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Defining what counts as a treatment breakthrough in CNS

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At a glance

We helped a top five pharma client decide which CNS drugs in their pipeline were worth investing in. In a disease area where real innovation has been rare, they needed a clear definition of what would actually count as a breakthrough. We anchored that definition in what matters most to patients day-to-day, rather than in the clinical measurements typically used in clinical trials. That gave the client a confident way to prioritise where to invest across key European markets.

The challenge

The CNS category has seen little real innovation for decades. Existing treatments handle the main symptoms but leave patients with significant residual burden, and physicians have come to expect little from new therapies. The client needed to know where genuine opportunity lay: what level of improvement would actually matter to physicians and patients, and what evidence it would take to shift entrenched prescribing habits.

Our solution

We shifted the lens away from symptoms and onto how physicians judge patient progress in real life. Working with neurologists across three European markets, we ran scenario-based interviews built around realistic patient cases, asking physicians to walk through how they would assess, treat and re-evaluate each patient over time.

We then used those scenarios to pressure-test future drug concepts — exploring what it would take for physicians to change their prescribing. The result was a clear, decision-ready framework the client could use to evaluate every opportunity in their pipeline.

The impact

The client could clearly see which pipeline opportunities to pursue: those that could significantly improve patients’ day-to-day function compared to current standard-of-care treatments. The work shaped early clinical decisions, such as which trial endpoints to prioritise and how to position future therapies.

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