Qualification of novel methodologies - “Proof-of-Principle” study

Challenge

The European Medicines Agency (EMA) has an existing pathway that provides EMA opinions on the validity of new methods relating to the generation or synthesis of evidence submitted for regulatory assessment, called “Qualification of Novel Methodologies.” 

A joint parallel procedure, “Regulatory -HTA/Payer Qualification of Novel Methodologies”, could be conducted in the context of HARMONY. There is currently no equivalent of this process for payers and HTAs in different countries.

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Project Partnership

• UoY, AEMPS, BfArM, UULM, CELGENE, JANSSEN, LeukaNET

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Project Leadership

• NICE

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Impact

• Conduct “Proof-of-Principle” study to explore HTA Evidence Framework and Clinical Value Framework
• Developing “Proof-of-Principle” study stakeholders guidelines for payers and HTAs in different countries

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Project Summary

HARMONY will explore how “qualification of new methodologies” can be run operationally and whether it can deliver meaningful outcomes for three types of decision-makers: payers, regulators and HTA agencies. It will also be considered whether this approach is viable in the future, evaluating resources and costs of participation. 

A pilot, joint qualification procedure could include deliverables from:

• HARMONY Work Package 2 - Definition of Outcomes: Outcome definitions and coordination of proof of principle HM studies; 
• HARMONY Work Package 5 - Data Analytics for Evaluation of Therapies: Identification of specific biomarkers, which better define outcome parameters;
• HARMONY Work Package 6 - Work Package 6: Payers/Providers/HTAs, EMA Alignment and Optimization: – tools to quantify the added value of new technologies. 

The final selection of a deliverable to undergo this proof of principle approach will need to be agreed upon and further defined by the consortium. 
 

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