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Support of Real-World Evidence in the Price and Reimbursement Decision-Making​ Process for Rare Diseases in Spain

Real-World Evidence (RWE) is a promising source of complementary evidence to accelerate drug access in rare diseases. However, RWE applicability in price and reimbursement (P&R) is still limited due to a number of challenges.​ This article explores the potential and challenges related to RWE in P&R decision-making for rare disease products in Spain, one of the most attractive markets in Europe because of universal healthcare.

Published on:
November 7, 2023
RWE Can Support P&R Negotiations to Accelerate Rare Disease Drug Access

While Spain is a highly attractive market for pharmaceutical and biotech companies, we have observed that 50% of new orphan drugs approved by the EMA are not approved by the payers in Spain; lack of evidence is one of the possible reasons. RWE can potentially help companies to provide Spanish payers with the evidence they want to see.

Currently, RWE has a minor impact on the P&R decision-making for rare diseases in Spain, limited to a supporting tool for outcome-based agreements. This limitation is caused by several issues, including a lack of regulatory framework, insufficient RWE data quality in Spain, and late involvement of the payers in the clinical trial design.

However, RWE can play a key role in establishing innovative, dynamic, and value-based pricing strategies (currently under evaluation in Spain) and in re-evaluation processes and price reviews:

  • VALTERMED and early access programs have the potential to generate RWD that can be leveraged for evidence generation.
  • RWE can complement the evidence needed in P&R decisions and act as a supporting tool for outcome-based agreements and for the establishment of dynamic pricing strategies.
  • Newly generated evidence can translate into changes in drug pricing and positioning.
  • RWE is potentially key to drug re-evaluation if the evidence supports the value of the drug and to ensure real-life drug effectiveness; although currently, re-evaluation processes often result in a price decrease.

The new artificial intelligence (AI) technologies are the key tool for conducting these RWE studies. AI allows companies to analyze the RWD from many patients directly from the primary source (electronic medical records) with a minimal administrative burden. AI also allows complex analyses that facilitate the selection and positioning of new drugs in therapeutics by identifying patient profiles in which there is a greater clinical benefit.

​A key point to leverage RWE in P&R decisions is that pharmas and biotechs should engage in an early dialogue with payers to co-create a suitable study design that addresses payers’ uncertainties and to include RWE studies within the evidence generation plan.​


Learn More About Real-World Evidence for Pricing and Reimbursement Decision-Making

If you would like to learn more about RWE for pricing and decision-making in rare diseases, make sure you download the poster “Support of RWE in the Price and Reimbursement Decision-Making Process for Rare Diseases in Spain” presented at ISPOR Europe 2023.



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