n increase in advanced therapy medicinal products (ATMPs) being developed and approved is forcing health technology assessment (HTA) bodies and payers to come up with new value appraisal and payment models for these ground-breaking innovations. Cell- and gene-based therapies and other potentially curative treatments—particularly those developed under accelerated approval pathways—typically lack the long-term evidentiary and outcomes data required by HTA bodies to determine reimbursement levels. As a result, stakeholders must now embrace new ways of gathering, standardizing, and sharing real world evidence to better inform their decision-making process.

Key Takeaways

• Real world data can complement data from randomized controlled trials to generate more evidence and characterization of a new product when first introduced, but new approaches are needed to enable real world evidence to be captured over the course of several years to ensure cost-effectiveness and patient access.
• To better compare and track outcomes, stakeholders must work toward standardizing outcome measurements for different diseases to enable level comparisons across patient populations and geographies.
• Discussions on stakeholder alignment on evidence for AMPTs must include the whole healthcare ecosystem to ensure these treatments fit into healthcare systems and reach patients.

Why this is important: Evidence packages to support regulatory approval for novel therapies are not always designed to address the questions asked during the value appraisal process. “This is why multi-stakeholder engagement and dialogue are so important across the entire value chain,” said Claudine Sapède, Global HTA and Payment Policy Lead at Roche. “Because a lot of products are coming and there isn’t a one-time solution, we cannot repeat a big exercise; we need to have a long-term view but also be pragmatic.”

Defining the Value of Innovation

Uncertainty on the magnitude and duration of effect of novel AMPTs limits their pricing potential and adds to developer and payer concerns about financial sustainability. Additionally, the fragmented HTA process across national and regional level delays patient access to otherwise curative therapies. But according to the Alliance for Regenerative Medicine (ARM)—a global advocacy organization for regenerative and advanced therapies—cell therapy could actually be an ideal setting for new reimbursement models. With more precise patient outcomes data, patient-by-patient reimbursement would be more practical and often more cost-effective than with traditional drugs.

Defining the standards to assess the affordability of a curative product at launch is an area where HTA bodies and payers can learn from one another’s experience as they all face similar challenges. As more therapies are being developed with the potential to cure conditions in a single course of treatment, their initial high cost must be balanced with the potential for gains to accumulate long after treatment. In France, for example, HTA makes a strong distinction between a curative treatment that targets the roots of the disease versus one that merely improves disease-associated symptoms. Appropriately, the French Transparency Committee (Haute Autorité de Santé or HANS) has no HTA definition of “cure,” and focuses instead on additional medical benefit.

Methods for Combination Products

With so many different approaches being applied, sharing the long-term consequences of these mechanisms for healthcare systems, patients, and manufacturers will be particularly helpful for decision makers in other countries where the current system fails to address combination product pricing (e.g., Germany, UK).

Collaboration and Transparency

With multi-stakeholder engagement and collaboration, the entire constellation of biopharmaceutical stakeholders—HTA bodies, payers, developers, healthcare systems and patients—can learn from one another to standardize real world evidence for successful value appraisal of innovative therapies. It will require a long-term view and pragmatic solutions, but all parties benefit by improving patient access to medicines and reducing reimbursement uncertainty.