Health economics and market access

Understanding how price, revenue and profit margins can be maximised is key to any commercial success.

The differences between cell and gene therapies and other types of therapies mean developing robust pricing and reimbursement strategies demands additional considerations to those of conventional pharmaceuticals. These considerations also need to be addressed earlier in development given the higher commercial risk cell and gene therapies present.

The health economics and market access team provides health economics, pricing, reimbursement and market access expertise to accelerate and maximise cell therapy uptake across global healthcare markets.

Together, with our dedicated team, we can help develop your health-economic arguments and models, your value story and pricing and reimbursement strategy to accelerate and maximise your therapy’s uptake and revenue potential.

We can help you optimise the use of your resources – selecting the right target population and therapeutic position before you start clinical development so that there is enough room for innovation to accommodate a high cost therapy.

We’ll work with you on your product design, and target product profile, to help your therapy become a commercial success.

We can help with planning your evidence generation so that it meets the requirements of key market access stakeholders.

We can help with, defining product performance and manufacturing cost thresholds for commercial viability so that your clinical and manufacturing strategies are informed accordingly.

We’ll help you work out a clear pathway for reimbursement and adoption of your therapy across major global healthcare markets.

For typical assessments undertaken by our market access team, please view the following two documents: The Cell and Gene therapy Catapult approach to pricing and reimbursement strategy development and insights into the Big5EU pricing and reimbursement potential of a novel cell and gene therapy currently in early clinical development.

We provide health economic and market access support along all stages of a cell and gene therapy’s route to market. See below for the key codependent activity domains.

1. Shaping early development

Process

 Areas of focus

Benefits
  • Systematic evidence review and meta-analysis
  • KOL and payer engagement
  • Retrospective chart reviews
  • Headroom method of early economic evaluation
  • Epidemiology
  • Current and emerging Tx algorithm
  • Clinical and economic outcomes with current treatments
  • Pricing, reimbursement and funding of current treatments
  • Disease burden
  • Unmet need and headroom for innovation by therapeutic positioning/subpopulation
  • Most relevant comparators, clinical and economic outcome measures for the planning of the evidence generation
  • Relevant healthcare reforms & implications
  • Quantify unmet need and headroom for innovation
  • Inform on the interrelationship between therapeutic positioning, risk, reward and opportunity
  • Identify clinical and health economic value drivers for the target product profile (TPP)
  • Quantify minimal important difference in incremental benefit versus standard of care/best supportive care
  • Inform on clinical development specifications (e.g. inclusion criteria, comparators, outcomes) that help optimise market access opportunity

 

2. Formulate pricing and reimbursement strategy

Process  Areas of focus Benefits
  • Define target product profile (TPP) and account for:
    • clinical feasibility
    • clinical and health economic value drivers
    • minimal importance difference in incremental benefit versus standard of care/best supportive care
  • Conduct early health economic analysis (budget impact, cost-consequence, cost-effectiveness, cost-utility)
  • Sensitivity analysis to address uncertainty
  • Conduct analogue analysis
  • Use TPP, health economic and analogue analyses in exploratory discussions with key market access stakeholders
  • Identification of clinical and health economic arguments that best resonate with key market access stakeholders
  • Likely positioning, pricing, reimbursement, uptake
  • Interrelationship between supporting data and market access
  • Validate key value drivers for TPP and areas on which to concentrate data generation activities
  • Clarify market access requirements for RCT and modelled data (e.g. indirect comparisons, extrapolations)
  • Validate health economic model structure, assumptions and inputs
  • Formulate pricing and reimbursement strategy
  • Generate estimates of price and volume for forecasting and valuation
  • Inform go/no-go decisions

 

3. Value story and data optimisation

Process

Areas of focus Benefits
  • Address RCT data limitations, e.g.
    • Regression methods to account for trial imbalance and missing data
    • Adjust survival estimates in the presence of treatment switching
  • Address the evidence gap between RCT data and value proposition, advance health economic models
    • Elicit utilities for relevant health states
    • Delphi panels
    • Meta-analysis
    • Indirect treatment comparisons/mixed treatment comparisons
    • Extrapolations to substantiate long-term claims
    • Modelling of the cost-effectiveness and budgetary impact for subpopulations
  • Develop value story
  • Conduct qualitative primary research with key market access stakeholders to test and refine value story and supporting evidence
  • Develop value story, assess and improve its credibility and impact
  • Optimise supporting evidence
  • Optimise value story and supporting data in preparation for market access negotiations

 

4.    Global optimisation

Process

Areas of focus

 Benefits

  • Quantitative primary research with key market access stakeholders  across major markets
    • Conjoint analysis/discrete choice modelling
  • Country-specific adaptation of health economic models
  • Finalise target price and launch sequence
    • Account for international price referencing
  • Contingency planning:
    • Risk-sharing schemes
    • Post-launch evidence generation
  • Optimise interrelationship between price and volume of uptake
  • Identify price corridor in key markets of interest
  • Optimise launch sequence
  • Define revenue maximising price corridor and launch sequence

 

5.    Value dossier development

Process

 Areas of focus

Benefits
  • Medical writing activities covering the following three domains: clinical, health economics and outcomes research
  • Clinical, health economic and humanistic value proposition and corresponding supporting evidence compiled in a single document
  • A flexible document that can be used to support pricing, reimbursement, funding and formulary inclusion negotiations with key market access stakeholders (national/regional/local level)
  • Accounting for variation in market access assessment criteria and data requirements across geographies

 

Our health economists and market access professionals are the only team in Europe that focus exclusively on cell and gene therapies, which has helped us to understand and develop strategies that address the unique barriers to reimbursement you would face.

We’re also actively working with payers across European markets to help them develop and shape how they reimburse you. In response to the lack of sufficient information on market access processes for cell and gene therapies in the major European markets, we compiled a set of reimbursement roadmaps in-depth presentations covering the Big5 EU: France, Germany, Italy, Spain and the UK.

For additional examples of our health economics and market access work, please see:

Undertaking a health economic assessment to understand the reimbursed price potential of MULTISTEM ® in acute respiratory distress syndrome

How uncertainty around the value claims of cell and gene–based immunotherapies can be addressed in payer negotiations

For our peer reviewed-publications by our health economics and market access team, please see:

Annuity payments can increase patient access to innovative cell and gene therapies under England’s net budget impact test  European healthcare markets. J Mark Access Health Policy 2017 5:1, 1355203, DOI: 10.1080/20016689.2017.1355203

The High Cost of High Tech Medicine: Planning Ahead for Market Access. Stem Cell Translational Medicine 2017 6: 1723

A price comparison of recently launched proprietary pharmaceuticals in the UK and the US. J Mark Access Health Policy 2016 4: 32754

Reimbursement of licensed cell and gene therapies across the major European healthcare markets. J Mark Access Health Policy 2015 3: 29321

Market access pathways for cell therapies in France. J Mark Access Health Policy 2015 3: 29094

Bringing regenerative medicines to the clinic: the future for regulation and reimbursement. Regen Med 2015 13;10(7): 897-911

Cell-based therapy technology classifications and translational challenges. Philos Trans R Soc Lond B Biol Sci 2015 Oct; 370 (1680): 20150017

Innovator cell therapies present barriers to the entry of copy versions regardless of patent protection

Contact us to find out more about our health economics and market access capabilities.

Discover who we are