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EMA – Joint HTAb-regulatory perspectives on understanding evidence challenges, managing uncertainties and exploring potential solutions

The EMA recently published the Joint HTAb-regulatory perspectives on understanding evidence challenges, managing uncertainties and exploring potential solutions Outcome of a workshop series between HTA bodies and regulators.

Improving mutual understanding between Health Technology Assessment bodies (HTAbs) and regulators of the assessment challenges each decision maker faces as a result of current approaches to evidence generation, including the management of remaining uncertainties at the time of decision making, was explored in a series of workshops.

Typical methodological challenges faced by regulators and HTAbs were illustrated using three case studies:

  • Onasemnogene abeparvovec (Zolgensma), indicated for the treatment of patients with 5q spinal muscular atrophy (either clinically or genetically defined).
  • Amivantamab (Rybrevant), indicated as monotherapy for the treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) with activating epidermal growth factor receptor (EGFR) Exon 20 insertion mutations after failure of platinum-based therapy.
  • Axicabtagene ciloleucel (Yescarta), indicated for the treatment of adult patients with diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBCL) that relapses within 12 months from completion of, or is refractory to, first line chemoimmunotherapy.

Participants identified the main challenges during assessment and at fulfilling their respective roles focussing on the evidence available at initial market entry of the three products, discussed learnings and explored potential solutions in terms of alternative evidence generation strategies.

The agreed summarised conclusions and key considerations provide an important framework for future HTAb and regulatory collaboration on both improved evidence generation strategies as well as methodologies for mitigating the identified uncertainties. Such summary should help developers to design development plans that provide relevant evidence for each type of decision making.


OpenEdition vous propose de citer ce billet de la manière suivante :
vroby (19 mai 2025). EMA – Joint HTAb-regulatory perspectives on understanding evidence challenges, managing uncertainties and exploring potential solutions. ELSIBI. Consulté le 16 mars 2026 à l’adresse https://doi.org/10.58079/13y61


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