Rare Disease Appraisal Landscape and Bringing Novel Technologies to Market

This session, part of our 2021 Fall Pharma Progress event, provided an overview of the appraisal landscape environment for products for rare diseases, its challenges, and how gene therapies are transforming the lives of patients today.

James Horscroft, Principal Insight Analyst, addressed the perceived market access challenges with rare diseases followed by colleague Jane Moorhouse, Principal Insight Analyst, highlighting the historical challenges in evidence generation for HTA for such drugs and the adaptations that some agencies
have made to the appraisal process.

Peter Jackson, Chair of the NICE HST Committee, spoke of how to overcome
challenges in the selection and appraisal of new technologies. He argued
that managed entry agreements – short-term arrangements often touted as
a ‘panacea’ to the challenges posed by rare diseases – cannot address the
uncertainties that HTA and regulatory bodies want to capture, because many
of those uncertainties are far in the future. Yet the burdens of developing rare
disease treatment must be addressed now; Peter described the huge burden
on patient groups, and how manufacturers and the health service aren’t yet
equipped to pick up the work in their place.

Matthias Bischof, Senior Director, Global HEOR at Novartis Gene Therapies,
spoke of the need for a pragmatic and balanced view of the trade-offs needed
to bring treatments for rare diseases to patients. His personal view is that there
should be greater flexibility in terms of evidence requirements and greater
alignment between regulators and HTA agencies in terms of the evidence they
are willing to accept, given that evidence generation is particularly difficult
in this space. For Matthias, a more holistic view of the long-term benefits of
treatments for patients is key, rather than the initial cost-effectiveness. Matthias
also highlighted the different characteristics of conventional therapies and
gene therapies as more manufacturers focus on gene therapies as the future
for rare disease treatment.