On September 29, 2021, The Food and Drug Administration (FDA), released a proposed benefit-risk assessment for new drug and biological products. The document, published in draft guidance form, shines a spotlight on the FDA’s decision-making processes.
In particular, it provides key insights that trial sponsors, patient advocacy organizations, and other stakeholders can use when considering the benefits and risks of new drugs or biological products early in the stages of development. You can read the entire document by clicking here.
The FDA’s benefit-risk assessment covers various topics, but its main focus is the structured planning that trial sponsors should take to reduce uncertainty and demonstrate a specific product’s favorable benefit-risk profile. While this goal might seem vague, the draft document makes 4 specific suggestions. Let’s take a closer look:
1) Trial sponsors must engage in benefit-risk planning throughout the lifecycle of every drug or product. Considering that more than 30% of Phase II drugs and more than 58% of Phase III drugs fail, benefit-risk planning should never be an afterthought. The amount of time, money, and effort dedicated to developing new drugs and therapeutics requires all stakeholders to be on their A-game all of the time.
A thorough benefit-risk assessment considers every aspect of the trial process, including the identification and recruitment of patients, establishing controls to identify the benefit-risk profile, and monitoring of anticipated serious adverse events.
2) Trial sponsors must pay special attention to the patient experience. The FDA’s new draft guidance expresses support for the Center for Biologics Evaluation and Research’s (CBER) belief that all benefit-risk assessments should consider the patient experience.
By incorporating patient feedback into drug development and evaluation, researchers and pharmaceutical companies can access invaluable insights. What’s more, by encouraging patients to document and share their treatment experiences, it’s possible to build trust and increase public confidence in the safety and efficacy of new drugs and therapeutics.
3) Trial sponsors should seek to minimize bias and maximize precision. Even though the FDA is sharpening its focus on improved benefit-risk assessments, that doesn’t mean it's willing to accept faulty research or bias.
The agency’s draft guidance reinforces this message, by noting that the approval standard for all drugs, including rare diseases –– remains the same –– substantial evidence. To achieve these goals, all trial sponsors and stakeholders are encouraged to incorporate specific trial design features, including randomization, blinding, and adequate trial duration. Controls like these can reduce the risk of junk science and poor-quality data.
4) Guidance on post-approval benefit-risk considerations. Last, but certainly not least, the FDA’s guidance document provides instruction on benefit-risk considerations that occur after a drug or biological product receives approval. Areas of interest include how to collect and share important information with patients post-launch and how to alert users of potential safety risks.
What part of the FDA’s draft guidance on benefit-risk assessment is most interesting to you? Is there anything, in particular, that stands out? If so, we’d love to hear your thoughts. Please fill out a comment form below and then click submit.
If you’d like to respond to the FDA’s draft document, you can do that too. Click here and then click the blue button that says “submit comments online.”