to support multiple programs. Finally, the group explored alternative funding strategies, particularly the use of equity crowdfunding platforms, as a way for early-stage biotech companies to raise capital.  Overall, the groups emphasis was to adopt a mantra that it is never too early to get in and strategically plan the next steps.

Observations

• Team building.  There is a need to get right team in place, especially an the CMO and medical advisory board.

• Patient Voice.  Partner with patient advocacy groups to get growth of patient registries and natural history data.

• Diversity and Inclusion. Clinical trial expansion around the globe is challenging and often results in restrictions to patient access., so engagement with various patient communities and regulatory bodies early on is needed.

• Adequate Liquidity.  The challenges were discussed of how to get a small biotech sufficiently funded so that value can be created and crowdfunding may be a solution for some.

Issues and Solutions

Issue 1. Brought up was the challenges in demonstrating meaningful efficacy in complex diseases like osteoarthritis. 

Solution. There is need to carefully design clinical trials to enrich the patient population, leverage natural history data as a reference control, and focus on endpoints that are meaningful to patients. Develop a clear target product profile and thoroughly characterize the pharmacodynamics in multiple animal models to inform the clinical development plan and increase the chances of success in early clinical testing.

Issue 2. There are difficulties in transitioning from successful preclinical development to early clinical testing. 

Solution. Suggested is to develop a comprehensive target product profile and thoroughly characterize the pharmacodynamics in multiple animal models to inform the clinical development plan. Invest in building comprehensive patient registries and natural history data to support rare disease drug development, leveraging this data as a reference control in clinical trials.

Issue 3. There is lack of experience in the drug development industry among academic researchers. 

Solution. Bring in experienced clinical and regulatory experts to guide the process and help navigate industry standards and requirements. Establish a strong clinical and regulatory team, including an experienced Chief Medical Officer and medical advisory board, to guide the clinical development strategy and trial design.

Issue 4. Challenges occur in global clinical trial expansion and patient access.  

Solution. Engage with patient advocacy groups and regulatory bodies early on, build patient registries in diverse regions, and consider hybrid/decentralized trial designs to improve accessibility. Engage with patient advocacy groups and regulatory bodies early on to understand the needs and requirements for global clinical trial expansion and patient access.

Issue 5. How to overcome funding challenges for early-stage biotech companies.  

Solution. Explore alternative funding strategies like equity crowdfunding platforms to raise capital and build a diverse investor base.

Two Main Takeaways:

1. How do you get across the "Valley of Death" in research and get the Big Idea into trials? Emphasis was made on engaging with regulatory early and getting that IP captured as key value add steps in matriculating a program towards the clinic.

2. What are good clinical trial design practices? A good value hedge offered was that phase II hold emphasis on getting a variety of endpoints to detect efficacy and to save the Power Analysis for phase III. You want multiple shots on goal - biomarkers and outcomes that allow you to say "Efficacy was demonstrated with X"