To analyse the overseas approval of aducanumab for Alzheimer’s disease, in order to derive lessons of potential interest to individuals and groups involved in dementia drug development and regulation in Australia.
Conclusions
Opportunities were identified regarding optimising clinical trial design and solidifying regulatory responsibilities. Increased awareness of the attitudes and needs of patients and patient advocacy organisations would likely improve the patient-clinician alliance. Incorporation of these factors into research design has the potential to improve patient satisfaction with outcomes.
HuangL-KChaoS-PHuC-J. Clinical trials of new drugs for Alzheimer disease. J Biomed Sci2020; 27: 18.
2.
OxfordAEStewartESRohnTT. Clinical trials in Alzheimer's disease: a hurdle in the path of remedy. Int J Alzheimers Dis2020; 2020: 5380346.
3.
TeunissenCEVerberkIMWThijssenEH, et al.Blood-based biomarkers for Alzheimer's disease: towards clinical implementation. Lancet Neurol2022; 21: 66–77.
4.
GoldmanDMalzbenderKLavin-MenaL, et al.Key Barriers for Clinical Trials for Alzheimer’s Disease. Los Angeles, CA: The USC Schaeffer Center, 2020.
5.
GrillJD. Recruiting to preclinical Alzheimer’s disease clinical trials through registries. Alzheimers Dement2017; 3: 205–212.
6.
GrabherBJ. Effects of Alzheimer disease on patients and their family. J Nucl Med Technol2018; 46: 335–340.
7.
GleasonAAytonSBushAI. Does the FDA-approved Alzheimer drug aducanumab have a place in the Australian pharmacopoeia?Med J Aust2022; 216: 172–174.
8.
LiuKYHowardR. Can we learn lessons from the FDA’s approval of aducanumab?Nat Rev Neurol2021; 17: 715–722.
9.
DyerO. Medicare’s decision not to fund Aduhelm changes landscape for US pharma industry. BMJ2022; 377: o996.
10.
MullinsCDVandigoJZhengZWicksP. Patient-centeredness in the design of clinical trials. Value Health2014; 17: 471–475.
11.
KnopmanDSJonesDTGreiciusMD. Failure to demonstrate efficacy of aducanumab: an analysis of the EMERGE and ENGAGE trials as reported by Biogen, December 2019. Alzheimers Dement2021; 17: 696–701.
12.
Biogen. A Phase 3 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Aducanumab (BIIB037) in Subjects with Early Alzheimer’s Disease. Cambridge, MA: Biogen, 2015.
13.
AisenPSBatemanRJCarrilloM, et al.Platform trials to expedite drug development in Alzheimer’s disease: a report from the EU/US CTAD task force. J Prev Alzheimers Dis2021; 8: 306–312.
14.
SeoYJangHLeeH. Potential applications of artificial intelligence in clinical trials for Alzheimer’s disease. Life (Basel)2022; 12: 275.
15.
VeitchDPWeinerMWAisenPS, et al.Understanding disease progression and improving Alzheimer’s disease clinical trials: recent highlights from the Alzheimer’s disease neuroimaging initiative. Alzheimers Dement2019; 15: 106–152.
16.
BelluckP. Inside a Campaign to Get Medicare Coverage for a New Alzheimer’s Drug. The New York Times, 2022.
17.
BelluckPKaplanSRobbinsR. How an Unproven Alzheimer’s Drug Got Approved. The New York Times, 2021.
18.
BootesAMaunduJGoldingS, et al.Fast-track pathways for drug approvals: the Australian experience so far. Aust Prescr2019; 42: 118–119.
