The Off-Label Gap: What Top-Down Forecasts Can't See

Conventional forecasting approach misses out on the “off-label usage” gap

Conventional forecasting methodologies follow a top-down approach, of overall market size estimation and subsequently modelling in brand share within that market. Conventional drivers typically involve market share dynamics, label expansion and similar macro-level levers that shape revenue projections.

Patient based forecasting can be accurate for most of the products, however, for forecasting in-hospital products, relying on standard methodology may result in a less accurate forecast. The challenge intensifies with off-label drug usage in such markets, which creates a gap between approved indications and the on-ground clinical practice.

As off-label prescriptions fall outside regulatory boundaries and tracking mechanisms, it would be difficult to capture the true patient population utilizing the drug. This discrepancy between approved and on-ground usage patterns undermines the accuracy of traditional top-down models for such products, necessitating more sophisticated approaches to validate and account for real-world prescribing behaviours and patient populations beyond labeled indications.

Furthermore, we will try to understand why the traditional forecasting models can be inadequate for capturing its true market potential in case of in-hospital use products.

Real-world usage vs approved label

Regulatory labels outline the approved indications, however the real-world use communicates a different story. One such unique example is the use of 4F-PCC products in the U.S. These products, although approved for urgent reversal of acquired coagulation factor deficiency induced by Warfarin, appear to have substantial utilization as off-label treatments in DOAC (Direct Oral Anticoagulant) and Non-OAC (Non-Oral Anticoagulant) settings.

Therefore, in some cases off-label usage can contribute to business significantly. As the non-OAC and DOAC bleed markets combined are larger than the Warfarin bleed market, the off-label use of 4F-PCC products in these indications can provide an incremental volume, which could even surpass the volume from the approved label. This contradicts the conventional understanding that off-label use is generally a smaller part of revenue and thus creates an opportunity for companies to understand and analyze the critical perspective of the “off-label usage gap.”

Bridging conventional forecasting with contract-level dynamics

In hospital driven markets, it is essential to not only understand the patient pattern or prescribing pattern but also to consider contracting patterns in the forecasting scenario. Market access and contracting dynamics hold potential to differentiate the product in such markets. Modelling in hospital contracts, formulary positioning, competitive contracting and purchasing deals can have a major impact on the product share.

Contract based forecasting provides a realistic view by consideration of contract wins and losses at the account or hospital level. This in turn provides an in-depth understanding of how the dynamics of contract deals can translate into volume shifts. Thus, such forecasting enables companies to directionally understand and target their efforts towards winning newer contracts while seeing the losing contracts.

Pricing strategies, discounts and rebased contract terms can be utilized to win newer contracts, which can provide companies an edge over their competitors. This approach recognizes that, instead of allocating additional spend towards promotional activities, if such companies zoom in more at contract level data, it could aid the product's growth over forecast years.