EU and US regulators are seeking to enable early access to novel treatments for patients with severe unmet need. In the EU, the Medicines Adaptive Pathways for Patients (MAPPs) shown in Figure 1 is an initiative looking at ways to enable early access including:
- Flexible development and access pathways within the current regulatory framework that balance early patient access, public health and societal benefits.
- Early authorization of a product focused on a well-defined and targeted population with a clear safety and efficacy profile.
In collaboration with NICE, MIT and NEWDIGS, Exploristics examined how the MAPPS concept could be applied in development, evaluating the benefits and risks of a treatment, and the commercial impact of the development options on the treatment.
Figure 1. The MAPPs Development pathway.
Information on a marketed treatment for relapsing Multiple Sclerosis (MS) was used by KerusCloud to generate realistic patient level data in silico and design alternative MAPP development plan scenarios in a virtual environment to:
- Identify a higher benefit moderate & severe (M&S) sub-population for early authorization and real-world evidence collection.
- Continue to develop the All-comers indication with no launch delay.
- The outputs from KerusCloud including probability of success and sample size were used as inputs for commercial forecasting.
- The outcomes of simulated scenarios in KerusCloud broadly supported the MAPPs approach and demonstrated that:
- The MAPPs design would provide sufficient evidence for approval in a larger Phase II trial.
- The Phase II trial would need to randomize 450 patients rather than 240 in the original design.
- Initial approval could be in 5 rather than 8 years.
- Financial modelling of the impact of MAPPs for Patients, Payers and Sponsors (Figure 2) indicated an increased expected Net Present Value (eNPV) of $600M rather than $460M for sponsor.
Figure 2. Financial modelling of the impact of MAPPs for Patients, Payers and Sponsors
With KerusCloud it was possible to show that:
- The MAPPs approach can provide access for patients up to 3 years earlier.
- MAPPs was beneficial for developer and payer economics.