US FDA's patient-focused drug development meeting on sickle cell disease inspired Global Blood Therapeutics to develop a new patient-reported outcomes instrument, while an externally led meeting on amyloidosis confirmed for Alnylam that its pivotal trial was studying clinically meaningful secondary endpoints.
The philosophy underpinning the patient-focused drug development (PFDD) movement – that it's important to find out what symptoms are most bothersome to patients, and what they would like to see in new treatments – is increasingly being embraced by drug developers as they plan their clinical programs for new compounds.
Below are two examples of how the growing attention to the importance of the patient voice, coupled with the US FDA's own PFDD initiatives,
Marcus Vass and Vladimir Murovec of international law firm Osborne Clarke tell the Pink Sheet what the government's planned HDRS might mean for industry, and how it compares with the European Health Data Space.
The European Health Data Space framework will allow companies to accelerate R&D processes and identify new molecular targets faster by facilitating centralized access to certain types of high-quality data, Finland’s Orion Pharma says.
EU-level joint clinical assessments conducted under the Health Technology Assessment Regulation need to be more flexible when it comes to evidence requirements, according to experts speaking at a gene and cell therapy conference.
Both the EU Clinical Trials Regulation and the European Health Data Space Regulation have the potential to improve harmonization and be highly valuable for industry – but the importance of protecting company data will be paramount, a life sciences consultant says.
The European Medicines Agency, like its counterpart in the US, is increasingly focusing on the use of alternatives to animal testing.
European pharma trade associations EFPIA and EUCOPE outline their respective views on how the EU’s pharma legislation overhaul should tackle antimicrobial resistance, and why transferable exclusivity vouchers alone will not suffice as incentives.
EU-level joint clinical assessments conducted under the Health Technology Assessment Regulation need to be more flexible when it comes to evidence requirements, according to experts speaking at a gene and cell therapy conference.
