The next Advanced Therapies Network (ATN) event on 17th September in London will cover the subject of reimbursement. The symposium aims to get to the heart of this complex issue which is central to ensuring current and future ATMPs reach the patients who need them. A recent report by Alliance of Regenerative Medicine (ARM) provided some interesting insights on the topic.

Reimbursement is becoming an increasingly common theme amongst the growing number of reports and conferences in the advanced therapies sector. The upcoming ATN event is part of the Phacilitate Leaders Europe conference where several sessions in the patient access and healthcare stream are examining the topic. Recently (July 2019), the Alliance for Regenerative Medicine (ARM) published a report which analysed Health Technology Assessment approaches to ATMPs in six European countries, including the UK.

This ARM report entitled ‘Getting Ready: Recommendations for Timely Access to ATMPs in Europe’ delivers a range of interesting comparisons and recommendations. Based on a literature review and stakeholder interviews it highlights the need to embrace different approaches to reimbursement that consider the potentially transformative nature of these therapies.

In the same month ARM produced a joint report with the UK Bioindustry Association (BIA) on the UK’s ATMP landscape, emphasising the growth of the sector. It celebrated the position the UK holds in the sector which is home to nearly a quarter of the 224 cell and gene therapy companies in Europe and Israel, a number which has grown over the years.

These reports suggest the potential value of this sector is well-recognised and well-known, and now it is down to finding solutions in this unique sector, which includes developing and implementing innovative reimbursement options. With more ATMPs receiving market access, appropriate reimbursement will be crucial to ensuring patient access to these potentially transformative therapies.

New approaches and evidence

Probably one of the strongest take-home messages from the ARM report is that the sector has seen positive adaptations in regulation pathways but that a parallel shift is also needed in current health technology assessment (HTA) methods and payment models to take into account the novel properties of ATMPs and their potential.

The report found that payers usually put more emphasis on uncertainties around long-term benefit, demonstration of efficacy and safety, validated clinical outcomes and perceived affordability. This means current HTA frameworks are not always appropriate for ATMPs in terms of managing uncertainties around long-term data on safety and efficacy.

“There is a paradox between regulators’ approaches in providing early access for ATMPs for patients’ benefit and HTA/payers’ reluctance to provide access until the long-term profile has been fully characterised. This challenge is faced in all the six countries examined in this report.”

ARM report: Recommendations for Timely Access to ATMPs in Europe

To enable development of appropriate reimbursement models the report suggests greater use of real-world evidence and further development of the infrastructure required to collect and use high-quality evidence of this type.

 

Opportunities for early dialogue

Another central recommendation by the ARM report is the establishment of greater opportunities for early dialogue between industry and payers.

In a recent interview, Pilar Pinilla Dominguez, Senior Scientific Adviser at NICE and one of the speakers at the upcoming ATN event, discussed the importance of dialogue and stressed that NICE encourages companies to engage early with both NICE and NHS England. She also mentioned the Cancer Drug Fund (CDF) and how this has provided a tried and tested framework for all parties to understand how the significant uncertainties with these novel cancer treatments could be managed, most notably in the examples of the two CAR-T cell therapies: Novartis’ Kymirah and Gilead’s Yescarta.

The ARM report cites the CDF as an example of new approaches to reimbursement in the advanced therapies sector and calls for more widespread use of this type of model where reimbursement is temporary and conditional on the collection and review of further evidence, potentially allowing for future price re-negotiations.

Early dialogue is also one of the priorities of the Cell & Gene Therapy Catapult who work with companies, ranging from SMEs to larger biotechs and also academics to encourage them to think about reimbursement during the early stages of development. Panos Kefalas, Head of Health Economics & Market Access at the Cell & Gene Therapy Catapult is an expert on the reimbursement of advanced therapies and will also be speaking at the ATN event on 17 September.

Alongside Pilar, Panos will join Tamir Singh, Head of Commercial Development at NHS England and, Francis Pang, VP of Global Market Access at Orchard Therapeutics. Francis will be giving the industry viewpoint on this topic and will also be providing a pre-event interview which will be published in the coming weeks.

Challenges in implementing changes across payment models and evidence collection

As well as highlighting the need for different approaches to reimbursement that are more attuned to the nature of ATMPs, the ARM report also recognises the challenges in choosing and implementing them. They will require new data collection infrastructures to generate and use real-world evidence. They will also need a new kind of expertise to put them in place and ensure they are working and, most likely, will need to be adapted to different health system and country specific requirements.

The report concludes that ATMPs are a broad, growing and highly heterogenous class of therapies. As such new approaches for accelerating access need to be tested and refined and the lessons learned must be shared. There is clearly a drive to bring together different viewpoints and collective expertise in this area in order to move forward, and we hope the next ATN event will provide the environment to do just that.

The ATN event on reimbursement is on 17th September at the Royal Lancaster, London. It is free to ATN members and will be part of the Phacilitate Leaders Europe conference. ATN members who wish to attend the rest of the conference can get a discount code. To register for the event log on to the ATN platform or become a member and visit the events page of the platform.