Beyond Registration: The Essential Role of Late Phase Research
Registration research is required by regulators and deals exclusively with safety and efficacy. Late Phase research is, for the most part, voluntary; sponsors choose to do it to develop information that will allow them to add new indications to the label, optimize treatment guidelines, dig out competitive advantages and develop rationales that will win over physicians, patients, advocacy groups, insurers and of course Medicare and Medicaid.
In addition, regulators are demanding more information about hidden safety issues that emerge only after repeated real-world product usage by large, diverse populations over long periods of time. That, too, is a critical objective of Late Phase research.
Unlike Registration research, Late Phase research can cover a wide spectrum of designs, from observational or interventional studies to retrospective studies that analyze existing data.
In observational registries, investigators observe real-world treatment and outcomes over time of numerous patients who have the same disease (a disease registry) and/or are receiving the same treatment (a product registry.) Registries can also be described as prospective open-label observational studies.
Registries can provide valuable data on the natural history of a disease, the clinical or cost effectiveness of a particular therapeutic approach, resource utilization, safety issues and adverse events, quality of life, patient compliance and, ultimately, outcomes.
Observational Registries: Strategies for Success
Premier Research has extensive experience managing these critical factors for success:
- Clearly defining the scientific objectives and other parameters of the study.
- Determining the study size based on the desired precision of effect estimates, feasibility, cost and whether or not the registry is intended to support regulatory decision making.
- Selecting the proper distribution of sites for accurate patient representation. (The study population should approximate the target population as closely as possible.)
- Developing and implementing strategies for patient recruitment and long-term retention.
- Building and sustaining community development using multi-level digital and social media strategies.
- Collecting, verifying and validating data to ensure registry integrity and interim analysis.
- Using call centers to take patient calls and enter data into the eCRF for almost real-time collection. This allows for real time access to the data, as well as taking the burden of data collection away from the sites so they can focus on study assessments.
- Optimizing site management through remote and risk-based monitoring.
Premier Research provides a full range of services for all types of Late Phase studies, from study planning, logistics and recruitment to site management and data processing and evaluation—a critical issue due to the massive volumes of data developed in large-scale Late Phase studies.
We can help you simultaneously streamline and optimize the value of your next Late Phase study.
Late Phase Services
- Ongoing Pharmacovigilance Studies
- Risk Management
- Comparative Effectiveness Research
- Observational (Non-interventional) Studies
- Health Economics Research
- Patient and Disease Registries
- Health-related Quality of Life Studies
- Expanded Access Programs
Potential Benefits of Late Phase Studies
- Generate knowledge and evidence that appeals to payors, patients and physicians
- Demonstrate effectiveness or comparative effectiveness in different populations
- Meet post-marketing commitments
- Generate evidentiary requirements for coverage
- Meet regulators’ demands to monitor long-term safety and safety in different populations
- Manage risks and benefits
- Generate data for new indications or label extensions
- Obtain or expand market access
- Change behavior
- Improve quality of care
- Prove product value through economic or quality of life benefits
- Bring scientific information to stakeholders sooner
- Enhance the practice of personalized medicine
- Understand the natural history of disease and treatment
- Generate evidence for product differentiation and positioning