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As real-world evidence (RWE) is increasingly being used to inform guidance and approval decisions by regulators, health technology assessment (HTA) bodies, and payers across the US, Europe, and other jurisdictions, biotech and pharma are investing more into generating real-world data (RWD).
Disease registries: a valuable source of RWD
Disease registries are a particularly valuable source of RWD in that they:
a) allow for the prospective capture of fit-for-purpose demographic, clinical, and patient-reported outcomes (PRO) data.
b) can be built to recruit globally from the onset, thereby facilitating the inclusion of a larger and more heterogenous participant population than may be possible when using other institutional sources of RWD.
c) are usually open-ended and can provide long-term outcomes from a typical care setting.
Figure 1: Disease registry data use cases across the product lifecycle
*Abbreviations: R&D – Research and development, HRQoL – Health-related quality of life
Value of early disease registry data analyses
Descriptive analysis of early registry data can be particularly valuable in addressing common challenges in disease research and drug development¹, particularly in rare and orphan diseases, such as:
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- understanding natural history and progression of the condition
- describing the range of and gaps in current treatments
- understanding patients’ health-related quality of life and preferences
- serving as external control arms for clinical trials
- identifying suitable participant pools for recruitment to different types of studies e.g., clinical trials and effectiveness studies.
Start early and make the most of your investment
Establishing a robust disease registry early on could be a hugely valuable investment for all stakeholders. It will maximize the potential insights from RWD across the drug development life cycle. McKinsey estimates the potential return on a well-applied RWE generation strategy to be $300 million per year for an established pharma company².
Return on investment for a disease registry relies on it being well-built. Potential issues can limit the ability to fully leverage data for insights and decision-making – key considerations include ensuring that:
a) data collection is patient-centric, thereby minimizing burden on patients and healthcare professionals.
b) data are fit-for-purpose – meaning that they are of good provenance and of sufficient quality and relevance to address the research question.
It is important to take into consideration input from different stakeholders such as industry, healthcare professionals, and patient representatives.
Conclusion
At Vitaccess, our experience recommends investing in building disease registries early in the drug development process and involving experienced partners in order to gain valuable insights and maximize the return.
Interested? Get in touch today at info@vitaccess.com
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By Tsitsi Monera-Penduka, Fatemeh Amini and Emma Bagshaw
