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RWE Decision Alert

French HTA Maintains Reimbursement of HYQVIA® in Adults and Extends Reimbursement to Children, Largely Driven by RWE


On November 22, 2023, HYQVIA® was re-evaluated by the French Transparency Committee (TC) of the National Health Authority/Haute Autorité de santé (HAS) for the treatment of primary and secondary immune deficiencies. Following the review of real-world evidence (RWE) generated in observational studies, Phase IV post-authorization safety studies (PASS), and single arm clinical trials, the TC determined that HYQVIA continues to provide significant benefit. Consequently, the TC maintained reimbursement for HYQVIA in the adult population and extended reimbursement to include children under 18 years of age.

HYQVIA is human normal immunoglobulin co-administered with recombinant human hyaluronidase. Manufactured by Takeda Pharmaceuticals, the drug was initially approved in 2015 by HAS in replacement therapy for adults with primary or secondary immunodeficiency. In 2017, the TC concluded that in children (<18 years) the actual benefit of HYQVIA was insufficient to justify reimbursement by the national health system, due to uncertainty in the treatment effect. The uncertainty regarding HYQVIA’s efficacy in reducing the rate of acute serious bacterial infections (ASBIs), which was the primary endpoint, stemmed from multiple factors. These included the reliance on a non-prespecified subgroup analysis from a single-arm Phase III trial, a limited sample size, unresolved questions about the drug’s impact on fertility, and the low number of observed events.

2023 HAS Evaluation

In the latest reassessment of HYQVIA, published on November 22, 2023, the TC of HAS thoroughly reviewed HYQVIA’s comprehensive body of data in both adults and children (<18 years). Overall, the TC valued that Takeda Pharmaceuticals submitted data from PASS, and a European observational study (FIGARO) which supported the demonstration of HYQVIA’s safety profile. However, the TC again criticized the non-comparative clinical trial study design and categorized the observational studies as “largely exploratory.” The TC valued the establishment of a pregnancy registry to assess the tolerance of HYQVIA and the impact of anti-rHuPH20 antibodies during pregnancy, however, it criticized the small sample size. Given the absence of comparative data with alternative therapies, the choice of immunoglobulin therapies should rely on patient characteristics and patient preferences.

In adults, despite the lack of new efficacy data, additional follow-up data from the single arm-clinical trial (Study 161406), Phase IV PASS, the observational study FIGARO, and a pregnancy registry to measure the impact of HYQVIA treatment during pregnancy in infants all contributed to demonstrating that HYQVIA has a “generally favorable safety profile.”1 The TC considered the actual benefit of HYQVIA to remain significant, issuing a favorable opinion on maintaining reimbursement of HYQVIA for use in the indications of the marketing authorization. However, the TC concluded that HYQVIA does not provide any improvement in actual benefit (noted as comparative level of effectiveness ASMR V) compared to other normal human immunoglobulins administered intravenously or subcutaneously. 

In children, the TC concluded that the new efficacy data from the single-arm clinical trial (American Study 161503) and safety data from PASS demonstrates that HYQVIA has a favorable safety profile. Therefore, the TC considered the actual benefit of HYQVIA as significant and issued a favorable opinion on reimbursing HYQVIA for use in children in the indications of the marketing authorization. However, the TC concluded that HYQVIA does not provide any improvement in actual benefit (ASMR V) compared to available therapeutic alternatives.

In a new development for the drug, on January 16, 2024, the Food and Drug Administration approved HYQVIA for the treatment of chronic inflammatory demyelinating polyneuropathy, a rare neurological disorder leading to progressive weakness and reduced sensibility in the limbs. RWE was used as supportive safety data and as a historical benchmark to provide baseline data as the primary endpoint.


Although the TC categorized the post-marketing studies as “exploratory,”1 and criticized the non-comparative clinical trial design, the evidence presented was deemed sufficient to maintain the reimbursement status of HYQVIA in the adult population and to grant reimbursement in children. The RWE derived from the PASS and FIGARO studies played a crucial role, supplying safety data that informed the decision-making process in both target populations.

Author: Catia Proenca, Director in Real-World Solutions

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RWE Decision Alerts provide subscribers with timely analyses of published reports from HAS, AEMPS, NICE, EMA, and the FDA where RWE is used to support HTA and regulatory decision-making. We identify if a submission included RWE and assess the value of the RWE submitted.

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