Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Experiences with PIPs and their required revisions on the critical path of the development of medicines in indications for adult patients ***

Dr. Heidrun Albrecht (Abschlußjahr: 2013)

Summary
Language: English
The present master thesis focuses on the characterisation of PIP modifications. 238 EMA decisions on PIP modifications, as available on the EMA webpage on the 30th May 2013, build the data base. The characterisation of the evaluated EMA decisions on PIP modifications reveals that 43 % of the EMA decisions on PIP modifications regard medicinal products without an authorised indication. The most prominent therapeutic areas reflect the prominence of medicinal products developed for these therapeutic areas in the adult population. The most frequent paediatric subset in the evaluated decisions of the present thesis is children aged two to eleven years. Most of the investigated decisions on PIP modifications (82.8 %) contain at least one waiver. A high proportion (84 %) of the evaluated EMA decisions on PIP modifications include a deferral for one or more studies contained in the PIP. Most of the investigated decisions on PIP modifications will be completed in the years 2014 and 2016. A high proportion (68.5 %) of the evaluated decisions on PIP modifications require a follow-up of potential safety and efficacy issues on paediatric measures or reference an EU risk management plan. More than half (52.1 %) of the investigated EMA decisions on PIP modifications already have a history of one to six previous decisions. In rare cases the PIP modification procedure from start of procedure to EMA decision may last only a few days. Most frequently the evaluated PIP modification procedure lasts 104 to 106 days. The longest investigated period is 126 and 127 days due to a re-examination procedure. The scopes of the PIP modifications of the investigated EMA decisions are diverse. Most frequently the measures and/or timelines are changed during a PIP modification procedure. The comparison between the most current PIP modification and the corresponding PIP agreement (due to the available data on the EMA web page possible for 20 % of the investigated EMA decisions on PIP modifications) reveals that the completion of the PIP as stated in the PIP agreement might be delayed for three to three and a half years. The present master thesis comes to the conclusion that every modification of an agreed PIP bears the risk that initial timeframes of the adult marketing authorisation might not be kept as compliance to the agreed PIP has to be verified prior to the validation of the marketing authorisation application for the adult population. Therefore the prevention of PIP modifications in the first place is advisable.
Pages: 57, Annexes: pages: 24