Innovation in Combo Product Clinical Development – Harnessing Patient Insights from Reported Usability Outcomes

Industries Profiled:
Pharmaceutical; Consumer Products; Biopharmaceutical; Health Care; Biotech; Clinical Research; Laboratories; Medical Device


Companies Profiled:
Novartis; Windtree Therapeutics; EMD Serono; Merck; Sanofi; Shire; Bausch + Lomb; Alkermes

Study Snapshot

Best Practices, LLC engaged 11 industry leaders from 8 companies through a benchmarking survey.
Included in this research are qualitative insights and case studies generated from partnering with these industry leaders.

Key topics covered in this report include:

• Combination product clinical development team structures
• Effective sub-teams for critical development activities
• Reported usability outcome measure data ownership and use
• Late phase trial methods: Home-use vs. In-clinic vs. mix of both, timing of to-be-marketed device use
• Observer & Patient reported usability outcomes by phase, effectiveness, and volume of implementation
• The most effective and essential observer and patient outcome measures

• Late Phase Trial Analysis for Combination Products:

• When first testing the to-be-marketed form of a combination product, structure trials with at least some component of in-clinic administration.
• In-clinic administration is an essential trial design component when deploying usability outcome measures prior to Phase III, using a mix of home & in-clinic is more prominent for Phase III and Open label extensions.

• Development Team Composition: 100% of large companies implement a formal team for combo product clinical development. Team composition trends towards two forms – (1) Device oriented teams, and (2) Cross functional teams. Cross functional teams saw greater effectiveness in using reported usability outcome measures from clinical trials to improve products.

• Essential Roles: Key roles represented on cross functional teams include: (1) HEOR representative, (2) Marketing representative, (3) Product representative.

• Interaction of Clinical Development function with other functions for combination product development
• Role of formal cross functional teams and key contributors / supporters
• Leading and supporting role of cross functional teams during late stage trial design & protocol development, late stage outcome measure decisions, and late stage trial execution
• Roles of functions that are responsible or interact with patient feedback data
• Frequency and mix of standing and functional meetings for communication with internal stakeholders
• Utilization of SOPs for patient feedback data collection in late stage clinical trials for combination products and areas addressed in the SOPs

• Type of clinical trial for first testing of to-be-marketed products, and administration site for first testing
• Mode of combination product administration for the selected clinical trial(s) - home use, in-clinic administration, or a mix of both
• Benefits and detriments of home and in-clinic administration; deployment of mixed administration site trials; and average interval for in-clinic visit
• Frequency of use of combination product administration sites among all trial types - home use, in-clinic administration, and a mix of both
• Incorporation of patient, investigator, or observer (i.e. site staff) based device usability feedback in late stage trials

• Collection of usability measures from patients, and primary investigators/ site staff for combination product – total benchmark class
• Collection of usability measures from patients, and primary investigators/ site staff for combination product - injection and auto injector segment

• Types of observer based measures for which data is collected - cumulative use across phases
• Frequency of observer based measures used – total benchmark class
• Frequency of observer based measures used – injection segment
• Frequency of observer based measures used – auto injector segment
• Effectiveness of observer-based measures in characterizing device usability performance – total benchmark class
• Effectiveness score of observer-based usability measures - total benchmark class and injection segment
• Effectiveness of observer-based measures in characterizing device usability performance – injection segment

• Types of patient usability measures for which data is collected – total benchmark class; phases for patient usability measure implementation
• Case study for large scale implementation – company’s formal team, observer usability and patient usability in phase II and phase III
• Case study for large scale implementation - patient usability measures effectiveness