Discover why none of the listed therapies (Tecartus, Rozlytrek, Zolgensma, etc.) achieved full agreement between regulatory approval and HTA reimbursement decisions.
Question
Select the therapies where Regulators and Health technology assessment bodies agreed on the decision
A. Tecartus
B. Rozlytrek
C. Polivy
D. Zolgensma
E. Yescarta
F. Kymriah
G. Luxturna
H. Evrysdi
I. None
Answer
I. None
Explanation
There is no full agreement between Regulators and Health technology assessment bodies on any of the therapies listed.
Regulatory agencies (e.g., Health Canada, EMA) and Health Technology Assessment (HTA) bodies often evaluate therapies through distinct lenses: regulators focus on safety and efficacy, while HTAs assess cost-effectiveness, clinical value, and budget impact. None of the listed therapies achieved full alignment between these two decision-making frameworks, as highlighted below:
Key Analysis of Each Therapy
Tecartus (A)
- Regulatory Approval: Authorized by Health Canada and EMA for relapsed/refractory mantle cell lymphoma (MCL) based on high response rates (91% ORR) and durable remissions.
- HTA Perspective: No direct HTA agreement cited. Long-term evidence gaps (e.g., 24-month follow-up commitments) suggest conditional approval.
- Outcome: No alignment.
Rozlytrek (B)
- Regulatory Approval: Received conditional authorization in Canada and EMA for NTRK-fusion cancers under Notice of Compliance with Conditions (NOC/c).
- HTA Perspective: Required post-market studies to confirm clinical benefit, indicating unresolved uncertainties.
- Outcome: No alignment.
Polivy (C)
- Regulatory Approval: Initially rejected for Priority Review in Canada due to insufficient evidence. Later authorized for DLBCL under NOC/c.
- HTA Perspective: Limited clinical data led to exclusion from expedited reimbursement pathways.
- Outcome: No alignment.
Zolgensma (D)
- Regulatory Approval: Approved for spinal muscular atrophy (SMA) but criticized for high costs (e.g., R$944.8 million in Brazil).
- HTA Perspective: Judicialization and risk-sharing agreements (e.g., UK’s NHS) highlight misalignment on cost-effectiveness.
- Outcome: No alignment.
Yescarta (E)
- Regulatory Approval: Approved for relapsed/refractory large B-cell lymphoma.
- HTA Perspective: UK’s NICE required discounts and enrollment in the Cancer Drugs Fund due to uncertain long-term benefits.
- Outcome: No alignment.
Kymriah (F)
- Regulatory Approval: Authorized for ALL and DLBCL.
- HTA Perspective: UK’s SMC rejected DLBCL indication over cost-effectiveness concerns despite regulatory approval.
- Outcome: No alignment.
Luxturna (G)
- Regulatory Approval: EMA-approved for inherited retinal dystrophy.
- HTA Perspective: Reimbursement left to individual EU states, with no unified HTA endorsement.
- Outcome: No alignment.
Evrysdi (H)
- Regulatory Approval: Granted Priority Review in Canada for SMA.
- HTA Perspective: Limited data for patients >25 years and no HTA consensus cited.
- Outcome: No alignment.
Why I. None is Correct
- Regulatory-HTA Divergence: Regulatory approvals often rely on surrogate endpoints (e.g., response rates), while HTAs demand long-term survival data and cost-effectiveness.
- Case-Specific Conflicts: Examples include Zolgensma’s judicialization (Brazil)4, Kymriah’s rejection by SMC14, and Yescarta’s conditional NHS funding.
- Structural Misalignment: Sequential evaluation (regulatory first, HTA second) inherently delays consensus, as seen in Canada and the EU.
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