The Dutch advanced therapy medicinal products (ATMPs) field is highly fragmented and needs central coordination to unblock bottlenecks. Dutch researchers made the recommendations in a recent report offering insight gaining an efficient overview of ATMP regulations.
The researchers mapped the Dutch ATMP field and performed a bottleneck analysis to provide recommendations to various stakeholders as the EU starts to revise its pharmaceutical legislation. They encouraged the different parties to analyse their needs and actively contribute to the consultation processes.
“If we put it bluntly, we should stop doing more bottleneck analyses and start acting. Because, over the years, specific stakeholders have performed multiple bottleneck analyses about specific topics,” Christine van Hattem, one of the report’s authors, told EURACTIV.
They aimed to produce a comprehensive bottleneck analysis and to also look at the solutions. While the report was heavily focused on the situation in the Netherlands, it also took into account the European context.
“Now we know where the gaps are and what we need to do,” van Hattem said.
Patient access to novel therapies
The European Medicines Agency describes ATMPs as medicines for human use that are based on genes, tissues, or cells. They offer groundbreaking new opportunities for the treatment of disease and injury. ATMPs are classified into three main types – gene therapies, somatic cell therapies, and tissue-engineered therapies. When ATMPs contain one or more medical devices as an integral part, they’re referred to as combined ATMPs.
Researchers from the University of Utrecht and the University Medical Centre (UMC) Utrecht compiled a report that offers actionable insights and recommendations for developers, assessors, policymakers, healthcare professionals, and other parties in the Dutch ATMP field.
The ultimate aim is to facilitate patient access to these novel therapies. The research was funded by ZonMw, a Dutch research foundation, as part of a wider project on pandemic preparedness.
Apart from conducting desk research, the authors also held discussions with different stakeholders.
“The willingness of everyone to collaborate and work on better solutions was impressive,” Jurriaan Gort, who also helped author the report, told EURACTIV.
Helpdesk answering regulatory questions
The report’s authors suggest creating a national regulatory helpdesk to address stakeholders’ questions on regulations, particularly those from small private and public developers. The focus would be to provide answers in an accessible way on the ATMP regulatory framework, the different parties involved, and how to navigate the field properly.
“The recommendation of having a national central organ to coordinate stakeholder interaction and the initiatives for ATMP development cannot be overstated,” van Hattem said.
Other recommendations include ensuring the involvement of patient organisations in ATMP development, examining the growing role of academic drug development, and the creation of accessible information about ATMPs to help the general public create an informed opinion about them without creating unrealistic expectations of the effectiveness and safety of the new medicines.
The report’s authors say that ATMPs include high-tech medicines that can offer new treatment options for previously difficult-to-treat or even untreatable conditions. They added that they have the potential to revolutionise healthcare and can be considered key technologies.
Harmonisation, a more competitive EU
While the researchers argued that it is crucial that ATMPs can be deployed quickly, safely, and efficaciously in clinical practice, they identified new bottlenecks concerning alternative preclinical models to better predict clinical effects and the applicability of payment models.
Previously described bottlenecks in the development and application of ATMPs include a lack of clarity about the regulatory pathways to make them accessible and bottlenecks in the related record-keeping requirements.
At an EU level, the authors encouraged better harmonisation between EU countries, given the fragmentation of documents, requirements, and procedures.
“Harmonisation might help to increase the competitiveness of the EU,” explained Gort, with the disclaimer that competitiveness itself was not included in the aim of their research.
Using reimbursements as an example of a process that’s fairly different in each European country, Gort highlighted how complying with all the different regulations in all the different EU countries can be fairly difficult for developers.
“To make it easier for developers to get their ATMPs to reach the patients with a feasible business strategy, having the process further harmonised between different countries might help,” Gort said.
New HTA framework
The EU is on track to ease at least some of these issues related to a lack of harmonisation with its new regulatory framework for Health Technology Assessments (HTA). In 2025, cell and gene therapies will undergo a single EU assessment rather than one for each member state. Gort believes the new EU-HTA Regulation is expected to partially address the differences mentioned earlier.
“ATMPs have a future, but we’re still figuring out the details [aligning current development and the regulatory framework] and how to make them accessible and affordable to everyone,” said Gort.
[By Christoph Schwaiger, Edited by Vasiliki Angouridi, Brian Maguire | Euractiv’s Advocacy Lab ]
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