Adaptive licensing could ‘transform industry’
pharmafile | December 17, 2014 | News story | Research and Development, Sales and Marketing | Clinical Pharmacology & Therapeutics, EFPIA, EMA, MHRA, adaptive licesning
Drug regulators should switch to adaptive pathways as the preferred approach to bringing future new medicines to patients, according to new research.
A paper published online in the journal Clinical Pharmacology & Therapeutics, says adaptive pathways to drug licensing have the potential to be ‘transformative’ for the industry and “highly beneficial for both patients and the healthcare ecosystem” – but only if there is pressure on the pharma industry, investors and politicians to ensure sustainability of drug development, the group concludes.
The work was led by Dr Hans-Georg Eichler, senior medical officer at the EMA, jointly with senior figures from across the industry including pharma firms, the MHRA and patient groups.
Adaptive pathways (formerly known as adaptive licensing), is the early approval of a medicine for a restricted patient population based on small initial clinical studies. The first approval is followed by progressive adaptations of the marketing authorisation to expand access to the medicine to broader patient populations based on data gathered from its use and additional studies.
But adaptive pathways create a ‘conundrum’ the paper says, as the pharma industry needs to balance the “delicate trade-offs between encouraging rapid patient access to promising therapies on the one hand and ensuring patients, and their regulatory and physician proxies, possess adequate information on benefits and harms at the time of marketing authorisation on the other”.
The EMA launched a pilot project on adaptive pathways in March 2014 to explore this approach with real medicines in development. As of November 2014, the it had received and assessed 29 applications as part of the pilot, nine of which had been selected. The first stage of the pilot ends in February next year, with a report on the pilot due before the end of 2014.
Although a growing number of pharma executives and bodies including the European Federation of Pharmaceutical Industries and Associations (EFPIA) have thrown their weight behind adaptive pathways, there are still a number of “environmental changes that will likely make adaptive pathways the preferred approach in future”, the report says – not least “political will… [and] substantial political debate among the various stakeholders”.
The group concludes: “While the conceptual change of the adaptive pathways concept is transformative, implementation is expected to be evolutionary rather than disruptive, and will likely progress differently across jurisdictions.”
Lilian Anekwe
Drivers and enablers of adaptive pathways
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Drivers |
Enablers |
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Patient expectations: demand for timely access and emphasis on unmet medical need |
Improved understanding of disease processes, better knowledge management
|
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Emerging science: fragmentation of treatment populations and early disease interception |
Innovative clinical trial designs
|
|
Healthcare systems under pressure: rise of payer influence |
Rapid learning systems in the healthcare environment
|
|
Pharma/investors under pressure: sustainability of drug development |
Bringing patients to the table: understanding acceptable uncertainty
|
|
|
From prediction to monitoring
|
|
|
Targeted prescribing |
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