Leveraging real-world evidence to bridge the access gap in multiple myeloma

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An Expert View from Tomislav Kamenski, Senior Director, Therapy Area Market Access Leader, Hematology, Johnson & Johnson (NYSE: JNJ).

In the field of oncology, we are currently witnessing the most rapid period of innovation in medical history.1 The approval of new cancer therapies, alongside advancements in diagnostics, surgery, and overall healthcare, are offering optimism to individuals facing cancers traditionally associated with poor outcomes, low survival rates, and treatment challenges.1 Take, for instance, the remarkable progress in multiple myeloma. Over the past two decades, survival rates have drastically improved – in some cases, doubling.2 I am optimistic that these encouraging statistics are set to continue improving with the introduction of increasingly targeted immunotherapy agents, such as bispecific antibodies and CAR-T cell therapies, as well as a focus on diagnosing and treating multiple myeloma earlier.3

However, amidst the backdrop of this scientific progress lies a stark reality—a disconnect between innovative therapies and patient access. In Europe, only half of the innovative oncology medicines currently approved are reaching patients, and they are waiting on average nearly 18-months before treatment reimbursement is secured.4 For those battling cancer, this time waiting can be a matter of life or death.

Central to this challenge are the value assessment frameworks within European healthcare systems. These frameworks often require statistical proof of enhanced overall survival to secure favorable reimbursement outcomes.5 Yet, such metrics are no longer feasible when it comes to conditions like early-stage multiple myeloma, where natural disease progression may take years, and patients treated are living longer than the timelines of clinical trials.6,7  Consequently, residual data uncertainties may remain at the time of regulatory approval and can lead to delayed access to potentially life-extending innovations.8 Paradoxically, and rather counterintuitively, the better a drug is in achieving life extension, the longer the delay to access will be.

The potential of real-world evidence as a solution to this challenge

To overcome these barriers and improve patient access to innovation, we must shift towards tangible solutions that address residual uncertainties in clinical data at the time of regulatory approval. Real-world evidence can complement and fill evidence gaps, offering nuanced insights into patient unmet needs, treatment effects, safety and how the treatments are being managed in routine practice - for example, relating to specific subpopulations or long-term follow-up.9 Importantly, this evidence can also provide valuable insights into healthcare resource utilization and costs associated with treatments, helping payers assess the economic value of interventions in actual practice settings.10

Real-world evidence can also be leveraged to support more flexible payment models, such as outcomes-based agreements, which offer a viable way to reward innovation while managing payer concerns regarding data uncertainty.8 These agreements hold manufacturers accountable for demonstrating the post-approval, real-world value of their therapies over extended periods, while enabling patients to reap the benefits of cutting-edge treatments when they need them.10

One of the keys to unlocking the full potential of real-world evidence is in ensuring the quality, availability, and homogeneity of underlying data structures.10 This is because health systems are often fragmented due to differing levels of investment in data platforms, and a wide variance across regions in policies promoting the collection of real-world data.11

For Johnson & Johnson (NYSE: JNJ), initiatives like the Haematology Outcomes Network in Europe (HONEUR) – a federated data network drawing on partnerships with research institutions to analyze real-world data  - underscore a commitment to leveraging real-world data for meaningful insights.10 Similarly, participation in projects like the European Health Data and Evidence Network (EHDEN) signals a collective effort to harmonize electronic health records for robust real-world data research.12

The role of real-world evidence to drive us towards more efficient and personalized healthcare systems centered on patient needs, is significant. Optimizing how we use it requires coordinated action among multiple stakeholders and finding ways of working together on a larger scale. Moreover, early alignment with decision-makers to establish real-world evidence requirements and data standards upfront, may mitigate reimbursement delays. By embracing these principles, we can better harness the vast capabilities of real-world data, with the end goal of ensuring innovative therapies reach those in need as quickly as possible.

References

1 Fasola, G., et al. (2021). Impact of innovation in oncology: more questions than answers. Tumori, 107(6), 478-482.

2 Lopez-Muñoz N, Hernández-Ibarburu G, Alonso R, et al. (2023) Large-scale real-life analysis of survival and usage of therapies in multiple myeloma [published correction appears in J Hematol Oncol. 2023 Jul 25;16(1):81]. J Hematol Oncol. 16(1):76.

3 MayoClinic. Multiple myeloma: Its evolution, treatment and the quest to catch it early. Available at: https://cancerblog.mayoclinic.org/2024/03/05/multiple-myeloma-its-evolution-treatment-and-the-quest-to-catch-it-early/. Last accessed: March 2024.

4 IQVIA. (2022). EFPIA Patients W.A.I.T. Indicator 2022 Survey. Available at: efpia.eu/media/s4qf1eqo/efpia_patient_wait_indicator_final_report.pdf. Last accessed: March 2024

5 European Medicines Agency. (2017). Guidelines on the evaluation of anticancer medicinal products in man. Available at: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-evaluation-anticancer-medicinal-products-man-revision-5_en.pdf. Last accessed: March 2024.

6 Sola-Morales O.T., et al. (2019). Perspectives to mitigate payer uncertainty in health technology assessment of novel oncology drugs. J Mark Access Health Policy, 7:1562861.

7 Morrell, et al. (2018). Will the reformed Cancer drugs fund address the most common types of uncertainty? An analysis of NICE cancer drug appraisals. BMC Health Serv Res,18(393).

8 Dabbous M., Chachoua L., Caban A., Toumi M. (2020). Managed Entry Agreements: Policy Analysis From the European Perspective. Value Health, 23(4):425-433.

9 Bardenheuer K., Van Speybroeck M., Hague C., Nikai E., Price M. (2022). Haematology Outcomes Network in Europe (HONEUR)—A collaborative, interdisciplinary platform to harness the potential of real-world data in hematology. Eur J Haematol,109(2): 138-145.

10 Dang A. (2023). Real-World Evidence: A Primer. Pharmaceut Med, 37(1):25-36.

11 Cavlan, O., Chilukuri, S., Evers, M., and Westra, (2018) Real-world evidence: From activity to impact in healthcare decision making. Mckinsey. Available online: https://www.mckinsey.com/industries/life-sciences/our-insights/real-world-evidence-from-activity-to-impact-in-healthcare-decision-making#/. Last accessed March 2024

12 Innovative Health Initiative. EHDEN: European Health Data and Evidence Network. Available at: https://www.ihi.europa.eu/projects-results/project-factsheets/ehden. Last accessed: March 2024