Nancy Risebrough

Nancy Risebrough

Senior Principal, Global HTA, Health Economics, Reimbursement and Outcomes

Mia Malmenas

Mia Malmenas

Senior Principal, Global HTA, Health Economics, Reimbursement and Outcomes

Early access schemes demand new ways of performing health economic evaluations.

This means developing health economic models early to evaluate gaps and determine what level of investment makes sense. And, it means designing more rigorous Phase I and Phase II trials, as well as follow-up studies using real-world evidence (RWE). Starting early in generating the right evidence will ensure that sponsors face fewer surprises as they seek reimbursement.

Companies can follow a well-paved path to success in developing the evidence required by Health Technology Assessment (HTA) bodies and payers for products pursuing early approval from regulators. First and foremost, sponsors should begin thinking about their evidence plan as soon as they know that their drug has the potential to be a candidate for an early access programme. At that point, they should engage their health economics and outcomes research (HEOR) partner and together determine the basics:

  • What data is needed
  • What data is available and how it will be accessed
  • What investment will be required to gain the needed evidence

When developing an early-stage economic model, the following steps should be taken:

  1. Engage internal stakeholders to ensure that they buy into the objectives of the model
  2. Define the model’s purpose and limitations
  3. Build a conceptual model based on the available data
  4. Review and revise the model, incorporating feedback from stakeholders along the way
  5. Identify the required model outputs
  6. Program and test the model
  7. Test assumptions and uncertainty
  8. Validate the model results

In terms of the economic model itself, best practices include:

  • Focusing on a few select markets that are representative of different systems and perspectives on cost effectiveness and economic valuation.
  • Designing the model to accommodate several comparators or positions in the treatment pathway, since in the early stages, it is often unclear how the product will fit into the treatment pathway.
  • Designing models such that they can evolve into late-stage models, which means starting with a robust framework to avoid duplicating efforts at a later stage.
  • Performing real-world evidence studies, such as database analyses, to provide additional support for the assumptions made in the economic model, thus reducing uncertainty and making the outputs more robust.
  • Building in a time horizon that is long enough to capture all of the consequences of the new product.

For more information on this topic, please contact us.

 

This blog is an edited version of “Using economic models to gain early access” which appeared in the October 2018 edition of Pharmafocus. To view the full article, please visit http://www.pharmafile.com