Improving human health is the ultimate aim for all participants in the provision and receipt of healthcare. Patient-centered medicine changes the emphasis of medicine from a 'one size fits all' solution to one that engages with individual patients and empowers them to take back a degree of control of their own health. Patient-centeredness has the potential to create better clinical outcomes, improve quality of care, optimize resource utilization and reduce system-wide costs.
For biopharma, patient-centeredness involves realigning departments from existing goals such as achieving regulatory approval, favorable pricing terms and reimbursement to a unifying goal of improved patient outcomes. A one-goal approach facilitates greater departmental cooperation ensuring that all teams are driving towards the same destination. It is never too early to bring a patient-centric approach into drug development and market access, and the following nine steps provide a practical guideline on how to do so.
1. Appointing dedicated leadership of patient-centeredness
Appointing the right senior people with extensive experience to drive patient engagement from the early stages of the research and development cycle will help to keep all teams focused on the importance of a patient-centric approach. This is crucial, from choosing the indication and lead clinical candidate in preclinical trials right through to stakeholder engagement and patient-support programs after drug approval. Many companies are now showing best practice in this area with dedicated senior appointments who have a singular focus on patient-centeredness.
2. Conducting early development real-world studies
Capturing real world data, beginning early on in the drug's lifecycle, informs both the development program as well as future market access. This includes understanding the burden of disease, from both a social and an economic perspective, which will provide information on resource utilization and healthcare costs. Knowing more about the patient characteristics, at population, sub-population and individual levels, and treatment pathways, will shed light on how a treatment may be best used by patients, carers and physicians.
3. Engaging with regulators and payers
By starting to plan how to measure and analyze patient-centric outcomes earlier in the drug development cycle, companies have the opportunity to seek advice from regulatory agencies, health technology assessment bodies and payers. This will help the drug developers to understand which tools and outcomes will provide the best support and value in approval and reimbursement decisions, potentially allowing faster treatment access for patients, as well as a quicker return on investment for the company. It is important that any companion diagnostics are also approved during the same timeframe as the main treatment and early external guidance can ensure that this happens.
4. Engaging patients at all stages of development
Patients are undoubtedly the best group to guide their needs. There are examples of where products have failed simply due to lack of patient demand. If patient groups were drawn in earlier, losses could have been cut by an earlier go/no go decision, or a design created that fitted in more closely with patients' needs and lifestyles.
5. Generating the right data in the right patients
It is important to generate evidence in patients representative of the target population who are likely to use the product. Lack of patient representativeness has been cited by regulators and payers as an issue. Early real-world studies that profile patient populations can guide patient selection for clinical trials and post-marketing studies ensuring that the data produced is relevant.
6. Capturing relevant patient outcomes
Researchers need to ensure that they are collecting pertinent patient-reported outcomes in addition to clinical end-points in the pivotal trials. On an individual level, this could include symptom relief, improved functional status, or slowed deterioration. On a community level, this could include better clinical outcomes; on a population-wide level, improved quality of care, and lower healthcare costs.
7. Developing or selecting measures to evaluate patient outcomes using established qualitative and quantitative methods
Once relevant patient outcomes have been identified these need to be carefully measured and evaluated, using validated qualitative and quantitative tools and methods. This should be completed before pivotal trial design to ensure appropriate selection of key and exploratory patient-centered end points, adequate understanding of how measures will perform, anticipated effect sizes, and adherence to regulatory guidance. It is important also to consider companion diagnostics, the use of biomarkers and genetic profiling which play a crucial role in the management of conditions such as cancer.
8. Including PRO and other patient-centered measures in pivotal trials
The validated PROs and other patient-centered measures can now be included in pivotal trials, with protocol-specified plans for statistical analysis as well as minimizing and handling missing data. Once captured, the patient-reported outcomes can be used in drug labels to help patients, caregivers and healthcare professionals make the right choices.
9. Building on the right outcomes and the right drugs for the right patients out in the real world
Steps 1 to 8 ensure that a product comes to market with a patient relevant profile but real-world evidence gaps remain. It is important to evaluate product use in the real-world: safety & effectiveness; off-label use; compliance, adherence, persistence; treatment satisfaction using robust research methods. Patient programs may be needed to provide education and support for new therapies. Patient focus groups and surveys are helpful for future product enhancements.
We know that patients need affordable medicines to meet their unmet medical needs with predictable outcomes and few side effects. That doesn’t mean that we know what patients want. A patient-centric approach to drug development and market access shines light on what is most important, the patient voice.
If you want to learn more about including the patient voice in global market access strategies you can view the recording from our recent webinar here
1. Mack, GS (2007) Pfizer dumps Exubera. Nat Biotech 25: pp. 1331-1332