Advanced therapy medicinal products (ATMPs), like gene therapies are at the forefront of innovation offering potentially long lasting and life-changing solutions for people living with complex and rare diseases. Their inherent nature – single-administration treatments with great therapeutic potential, high upfront costs, and limited evidence on long term durability of effects at the time these medicines are launched – make their access and availability to patients complex and slow. Policy makers have a crucial opportunity in this mandate to leverage this critical moment of change and enable equal access for patients through the EU.
In the past years, several Member States have piloted innovative procurement and contracting, such as outcomes-based agreements, to balance affordability and sustainability, while recognizing the value these therapies bring to patients, their caregivers and healthcare budgets.
“We are encouraged by European governments who send a strong signal of how innovative and collaborative thinking can make gene therapy a reality for patients”, commented Diego Sacristan, Senior Vice President International at CSL Behring. Significant access milestones have recently been achieved in EU countries including Germany, Spain, Denmark, and Austria, where patients with a rare bleeding disorder can now benefit from gene therapy.
Healthcare experts discuss how to build on these access milestones in more countries. “Advanced therapies are the future of personalised medicines, and Europe should be a global lighthouse in both developing these life-saving therapies and, crucially, getting them to patients. The new EU Life Sciences Strategy and the Biotech Act must ensure that new, innovative therapies will be both developed, trialled and launched here in Europe” says MEP Stine Bosse, who is the vice-chair of the health committee in the European Parliament and co-Chair of TRANSFORM.
Advanced therapies are the future of personalised medicines, and Europe should be a global lighthouse in both developing these life-saving therapies and, crucially, getting them to patients
At this year's Cell & Gene Meeting on the Mediterranean, participants addressed how the ATMP sector and healthcare systems can work together to deliver these therapies. "There are promising examples of innovative payment arrangements in both the US and Europe that we can all build upon to improve access for patients," said Paolo Morgese ARM's Vice President of Public Affairs for Europe.
“It is very useful to look for innovative financing models at an early stage for new innovative treatment options that are not as easy to finance as previous therapies, together with politicians, the pharmaceutical industry, scientific societies and practitioners, so that patients are not deprived of this therapy”, says Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, Germany. His country recently agreed a first in time nationwide patient-individual payback model for a gene therapy. This model essentially offers to pay for a gene therapy in a budget-neutral manner, when compared to the chronic therapy it replaces.
“We will always lack large-scale data on the efficacy of innovative therapies to treat rare diseases. There are a couple of ways to address this. First, we must get multi-country clinical trials and health data sharing right to help innovative companies choose Europe for their most innovative trials. Second, public health systems can control costs and ensure high efficacy by only paying for what works. That’s value-based healthcare.”, says MEP Bosse. Her country recently became the first Nordic and European country to adopt a performance-based model, where costs are incurred only as long as the gene therapy proves effective over the agreed long-term period.
“As the Commission updates the EU public procurement rules later this year, it should consider launching innovation-based criteria that incentivise the development of new, ground-breaking therapies”, MEP Bosse adds. Additionally, “the revision of the public procurement directive should better support and clarify how to implement tailored procurement solutions with innovative payment models for gene therapies”, commented Diego Sacristan. “Another key aspect is to engage in early and iterative dialogue with payers and governments to pilot and advance novel HTA assessments”, Diego added.
Professor Wolfgang Miesbach adds that “optimal infrastructure of specialized centers, resources, healthcare and patient trainings are key to ensure the administration of the gene therapy to patients. When the right access pathways, infrastructure and expertise is missing, the cross-border healthcare directive should be a solution”. “I am confident that our citizens and patients see the value of more Europe in healthcare very clearly. As we review EU rules for access to cross-border healthcare, we policymakers must build a framework that ensures that all Europeans have access to life-saving therapies no matter where in Europe they live or where they were born.”, MEP Bosse concludes.
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