Sharpening the Blunt GPP Instrument: A call for focused stakeholder engagement in designing the efficacy trials of the future

January 10, 2019

Stacey Hannah is AVAC’s Director of Research Engagement. Ruth Assefa is an AVAC consultant, aspiring medical student and formerly AVAC’s program coordinator for research engagement.

Is HIV prevention research seeing its last placebo-controlled efficacy trials? Will the field have to radically reimagine how to answer efficacy questions?

HIV prevention research is indeed set to see changes in clinical trial conduct. Successes in research and implementation are making it easier for many people across the globe to protect themselves from HIV—and, at the same time, harder for researchers to test new interventions. Trials of the future might be bigger, more expensive, and require new types of analysis. As researchers and regulators navigate this territory, they’ve made a call for community input. Attendees recently heard this call at HIV Prevention Efficacy Trial Designs of the Future, a symposium convened by a group of leaders in prevention research in November to discuss new approaches to trial design.

With equations, statistical models, and new trial-design concepts, these were complex conversations indeed. And community input did have a place at the symposium. Jeremiah Johnson of the Treatment Action Group gave a plenary articulating the priorities of some communities, and the day’s panel discussions each included a community representative.

It was a good start, but only a start. As Jeremiah made sure to say, a white, cis-gendered man can’t represent all communities who will be affected by new and complex trials in HIV prevention research.

At AVAC, we’ve given a lot of thought to how to sharpen implementation of the guidelines on Good Participatory Practice to get the field beyond the mere rhetoric of community engagement and towards a strategy-driven engagement of stakeholders. A critical first step for researchers and civil society alike is to define the ongoing role community representatives will have as issues arise from evolving trial design. Will engagement focus narrowly on securing broad support for trials—or something more? (We hope the latter.) Specific objectives of engagement will certainly develop and change with the issues, but they need to be articulated clearly and planned for accordingly. And this should be a two-way street. Those who lead trials have a duty to articulate what they need and want from affected communities and stakeholders, but those stakeholders have an equal duty to probe the issues and articulate their priorities.

What should advocates be thinking about now to prepare for this issue in 2019? For some time, prevention researchers have discussed the possibility of screening trial participants before randomization to identify individuals who may be more or less able to use a given product. Some years ago, microbicides researchers actively explored the idea of screening cis-gender women after enrollment and before randomization to identify those likely to be adherent to a product, since low use can return a low-efficacy result that doesn’t reflect the actual prevention benefit of a product.

Now the question is whether it is feasible, acceptable and ethical to randomize participants who are low adherers, or who don’t want to use a daily product like tenofovir-based oral PrEP, into a trial arm where daily oral PrEP wouldn’t be a part of the control arm. (In all trials, all participants should be provided with a comprehensive prevention package that includes the offer of or referral for PrEP wherever it is available; even in these trials, all participants would be free to choose PrEP on their own. For a lexicon on emerging issues in HIV prevention trials, checkout AVAC’s HIV Prevention Trial Terms: An advocate’s guide.) Communities and advocates will have critical and unique insights into whether affected communities might support such a design–or whether it might provoke controversy—and how such a trial might be implemented.

Discussions and decisions should be as locally relevant as possible, and one can expect they will be moving to Southern and East Africa, where a bulk of these new, innovative trials for next-generation products will happen. HIV-focused civil society networks at country and regional levels need to be involved with trial leaders, product developers, statistician and ethicists in providing input and engaging constituents about these and other issues.

Check out our most recent podcast episode on Px Pulse for an in depth discussion exploring advocate views on this fast-moving issue. Stay tuned for more AVAC resources to help advocates navigate the ins and outs of the trial designs of the future.