A Sign of Something Larger: The FDA’s Recent Draft Guidance for Oncology Medicines

The Food and Drug Administration (FDA) recently issued draft guidance for the life sciences industry on clinical trial considerations to support accelerated approval of oncology drugs. The FDA asserts that single-arm trials can add uncertainty to the assessment of safety and efficacy, which randomized control trials (RCT) can better address (and, as manufacturers know – RCTs are more expensive to conduct).

But what is the real driving force for this close examination of clinical trial design? Understanding this broader context can help manufacturers best position themselves in the evolving world of FDA regulatory requirements and access & payment models (such as the CMS Innovation Center’s recently announced three test models).

Essentially, after a decade of talk around patient-focused drug development and the importance of the patient voice, these concepts are shaping real programs. Through actions such as this new guidance, the FDA wants to ensure that oncology trials generate data about safety and efficacy that is actually meaningful to patients.

At the end of the day, it can be difficult to keep pace with all these evolving programs and proposals, but here are three areas of focus that will help manufacturers to stay ahead of the curve:

• Meaningfulness to patients. It will be important for manufacturers to keep this at the center of their discussions and decisions. It will be vital to have data to support how products are beneficial to the patient and to be able to talk about specific plans to develop and measure this.
• Build stakeholder relationships early. The reality is that external stakeholders may still regard these data points with skepticism when it comes from the mouth of a drug developer. So, be sure to continuously cultivate relationships with key patients and key opinion leaders to understand and demonstrate a nuanced appreciation for patient needs and how a medicine uniquely fulfills those needs. These stakeholders can provide a perspective or language to support your product while also serving as a credible voice to reinforce your own messaging.
• Take action. Now is the time for manufacturers to consider weighing in on the FDA’s draft guidance and/or mobilize important external stakeholders to weigh in - comment period ends May 26.

Keeping track of this evolving guidance is important to payers seeking to find ways to provide access to novel, high-priced, therapies (like cell and gene therapies).

Interested in learning more? Explore what the Syneos Health Reputation & Risk Management team, including Leigh Ann Bruhn can offer in terms of expertise and insights here.

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