The impact of parallel regulatory–health technology assessment scientific advice on clinical development. Assessing the uptake of regulatory and health technology assessment recommendations

• Published in: British journal of clinical pharmacology Vol. 84; no. 5; pp. 1013 - 1019
• Main Authors: Tafuri, Giovanni, Lucas, Inês, Estevão, Steve, Moseley, Jane, d'Andon, Anne, Bruehl, Hannah, Gajraj, Elangovan, Garcia, Sonia, Hedberg, Niklas, Massari, Marco, Molina, Andrea, Obach, Mercè, Osipenko, Leeza, Petavy, Frank, Petschulies, Marco, Pontes, Caridad, Russo, Pierluigi, Schiel, Anja, Van de Casteele, Marc, Zebedin‐Brandl, Eva‐Maria, Rasi, Guido, Vamvakas, Spiros
• Format: Journal Article
• Language: English
• Published: England John Wiley and Sons Inc 01-05-2018
• Subjects: clinical development; Drug Development - statistics & numerical data; Drug Industry - statistics & numerical data; European Medicines Agency; Government Regulation; health technology assessment; HTA bodies; Humans; parallel scientific advice; regulatory; Technology Assessment, Biomedical - statistics & numerical data; Therapeutics; health technology assessment; parallel scientific advice; clinical development; European Medicines Agency; HTA bodies; regulatory
• ISSN: 0306-5251; 1365-2125
• DOI: 10.1111/bcp.13524

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.