Issue 1
It is not rocket science: Less substantiated value propositions for molecules that launch with Phase II data translate into less positive benefit assessments by national agencies.
Payers are clear: Companies seeking pricing and reimbursement for molecules based on Phase II trials using surrogate endpoints, reduced patient enrollment, early crossover, and single arms often have a greater chance of obtaining results with less favorable pricing, reimbursement level of use, and management of restrictions.
Atypical Pathway includes EMA CMA – Conditional Marketing Authorization and other. G-BA – Gemeinsamer Bundesausschuss Schriftenreihe : Interdisziplinaere Plattform zur Nutzenbewertung, Heft 3, Sept 2016 ISSN 23 64 – 916X
Issue 2
In 2017, payers across the EU and US have shown significant concern when molecules are submitted for pricing and reimbursement with a value proposition supported only by Phase II data, specifically in the areas of oncology and CNS.
Level of unmet need and seriousness of condition do not justify making a new treatment available to patients with data that does not fully substantiate a value proposition.
Evidera Payer Investigation in 2017: 60 Payers across US and GER, FR, ENG, IT and SP. Mapping reflects the average of all payer responses per market.
Issue 3
Trial challenges meet payer and HTA requirements: Launching with Phase II data is like walking into an anticipated battle.
When launching with Phase II data, prepare yourself for battle by being aware of payer concerns about data uncertainties – ambiguity of clinical improvement, inconclusiveness of consequences of adverse events, incertitude of the individual patient benefit – and the possible impact on payer decisions.
EU: Opportunities to address challenges via Early Scientific Advice or Adaptive Licensing must be early (e.g., PI, early PII) and thus create data insecurity and development uncertainty; US: parallel review by FDA and CMS was originally intended to include drugs and might at some point though only device and diagnostics makers have used the approach; drug makers are free to approach US payers one by one for early consultation
Issue 4
According to payers, data uncertainties of single-arm trials and surrogate endpoints present the biggest blockage to value determination of molecules seeking price and reimbursement based on Phase II data.
Phase II trials were never intended to be the single, value-driving evidence source for supporting a value proposition for pricing and reimbursement.
It’s a fact: Payers and HTA organizations won’t accept data uncertainties that would force them to hypothesize, at best, the incremental benefit, cost-effectiveness, or the full patient benefit of the treatment.
Evidera Payer Investigation in 2017: 60 Payers across US and GER, FR, ENG, IT and SP. Mapping reflects the average of all payer responses per market.
Issue 5
Early strategizing and planning for market access at regional and local levels is even more imperative for molecules that launch with Phase II data than for regular launches.
National and regional payers are transparent about requirements for benefit assessments. When launching with Phase II data, manufacturers need to plan early for mitigating gaps in evidence that lead to only partial substantiation of the value proposition.
Mitigation options targeted at the national level may include: chart reviews linking surrogate to outcome (done in parallel to trial), database studies on SoC + outcomes, validation of surrogates, and naturalistic studies accompanying trials.
Mitigating early at the national level may avoid some of the regional level management and control measures.
Evidera Payer Investigation in 2017: 60 Payers across US and GER, FR, ENG, IT and SP. Mapping reflects the average of all payer responses per mark