Given the complexity and the many unknowns of NASH, pursuing a traditional approach to evidence generation, e.g. by combining RCT data with existing literature, will not be sufficient to provide robust answers and convince key healthcare stakeholders. And while Real-World Data (RWD), in principle is uniquely suited for facilitating a deeper understanding of NASH, the existing data landscape is not yet fit-for-purpose to support such efforts. This is especially in Europe, where the absence of relevant, off-the-shelf data sets, the generally poor coding of NASH diagnoses, and inconsistent data capture across different settings of care create more barriers than solutions.
In light of the sheer scale of this gap between stakeholder needs and available data in NASH, simply relying on one-off studies or setting up a long-term registry will be inadequate. There is also a risk that any solution will not be sustainable, given the evolving nature of evidence needs over time.
This situation calls for an innovative and agile solution, and presents an opportunity for first movers to gain a head start by making early investments in shaping the data landscape in NASH.
Early successes include building an algorithm to identify risk factors in NASH patients, and the development of a network approach to RWD, with the aspiration of establishing a pan-European collaboration of relevant NASH data source owners (e.g. NASH registries and liver specialist centers.) Ultimately, the future data landscape in NASH will emerge via a combination of efforts led by individual players as well as multi-stakeholder initiatives (e.g. LITMUS in Europe and NIMBLE in the US).
Considering the lead times involved in building a novel evidence infrastructure, as well as the fierce competition in NASH, it is critical for pharmaceutical companies to make important choices now, far earlier than they would typically do, or feel comfortable with. This can also ensure competitive advantage, establishing an approach which other manufacturers cannot easily replicate. Success will ultimately require:
- Understanding individual healthcare stakeholders’ specific evidence requirements, the burden of proof they are expecting (what is ‘good enough’?)
- Targeting investments on prioritized, consolidated evidence needs in the context of clinical development plans.
- Forward thinking about how their evidence portfolio will grow and be enhanced over time, in line with the evolving needs of key stakeholders.
Ultimately, engaging a strong partner early in the development of a NASH evidence strategy is essential for pharmaceutical companies to secure a stake in shaping the RWD landscape with a purpose, to accelerate evidence generation and gain competitive advantage in the evidence race.