For any medical condition, the care pathway is the patient’s journey through the healthcare system.
It is important for pharmaceutical companies to understand the care pathway, including the relevant patient population, the most relevant comparators and the potential drivers of effectiveness, which may create a gap between efficacy and effectiveness. This should inform the phase 3 trial design, but will also help to understand who to ‘market’ the medicine to once it has been authorised for use. The care pathway may vary in different jurisdictions and countries, so this should be explored for each place of interest.
It is also important for health technology assessment (HTA) agencies to understand where in the care pathway a medicine may be used, so they can understand the most relevant comparators and assess the potential impact of a medicine. This will usually be explored during the scoping stage (or before scoping, when liaising with a company who has requested scientific advice).
How to explore the care pathway
Activities to describe the patient journey are cross-functional and may include using:
- review of relevant existing guidelines or recommendations from HTA bodies and medical associations
- market research with clinicians
- patient surveys
- payer interviews
- focus groups
- advisory boards (involving clinicians, patient and patient associations, and payers)
- information obtained from social media.
These activities allow different aspects of the care pathway to be explored, such as the points of care; the sequence of events in a patient’s journey; what matters to patients, caregivers, healthcare professionals and payers; and the points at which there may be an impact on effectiveness.
The figure below describes a typical care pathway for a patient. Each node on the pathway represents a different stage in the patient’s journey. At each stage there are choices to be made by the patient and clinician. Patient preference, healthcare system restraints and clinical preferences will all have an impact on the decisions made. At nodes 4 to 7 in particular, the effectiveness might be affected by the decisions made within the pathway.
Figure. Stages in a typical care pathway (source: internal materials, Novartis)
After initial activities to explore the care pathway, quantitative analyses can be carried out to generate deeper insights into any unmet needs and patient subpopulations that may benefit most from the new treatment. This may involve:
- pharmacy claims data analyses
- electronic medical record database analyses
- retrospective patient chart reviews
- analysis of registry data
- cohorts or longitudinal studies
- epidemiology or treatment pattern studies
- burden of illness studies.
These activities can help describe patient characteristics in real-world clinical practice, and how they differ (or not) from the patients studied in the registration trials. Knowing the drivers of effectiveness in clinical practice can help predict the effectiveness of new treatments from the efficacy observed in randomised clinical trials.
Alexandre Joyeux, Novartis
Robert Olivaris, Sanofi