How Emerging Biopharmas and Biotechs Succeed: Insights from Medidata Experts
Emerging biopharma and biotech (EBP) companies drive innovation in clinical trials, with EBPs running 65% of all clinical trials in 20181. However, lean resources, limited access to sites and patients, lack of integrated systems, and uncertain funding impose restrictions on EBPs and slow the pace of drug development. How do EBPs position themselves to succeed and overcome challenges? This post brings insights from Medidata’s experts who have worked with thousands of EBPs and have insider perspectives on how to avoid study pitfalls and stay on the path to success.
Addressing common obstacles
Medidata experts have observed three key obstacles that commonly affect EBPs: budget issues, failure to meet critical end-points, and data access issues. Unanticipated costs arise as a common budget issue because clinical development and CRO costs can encompass an EBP’s entire budget. To plan for these cost changes and mitigate the budget and study delay risks, EBPs can compare price quotes from multiple companies, look for vendors that provide cost flexibility, and have open conversations with vendors about needs and pricing expectations.
Failing to meet critical end-points, such as not collecting enough study data or recruiting enough patients, delays studies and significantly impacts outcomes. To prevent any endpoint failures, EBPs can invest in EDC systems connected to a unified platform, which prevents issues with clinical data management and collection, allowing real-time data oversight and error correction. Additionally, consulting site performance data, including patient recruitment compared to industry benchmarks, helps EBPs make better site selection decisions and negotiate for lower costs.
Outsourcing to CROs, a common practice among EBPs, can complicate remediation of any last-minute issues. Successful EBPs have open conversations with CROs early on about data access to maintain visibility into the crucial study information they need to resolve issues and execute a successful trial.
Regulatory approvals require experience and manpower, especially for novel drugs with no clear path to approval. Foresight into the approval process helps EBPs successfully navigate it. Equipped with insights from experienced technology providers into what aspects of a study regulators commonly push back on, EBPs can proactively address issues and avoid delays in the approval process.
Regulatory bodies commonly request adjustments to a study before the study even begins. To avoid added costs and study delays from these types of changes, we recommend EBPs build relationships with regulators as early as possible to understand their thought process.
EBPs often struggle with cost predictability, especially before a study starts. Successful EBPs spend time designing efficient protocols, but even small protocol amendments can incur significant costs increases. While inexpensive technology solutions may seem appealing to sponsors with tighter budgets, rigid price models can be less forgiving to protocol changes and ultimately lead to much higher costs. Instead, flexible pricing models might better accommodate protocol changes and study fluctuations. To gauge more accurate cost estimates and avoid last-minute cost escalations, successful EBPs research costs for various trial scenarios, set aside additional funds for unexpected costs, and have conversations with technology vendors early in the process.
Stay tuned for a second blog post focused on how EBPs leverage technology and data to power better clinical trials.