Drug development is an entrepreneurial endeavor, fostered by a passion for scientific discovery of new treatments to help patients. The focus is to commit the right resources to reach the end goal of providing life-saving drugs to patients quickly and cost effectively.
A successful partnership with a CRO begins with a shared goal and passion for delivering good medicine. Though the dictionary definition links entrepreneurism with assuming risk, successful partnerships are often based on working with CROs that thrive on creating a business and culture that reduces risks.
A CRO partner with a track record of stability, low-employee turnover and long-standing relationships with customers can establish a solid foundation for a new business relationship.
Rho, a contract research organization (CRO), offers five tips to selecting the right development partner to accelerate approval for orphan products.
1) Work with CROs that have strong scientific, regulatory, and statistical expertise. A strategic approach with a focus on key milestones is critical to gain approval as quickly as possible. Look for CROs whose strengths include the ability to conduct challenging clinical trials, knowledge of the regulatory process, and scientific and statistical expertise to develop a plan for success at the outset to reach approval in an expedited, speedy manner. Your CRO should have successfully obtained marketing approval for other orphan products previously.
2) Know the “ins and outs” of the FDA’s approval mechanisms to help speed orphan drug approval. Many orphan diseases represent serious or life-threatening conditions. Consequently, working with a development partner that understands each of the accelerated development pathways (i.e., Accelerated Approval, Priority Review, Breakthrough Therapy, and Fast Track) and the potential benefits or lack thereof is critical.
3) Apply for U.S. and European Orphan Drug Designation simultaneously. There is a combined form that can be used to obtain orphan drug status simultaneously in the U.S. and EU. It is an option that is not being used broadly, but can result in significant reduction of time and effort.
4) Look for a CRO partner with experience working in small patient populations. Working with small patient populations requires building communities and developing close connections with research foundations, advocacy groups, patients and health care providers for a purpose-driven approach to product development. It will also be important to gain buy-in from key opinion leaders.
5) Validate your population. Before investing time and energy in an orphan drug application, make sure you are eligible. Regulators are on the lookout for developers who try to “slice the salami,” meaning that your orphan population is really just a subset of a larger population from which there is no substantive difference.
Whether seeking a CRO relationship for full-service development or a specific service expertise, some basic guidelines can help ensure a successful relationship:
• Cultural Fit: Select a CRO partner that views itself as an extension of your team and demonstrates a clear understanding of your specific program and its challenges. Strategic partnerships anticipate the need to quickly address challenges along the way. Involvement from senior management and the development of clear and effective governance at the executive, functional and clinical research levels can be the cornerstone of a successful relationship to ensure that time is spent effectively to achieve objectives and control costs.
• Flexibility: Working with a CRO that is experienced in developing long-term partnerships with customers and the flexibility to meet the needs of each customer can minimize the time required to achieve success. An ideal CRO partner is one that is nimble enough to quickly adjust to last-minute changes in the program to overcome potential work flow challenges. The results are faster cycle times and improved quality. How can you tell if the CRO is actually flexible? Ask for case studies and references.
• Cost Control Approaches: Defining goals, methods and what metrics to measure prioritizes key objectives and establishes a path to success. Whether the business relationship is driven by cost constraints, a desire to accelerate speed to market or both, the objectives should be explicitly addressed in governance and the contractual terms of the relationship.
• Quality Control: CROs with a reputation for providing high-quality results and meeting deadlines can reduce time and energy spent on project oversight. Work with a CRO that prides itself on scientific integrity and precision, and a culture of customer attention and reliability proven over time.
• Stability: The flurry of industry consolidation in the CRO industry has many observers speculating on the role small and mid-sized CROs will play in the future. These CROs — focused on long-term objectives and avoiding the turmoil of public stock offerings, mergers and acquisitions — can provide an advantage of stability.
In an industry where the goal is speed and efficiency to get drugs to market as rapidly as possible, consistent growth and stability is a winning formula to shape a CRO that customers can trust.
ABOUT THE AUTHOR
Tara Gladwell is vice president of operations for Rho, a full-service CRO based in Chapel Hill, N.C. She received her undergraduate degree in Biology from Guilford College and her M.B.A. from Duke University’s Fuqua School of Business. She joined Rho in 2001 as a project manager.