Patient-Centered Oncology: Transforming Drug Development

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

Advances in science and technology have enabled a significant shift away from broad histology-based classification and toward molecularly defined market segments. This trend is leading to more personalized treatment for patients, but it also means that developers must closely examine which patients and other stakeholders – not just which cancer type – are most likely to benefit from their innovation. In today's volatile, hyper-segmented cancer space, a strong value proposition that addresses the unmet need of a real patient is critical.

What changes have the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) implemented to emphasize patient involvement 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 such as FDA's Patient-Centered Drug Development (PFDD).¹ and the continued involvement of patients in EMA’s activities.² These efforts help regulators assess whether new medicines truly address unmet needs and offer truly meaningful benefits over existing treatments.

A comprehensive submission package that includes PRO and RWE is becoming the new expectation of regulators and payers, provided the studies were designed and conducted with the patient perspective in mind and the data meet the regulator's quality standards. FDA states under its RWE program that it will consider whether the RWE is of sufficient quality and designed to answer the regulatory question, rather than recommending one type of RWE over another.³ Therefore, understanding patient needs as early as possible can help developers prospectively build practical endpoints and data sets into the program strategy. Then, hopefully, the drug will arrive at approval with a strong patient-focused value proposition without the need for course corrections later in the game.

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

As mentioned above, 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 characterizations clinically actionable. In some cases, the ability to target a molecular pathway has exceeded historical histological definitions, leading to molecularly defined pan-histological indications (the FDA approval of pembrolizumab in high-grade microsatellite instability [MSI-high] Cancer was the first notable case⁴). This trend towards precision medicine has raised the therapeutic hurdles for new market entrants and added enormous complexity to the way developers must segment a market and define the value of their innovation. The simultaneous development and approval of a companion diagnostic (CDx) adds an additional 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) the experiences and unmet needs of a narrowly defined patient group can be a clear sign of the new product's value. Cancer patients have long been encouraged to play an active role in representing their needs compared to other therapeutic areas, meaning that the patient voice often carries more weight in oncology.

Only through a true two-way partnership with stakeholders at the outset and throughout the life of a program can a developer truly understand the needs of real patients, tailor their program to meet those needs, and benefit from their ongoing input and engagement. Everyone wins: the developer gains a more viable commercial opportunity, patients gain a greater voice, and society ultimately benefits from a bigger step toward better cancer outcomes.

What challenges remain to fully integrate the patient perspective into drug development and commercialization?

New innovations are constantly changing the standards of oncology. Ensuring that company strategies align with an ever-changing unmet need can be an overwhelming task. This is also true of taking on such a broad perspective in Phase I when there are still so many unanswered questions about the drug's efficacy.

External strategic partners can help developers develop and maintain this broad perspective, which includes a deep understanding of the science as well as the needs of real patients. They have the necessary expertise in patient advocacy, medical affairs, market access, and oncology science to identify patient experience challenges and turn them into opportunities for the company at a time when internal expertise may not be present. While this approach requires initial investment, it can pay off in the long run by increasing a developer's chances of success in volatile oncology markets.

References

1.CDER – Patient-Oriented Drug Development. FDA [Internet]. 27 July 2021 [cited 2024 Jul]; Available at:

2.Patients and consumers [Internet]. European Medicines Agency. 2018 [cited 2024 Jul]. Available at:

3. Framework for the FDA Real-World Evidence Program [Internet]. FDA. 2018 [cited 2024 Jul]. Available at:

4.Subbiah V, Gouda MA, Ryll B, Burris HA, Razelle Kurzrock. The evolving landscape of tissue-independent therapies in precision oncology. CA: A cancer journal for clinicians [Internet]. 30 May 2024; Available at: