Patient-centred oncology: transforming drug development

How has the emphasis on patient experience changed the drug development landscape?

Advancements in science and technology have enabled a noticeable shift away from broad histology-based classification and toward molecularly defined market segments. This trend results in more personalised treatment for patients but means that developers must thoroughly research exactly which patients and other stakeholders – not just which type of cancer – are most likely to benefit from their innovation. In today’s volatile, hyper-segmented cancer spaces, a strong value proposition that ties to a real patient’s unmet need is essential.

What changes have the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) implemented to emphasise patient input in drug development?

The FDA and EMA have taken steps to integrate patient-reported outcomes (PROs) and real-world evidence (RWE) into their expectations and processes; for example, through initiatives like the FDA’s patient-focused drug development (PFDD)¹ and the continued involvement of patients in EMA activities.² These efforts help regulators assess whether new drugs truly address unmet needs and provide truly meaningful advantages over existing treatments.

A well-rounded submission package that includes PRO and RWE is becoming the new expectation from regulators and payors, assuming studies were planned and executed with the patient lens in mind and that the data meet the regulatory agency’s standards for quality. The FDA, in the framework for its RWE programme, states that it looks at whether the RWE is of sufficient quality and is designed to answer the regulatory question, as opposed to endorsing one type of RWE over another.³ Therefore, understanding patients’ needs as early as possible in the programme can help developers prospectively build practical endpoints and datasets into the programme strategy. Then, hopefully, the drug will approach registration with a strong patient-centric value story, without the need to course-correct late in the game.

How have scientific and technological innovations contributed to the development of more personalised and effective therapies in oncology?

As noted, the field is moving towards market definitions based on specific molecular pathways or biomarkers; this is a direct result of new molecularly targeted treatment options that make these characterisations clinically actionable. In some cases, the power of targeting a molecular pathway has transcended historical histologic definitions, resulting in molecularly defined pan-histology indications (the FDA approval of pembrolizumab in microsatellite-instability high [MSI-high] cancers was the first notable instance⁴). This shift toward precision medicine has raised the therapeutic bar for new entrants and added tremendous complexity to the way developers must segment a market and define the value of their innovation. Concurrent development and approval of a companion diagnostic (CDx) adds an extra layer of complexity and, in many cases, requires coordination with an external diagnostic partner.

What are the benefits of involving patients earlier in the drug development phase, especially in oncology?

Understanding (and anchoring to) a tightly defined patient group’s experience and unmet need can provide a clear beacon of value for the new asset. Patients with cancer have long been encouraged to have an active role in advocating for their needs compared to other therapy areas, which means that the patient voice is often more powerful in oncology.

Authentic two-way partnership with advocacy groups early on and throughout the life of a programme is the only way for a developer to deeply understand the needs of real patients, anchor their programme to meeting those needs, and benefit from their ongoing input and advocacy. Everyone benefits: the developer gets a more viable commercial opportunity, the patients maximise the impact of their own voice, and society ultimately benefits from a larger step toward better cancer outcomes.

What challenges remain in fully integrating patient perspectives into drug development and commercialisation?

New innovations are constantly changing the standards of care across oncology. Making sure the company’s strategies stay aligned with an ever-changing unmet need can be an overwhelming task, as can adopting such a broad perspective at Phase I, when there are still so many unanswered questions about the drug’s performance.

External strategic partners can help developers adopt and maintain that broad perspective that includes a deep understanding of the science as well as the needs of real patients. They provide the necessary expertise in patient advocacy, medical affairs, market access, and the science of oncology needed to identify patient experience challenges and transform them into opportunities for the company, at a point when internal expertise may not be in place. Although this approach requires upfront investment, it can pay off in the longer term by increasing a developer’s chances of success in volatile oncology markets. 

References

1.CDER Patient-Focused Drug Development. FDA [Internet]. 2021 Jul 27 [cited 2024 Jul]; Available from: https://www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development

2.Patients and consumers [Internet]. European Medicines Agency. 2018 [cited 2024 Jul]. Available from: https://www.ema.europa.eu/en/partners-networks/patients-consumers

3.Framework for FDA’s Real-World Evidence Program [Internet]. FDA. 2018 [cited 2024 Jul]. Available from: https://www.fda.gov/media/120060/download?attachment

4.Subbiah V, Gouda MA, Ryll B, Burris HA, Razelle Kurzrock. The evolving landscape of tissue‐agnostic therapies in precision oncology. CA: A Cancer Journal for Clinicians [Internet]. 2024 May 30; Available from: https://acsjournals.onlinelibrary.wiley.com/doi/full/10.3322/caac.21844