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Accelerated access post Brexit – snippets from the Evidence Base webinar

Health regulatory bodies such as NICE, are often tasked to make decisions about health technologies with limited clinical and cost-effectiveness evidence, leaving much room for uncertainty. This is often the case for treatments that target rare diseases due to small patient populations. There is an increasing demand for early access programmes to allow for improved data collection, however it is important that manufacturers understand how to navigate uncertainty.

The Evidence Base, an online hub that provides insights on topics including real world data, health economics and outcomes research, hosted a webinar on 21st July entitled Accelerated regulatory approval in 2022 and beyond – managing uncertainty with the impact of Brexit and COVID.

The event, chaired by Martin Strandberg-Larsen, senior Director of Market Access at global diagnostics company, Veracyte, included the keynote speakers Leslie Galloway, chair of the Ethical Medicines Industry Group (EMIG) , and Melvin ‘Skip’ Olson, global head of integrated evidence strategy and innovation at Novartis.

Topics covered included:

  • How regulators have typically handled areas of unmet need
  • Navigating UK vs other drug reimbursement pathways
  • The future picture of accelerated regulatory approval for rare diseases
  • How the new NICE guidance on real-world evidence will affect the field

Galloway spoke about accelerated access in the UK and the EU, while Olson spoke about accelerated access in the US and the EU .

The keynote speakers highlighted the importance of timing if accelerated access is to be achieved. Olson highlighted the difference between accelerated  regulatory approval and accelerated reimbursement. The speakers agreed that engaging early and simultaneously with both regulatory and reimbursement bodies may mitigate delays to accelerated access. Olson added that, because the UK has one central reimbursement body, NICE, it might be  slightly easier to navigate what is accepted as evidence for rare diseases in the UK – compared with countries that have several reimbursement bodies, where standards for evidence may differ.

Keynote speaker Galloway contrasted the UK government’s spending on healthcare and medicines to that of countries in the EU. Galloway believes that the lack of increase in the percentage spent on healthcare and health medicines, has led to increased rebates and increases in the voluntary scheme, ultimately creating a difficult environment for pharma to achieve accelerated access and cost-effective reimbursement. However, he went on to highlight the innovation medicines fund, and its potential to increase access to innovative medicines. Galloway advised that engaging with NICE scientific advice may be helpful to organisations to identify the relevant gaps in evidence that should be answered from the real world evidence (RWE). He also briefly discussed the new severity modifier and highlighted that the definition of severity varies between people and may be an issue that will need to be clarified.

Both Olson and Galloway welcomed the new NICE guidance on RWE. The webinar host, Larsen, agreed, pointing out that the new guidance is welcomed because the way data is collected for rare diseases is  significantly different to way it is collected for other disease areas. For example, conducting a randomised control trial (RCT) for rare diseases is quite different to conducting RCTs for other, more common diseases for several reasons, notably the small number of eligible participants. Larsen also used the accelerated introduction and the simultaneous ongoing monitoring for the different COVID-19 vaccines as a good example of real world evidence in the area of an unmet need.

Larsen concluded the webinar stating that he ‘hopes’ the NICE RWE will take into account the patients perspective to ultimately accelerate access of innovative medicines to patients.

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