Top 5 questions biotech companies should ask to enable study success

Selecting the right endpoints is crucial for the success of a clinical trial. Biotechs should consider:

• Patient-centered endpoints: Don’t lose focus on your drug’s functional improvements and quality of life. Your endpoint strategy should go beyond regulatory minimums to demonstrate not only efficacy and safety, but also patient-relevant outcomes that may influence reimbursement decisions.
• Trustworthy, high-quality data: Optimize data by considering your study design holistically, including considerations of sample size, endpoint modalities and technologies, and the schedule of clinical assessments.
• Regulatory-accepted endpoints: Engage early with regulatory bodies to minimize submission delays and ensure endpoints are both clinically meaningful and regulatorily accepted.

Operational pitfalls can delay clinical trials. Key considerations include:

• Protocol design: Engage subject matter experts early on to ensure the protocol is scientifically sound and operationally feasible.
• Study start hand-off: Avoid ambiguity by establishing clear roles and responsibilities, defined schedules, and collaboration among all stakeholders.
• Flexibility: Be prepared to regroup and re-strategize if something isn’t working as planned.
• Role and responsibilities: Ensure that you and your CRO partner have a clear understanding of set up and monitoring responsibilities.

Choosing the right CRO or endpoint partner is critical. Biotechs should consider:

• Proven expertise: Choose a partner with expertise in the therapeutic area and indication you’re studying, and experience with delivery in countries where the study will be conducted.
• Scientific depth: Make sure your partner has access to the right subject matter experts and key opinion leaders, with a proven track record in your area of focus.
• Engagement model: Ensure your partner has demonstrated good collaboration, flexibility, and governance to manage study execution well.

While scientific rigor is essential, it must simultaneously be balanced with patient centricity. Considerations include:

• Participant burden: Minimize the burden on participants while ensuring data quality.
• Data quality: Leverage technologies that allow for accurate and timely data capture, such as Patient-Reported Outcomes acquired remotely between clinic visits.
• Engaging patient groups: Validate assumptions and gain insights from patient groups early on to optimize the study design and minimize burden.

Unexpected issues can arise during study conduct. Strategies to de-risk include:

• Study risk assessment kick-off meeting: Conduct a thorough upfront meeting involving all partners to walk through the protocol and identify potential risks.
• Real-time study surveillance: Leverage innovative technologies for real-time monitoring and data cleansing to identify and address issues promptly.