Giovanni Tafuri1, Inês Lucas2, Steve Estevão3, Jane Moseley2, Anne d'Andon4, Hannah Bruehl5, Elangovan Gajraj6, Sonia Garcia7, Niklas Hedberg8, Marco Massari1, Andrea Molina9, Mercè Obach9, Leeza Osipenko6, Frank Petavy2, Marco Petschulies5, Caridad Pontes9, Pierluigi Russo1, Anja Schiel10, Marc Van de Casteele11, Eva-Maria Zebedin-Brandl12, Guido Rasi2, Spiros Vamvakas2. 1. Italian Medicines Agency (AIFA), Via del Tritone, 181-00187, Rome, Italy. 2. European Medicines Agency (EMA), 30 Churchill Place, London, E14 5EU, United Kingdom. 3. The National Health Care Institute (ZIN), 4 Eekholt, Diemen, 1112 XH, The Netherlands. 4. Haute Autorité de Santé (HAS), 5 Avenue du Stade de France, 93210, Saint-Denis, France. 5. The Federal Joint Committee (G-BA), Wegelystr. 8, D-10623, Berlin, Germany. 6. The National Institute for Health and Care Excellence (NICE), 10 Spring Gardens, London, SW1A 2BU, United Kingdom. 7. Spanish Agency of Medicines and Medical Devices (AEMPS), Calle Campezo 1, Edificio 8, 28022, Madrid, Spain. 8. Dental and Pharmaceutical Benefits Agency (TLV), Fleminggatan 18, 104 22, Stockholm, Sweden. 9. Servei Català de la Salut (CatSalut), Travessera de les Corts, 131-159, Edifici Olímpia, 08028, Barcelona, Spain. 10. Norwegian Medicines Agency (NoMA), Strømsveien 96, 0663, Oslo, Norway. 11. National Institute for Health and Disability Insurance (INAMI-RIZIV), Avenue de Tervueren 211, 1150, Bruxelles, Belgium. 12. Main Association of Austrian Social Security Institutions (HVB), Kundmanngasse 21, A-1031, Vienna, Austria.
Abstract
AIMS: The parallel regulatory-health technology assessment scientific advice (PSA) procedure allows manufacturers to receive simultaneous feedback from both EU regulators and health technology assessment (HTA) bodies on development plans for new medicines. The primary objective of the present study is to investigate whether PSA is integrated in the clinical development programmes for which advice was sought. METHODS: Contents of PSA provided by regulators and HTA bodies for each procedure between 2010 and 2015 were analysed. The development of all clinical studies for which PSA had been sought was tracked using three different databases. The rate of uptake of the advice provided by regulators and HTA bodies was assessed on two key variables: comparator/s and primary endpoint. RESULTS: In terms of uptake of comparator recommendations at the time of PSA in the actual development, our analysis showed that manufacturers implemented comparators to address both the needs of regulators and of at least one HTA body in 12 of 21 studies. For primary endpoints, in all included studies manufacturers addressed both the needs of the regulators and at least one HTA body. CONCLUSIONS: One of the key findings of this analysis is that manufacturers tend to implement changes to the development programme based on both regulatory and HTA advice with regards to the choice of primary endpoint and comparator. It also confirms the challenging choice of the study comparator, for which manufacturers seem to be more inclined to satisfy the regulatory advice. Continuous research efforts in this area are of paramount importance from a public health perspective.
AIMS: The parallel regulatory-health technology assessment scientific advice (PSA) procedure allows manufacturers to receive simultaneous feedback from both EU regulators and health technology assessment (HTA) bodies on development plans for new medicines. The primary objective of the present study is to investigate whether PSA is integrated in the clinical development programmes for which advice was sought. METHODS: Contents of PSA provided by regulators and HTA bodies for each procedure between 2010 and 2015 were analysed. The development of all clinical studies for which PSA had been sought was tracked using three different databases. The rate of uptake of the advice provided by regulators and HTA bodies was assessed on two key variables: comparator/s and primary endpoint. RESULTS: In terms of uptake of comparator recommendations at the time of PSA in the actual development, our analysis showed that manufacturers implemented comparators to address both the needs of regulators and of at least one HTA body in 12 of 21 studies. For primary endpoints, in all included studies manufacturers addressed both the needs of the regulators and at least one HTA body. CONCLUSIONS: One of the key findings of this analysis is that manufacturers tend to implement changes to the development programme based on both regulatory and HTA advice with regards to the choice of primary endpoint and comparator. It also confirms the challenging choice of the study comparator, for which manufacturers seem to be more inclined to satisfy the regulatory advice. Continuous research efforts in this area are of paramount importance from a public health perspective.
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