Article
Navigating the Digital Frontier
Unleashing the Power of Real-World Evidence Studies with Clinigen's Innovative Approach
In clinical practice, real-world data (RWD) is a formidable asset derived from experiences outside the controlled environment of randomised control trials (RCTs). This dynamic resource can form the basis for well-informed decisions among healthcare providers, Regulatory bodies, and Pharmaceutical/Biotech companies, supporting the integration of safe and effective therapies into established clinical practice guidelines verified by real-world evidence.
Considerations in RWD Collection Design
When delving into RWD collection, a well-structured protocol becomes crucial, offering parameters that differ from traditional randomised control trials. Various factors, such as Informed consent, a cornerstone of ethical research, take centre stage and require a patient-friendly template integrated seamlessly into digital platforms. Ethical considerations surrounding data capture is another crucial element to consider, and should be defined as part of the protocol development.
Source and Value of RWD in Managed Access Programs
Exploring various avenues for RWD sources, from medical records to existing databases, underscores the importance of building relationships within clinical sites. Establishing Managed Access Programs (MAPs) with a data component emerges as a multifaceted approach to enhancing healthcare outcomes. These programs validate clinical trial outcomes, address knowledge gaps, and provide insights into diverse patient subgroups. Furthermore, they contribute to identifying safety signals, capturing patient experiences, and assessing drug effectiveness in new patient groups.
Additionally, RWD can play a significant role in the development, growth, and mature phases of a drug’s lifecycle and can support the development of treatment pathways while identifying areas of future focus.
Critical Considerations for Data Collection
When engaging in data collection in early access programs, two key considerations should guide the process. Firstly, it is crucial to evaluate the purpose of the data: Is it addressing any existing evidence gaps? Understanding the evidence gaps of the clinical trials ensures a systematic approach in the selection of data points to be collected during the early access programs. The pharmaceutical industry should bear in mind that data collection during the early access programs is driven by HCPs. Overburdening the HCPs by trying to collect too much will potentially result in not being able to get the benefit from the data. Clinigen suggest sticking to standard of care related data points for successful outcomes.
Maximising MAP Value in Market Access with Real World Data
Managed Access Programs (MAPs) significantly contribute value across four key sectors:
- Data Enhancement: For example, when planned correctly, the data captured from MAPs can play a pivotal role in addressing critical gaps in evidence or act as support for data captured in the clinical trial environment. Additional information can also help identify new areas of research or focus.
- Clinical Experience Generation: MAPs provide valuable experience to healthcare professionals, developing them as experts with specific products and the associated care, building future support and advocacy but also enabling engagement and insight via a consultative approach with regards to future development and market access.
- Patient-Expert Collaboration: Enabling early access via a MAP often warrants engagement and participation of patient organisations. A good level of engagement with patients and patient-centred organisations can, amongst other things, support quality data capture. Patient-reported outcomes and the unique perspectives they represent are increasingly important in the navigation of the regulatory landscape and successful launch.
- Service Design Improvement: A combination of patient-reported and clinical data, both in the real world setting, can help manufacturers and regulators best plan for product launch impact and assess any additional needs, services or patient considerations.
Clinigen’s Strategic Recommendations for RWD
Clinigen's strategic recommendations for RWD underscore the importance of early planning and cross-functional collaboration. Clear goals for data use guide the design process, emphasising minimal data collection and the creation of a Master RWD protocol. Aligning with ethical principles, incorporating patient-reported outcome measures, and budgeting for healthcare professional compensation ensures a comprehensive and compliant approach to real-world data utilisation.
Through carefully and specifically designing RWD capture as part of a MAP, we are able to see the true value of RWD in the early access setting, and we are filling knowledge gaps and amplifying the voices of diverse patient subgroups. These programs, anchored in ethical principles and designed with precision, serve as beacons illuminating the path toward more personalised and effective treatments.
Contact us to learn more about Real World Data collection within a Managed Access Program and to find out how we can support your project.