Looking back: Regulatory Science at the Partnerships MeetUp 2018

In the morning, an interesting Regulatory Science morning session explored ‘Extrapolation in Regulatory Science: Smarter use of available data’. The keynote lecture in the afternoon, by former CHMP chair Dr. Tomas Salmonson, highlighted the opportunities for regulatory science in the Netherlands.

 
Together is the only way forward

In his lecture, Tomas Salmonson presented his vision on the future of regulatory innovation: ‘Together is the only way forward’. While addressing recent developments in the regulatory landscape, he explained that a lot has been achieved by working together in previous years. Dr. Salmonson emphasized the Netherlands’ track record of building competence from different stakeholders, and our capacity for building networks with relevant stakeholders. These are important competences for regulatory innovation and will provide many opportunities for the Dutch regulatory science community in light of the EMA moving to the Netherlands. 

Extrapolation in Regulatory Science: Smarter use of available data

In this session we explored the need for and value of different types of extrapolation methods in drug development, regulation (Kit Roes, Julius Center/UMC Utrecht), HTA (Wim Goettsch, Zorginstituut Nederland), and clinical practice (Saskia de Wildt, RadboudUMC). 

Extrapolation is not a new topic. It has always been part of medical research, practice, and regulation. For example, clinicians translate results from studies into treatment decisions for individual patients, and regulators assess whether outcomes from trials in a selected population should allow a medicine to be placed on the market for a broader target group. However, in recent years we have seen increasing interest in how extrapolation can be achieved in a more explicit, reliable, and transparent manner, and how we can articulate the inherent uncertainties in any extrapolation step. 

This session highlighted the importance of progress in this field; if we are not able to communicate the extent to which research findings can be extrapolated to different populations, then a reduction of complexity in one place can cause uncertainty elsewhere, for example in HTA assessments or in clinical practice.  During the session, Kit Roes gave an overview of current EMA views on the methodological and regulatory status of extrapolation, while Wim Goettsch and Saskia de Wildt discussed the HTA and clinical practice challenges respectively. For example, in HTA, post-licensing data collection is much needed for a full HTA in support of appropriate (extrapolated) use. For example, data on real-world effectiveness is needed when medicines are provided as second line or third line therapy. In clinical practice, initiatives such as the Kinderformularium aim to translate scientific evidence and expert knowledge into best-evidence dosages of medicines for children. 

The session clearly showed the urgent need for building bridges between the regulators, HTA, and clinicians to come to relevant, feasible, and usable ways to communicate the extent to which evidence can be extrapolated between patient populations, and with what level of certainty.

Urgent need for building bridges between the regulators, HTA, and clinicians

Presentations 

• Kit Roes (Julius Center/UMC Utrecht) – Extrapolation: a view thoughts from a statistician & the reflection paper
• Wim Goettsch (Zorginstituut Nederland) – Extrapolation of data: Need for innovative HTA methods to combine RWD and RCT
• Saskia de Wildt (RadboudUMC) - Extrapolation in pediatrics: Science and reality

Other information

• EMA’s “Reflection paper on the use of extrapolation in the development of medicines for paediatrics” October 30 2018 update