Today’s post is by Marieke Dekker (Scientific Researcher at the Medicines Evaluation Board, CBG-MEB)
Currently, 4 different Alzheimer’s disease (AD) drugs are available in Europe. The acetylcholinesterase inhibitors rivastigmine, donepezil and galantamine are approved for persons with mild-to-moderate AD and memantine, a non-competitive N-methyl-D-aspartate receptor antagonist (NMDA), for patients with moderate-to-severe AD. All these medicines were introduced in the late nineties or at the beginning of our century and have only modest symptomatic effects. Although since then no new AD drugs have been successful, currently several new treatments are in late-phase development and will hopefully become available over the next years.
In Work Package 6, regulatory and health technology assessment (HTA) involvement, we review past experiences of regulatory and HTA bodies with AD approved drugs. We evaluate studies and types of evidence used in both procedures and focus on similarities and differences in evidence requirements. We pay special attention to the use of real-world evidence (RWE) in both procedures. So far we evaluated the old marketing authorization application dossiers and the latest NICE technology appraisal of the 4 AD approved drugs. Preliminary findings are that almost all pivotal trials that are included in the application dossier are also used in the NICE assessment and that the use of RWE is limited in both procedures. Our next step is to evaluate the old AD drugs assessment of Zorginstituut Nederland, the Dutch HTA body.
In the second phase of our research project, we will study possible discrepancies between outcomes of the evaluations of regulatory agencies and HTA bodies and real-world effectiveness. Or in other words: is there a gap between the efficacy (as assessed during marketing authorisation by the regulatory bodies) and relative effectiveness (as assessed by the HTA agencies) and the real-world effectiveness. If gaps are present, we aim to identify factors that might play a role.
With our research project we intend to learn from past experiences and contribute to a more harmonised approach of drug licensing and reimbursement in the near future.