In the first part of this series, we discussed how real-world evidence (RWE) helps mitigate risk and demonstrate value to stakeholders, as well as minimize unnecessary costs during the drug development process. In part two, we shift to how RWE can build your value story, improves treatment guidelines and places power in the hands of patients as their input becomes increasingly valuable during drug development.
RWE can redesign the patient care pathway
By using RWE to tweak, or even re-design the patient care pathway, developers can work with providers and payers to evaluate treatment guidelines, potentially reducing costs and improving care.
When Investment Rhymes with Canada
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For example, a client recently received EMA approval for a novel therapy. Despite positive recommendations from national payers in Europe, the drug was not widely regionally reimbursed. Due to its perceived high cost, the product was only used as a last-line treatment. PAREXEL performed a landscape assessment, finding a gap in how patients were being followed across primary and secondary care. Patients were treated with a first-line therapy in hospitals and then released into the community as though the treatment had worked. When they re-presented without improvement, they were logged as a new patient and treated again with second-line therapy. It was not until a patient presented a third time, that they were considered for the new drug. The value story of the product was compromised because providers were focused on the cost of buying the drug, not the overall economic burden of an inefficient patient journey involving costs and retreatments.
In collaboration with local providers and payers, who were interested in developing new care pathways, PAREXEL designed a RWE study where the product was given as a first-line treatment, rather than third-line. Patients were tracked to determine overall cost of treatment from initial presentation to cure, along with outcome measures. After 18 months, the data demonstrated that initial treatment with the product was more cost-efficient than using it as a third-line treatment and improved outcomes.
From this example, we see that data from well-designed RWE studies are generalizable and interpretable. The pilot study led to a new model of care that informed local clinical practice and decision making, and led to a change in National Guidelines in the therapy area and access to the product.
However, studies that examine patterns of care require adequate infrastructure to track patients across primary and secondary care. The skills required to plan and execute RWE studies analyzing patient care pathways include:
- Expert clinical guidance;
- Commercial strategy;
- Landscape assessment;
- Predictive health economic research modeling;
- Stakeholder engagement;
- Data capture;
- Results communication
Power is shifting to patients and to patient-relevant outcomes
The patient’s role in overall drug development, and RWE specifically, is expanding as regulators, payers and other critical stakeholders increasingly focus on patient perspectives. EMA Executive Director Guido Rasi acknowledged this when he cited “patient involvement” as one of the agency’s top priorities through 2020.
Consequently, patient advocacy groups are becoming essential to RWE planning. Companies should engage these groups in steering committees and panels to gather market intelligence and input on defensible patient-reported outcomes. In addition, national payers increasingly cite patient-reported outcomes in coverage decisions. In 2013, lung cancer drug Xalkori won the second-highest benefit rating from IQWiG despite showing no advantage in overall survival (regulatory/RCT gold standard of efficacy). German evaluators agreed patient-reported outcome measures showing the drug’s impact on morbidities in the target patient group offered a hint of considerable added benefit. Quality of life metrics showed improvements not only in patient well-being, but also in symptoms such as trouble breathing and coughing, illustrating a disease-specific and standardized QoL instrument tracking common symptoms could provide powerful evidence for adoption.
In 2015, the UK’s NICE agreed to cover Vimizim to treat the ultra-rare condition mucopolysaccharidosis type IVa (MPS IVa). Despite doubts about efficacy, NICE ruled positively, in part after hearing from patients, experts and PAGs about Vimizim’s “substantial benefits” that weren’t captured by the official, prospectively specified primary endpoint used in pivotal clinical trials as a proxy for endurance.
NICE gave weight to evidence from cross-sectional surveys in which patients and families said the effect of MPS IVa on QoL, and its effect on caregivers, are related to how much the patients rely on wheelchairs. The survey revealed MPS IVa patients required up to 15 hours of care per day, causing caregivers stress, lack of sleep, back pain, anxiety, and depression, with negative effects on family social life and finances. Families provided anecdotal evidence that some children receiving Vimizim continued to grow, experienced less physical deterioration, avoided surgery and remained independent of a wheelchair.
By engaging in the NICE review process, patients and their families helped define and validate a real-world endpoint.
Investing in a smart, tailored RWE plan from the beginning of drug development may seem expensive and premature, but it can lead to significant cost savings.
Some companies worry that RWE will not show the same magnitude of benefits as RCTs. But sponsors who include credible RWE in their development plans are being viewed more favorably by payers.
RWE should demonstrate the added-value a new technology can bring, what outcomes it can achieve, and what cost-savings are possible. Success demands integrating evidence collection, analysis, and medical communications throughout a product’s lifecycle. Effective integration can come from within, or through coordination with external experts.
Photo: Pixtum, Getty Images
