Having worked in the NHS as a hospital pharmacist before moving into market access roles in the pharma industry, Price has seen many changes that have affected the HTA process – and this has only accelerated in recent years.
“The scientific advances we are making today, many of which would have been improbable just a few years ago, are incredible. But they also present challenges to market access.
“Cell and gene therapies involve a one-time dose, which raises questions about how you measure treatment success when it may take some time to observe the long-term benefits, especially in terms of overall survival.”
Some of these therapies, such as Yescarta and Kymriah, have been approved based on compelling efficacy data from relatively small non-randomised phase 2 studies – meaning there is some reticence from patients to enter into randomised controlled trials (RCTs) in a space where equipoise is quickly fading. From an HTA perspective however, the lack of randomised data together with the short-term overall survival data are two key factors that create uncertainty when it comes to appraisals of evidence.
“On top of that there can be an uneven cost structure – the cost is upfront but the benefits accrue over many years,” says Price. “The unique characteristics of cell and gene therapies require us to look differently at how we achieve timely market access.”
Like many others in the industry, Price is particularly interested in the potential of real-world evidence (RWE) to address these challenges – collecting data post-launch could enable innovative payment models, such as spreading payment over time for one-off treatment administration.
As RWE becomes more commonplace it will help facilitate the use of flexible payment models – for example those using iterative evidence generation over time, rather than making binary decisions about the likely value of a new treatment at a single point in time. This could be important for the growing number of personalised medicines likely to be launched on the basis of single-arm data.
Price says that Janssen is “investing heavily” in RWE, particularly in initiatives such as federated data networks, where the company is linking information from many different databases across several countries into a common data model.
An example of this is the company’s HONEUR (Haematology Outcomes Network in Europe) network, which now comprises 144 sites with information on more than 18,000 blood cancer patients.
HONEUR’s goal is to enable participants to quickly scale and leverage RWE to answer questions in real time, which will accelerate research and improve conclusions by analysing treatment data from as many sources as possible.
“If we want to truly move towards a more value-based healthcare model where we track, monitor, measure and reward the delivery of outcomes in real-time, RWE is key,” Price says.
He notes, however, that the acceptance of RWE is variable between different countries, and that more investment in and wider acceptance of this type of evidence is needed.
“There’s certainly more we can do in that area. We’re seeing a range of responses from HTA agencies across the EMEA region.
These alternate data approaches could also include different endpoints for innovative cancer therapies.
“In many HTA systems a lot of weight is put on showing an overall survival advantage as a means to demonstrate the clinical value of cancer treatments.
“As treatments start to have longer-term impacts, HTA agencies need to become more comfortable with making timely decisions on the basis of intermediate or surrogate endpoints, such as minimal residual disease (MRD) negativity in multiple myeloma.”
Price is calling for more “open dialogue” between stakeholders on how the sector can address this new paradigm – where treatments are getting better, patients are living longer and, as a result, different kinds of data are emerging.
“A lot of these HTA processes were developed and set into place 15-20 years ago. The world has changed. Treatments are far more effective. We owe it to patients to make sure that we continue on that journey by modifying our processes.”
He says the best example of this is in HIV.
“When I started in the industry, the endpoint we used in HIV trials was overall survival. Today you wouldn’t dream of using that, because treatment advances mean many patients have a life expectancy that’s near-normal.
“We need to learn from that and evolve our decision-making in therapy areas such as cancer, and this needs to start with an understanding and recognition of the challenge we have ahead of us.”
The programme has three objectives.
“The first is to establish a common understanding among different stakeholders and communicate why we need to reform HTA appraisal processes,” Price says.
“The second objective is to come up with solutions. It’s incumbent upon pharma to produce ideas on how we can evolve HTA methods so they continue to be fit for purpose.
“The third element is working with the external world to try and effect change. We believe that we need more opportunities for dialogue and collaboration among different stakeholders – everyone from patients and patient advocacy groups, to clinicians, payers, policy-makers and health economists – so we can drill down into which of the different options need to be implemented. We can’t be successful if pharma is the lone voice in all this.”
He says these options could include things like intermediate endpoints, risk-sharing payment models, or ‘adaptive HTA’, where after an initial decision the company commits to collecting additional data, perhaps through RWE, and the drug is reassessed further down the line.
“Patient access has to be at the centre of all this,” says Price. “If stakeholders can recognise that HTA reform is needed to ensure access, then hopefully we will see increased collaboration to achieve that shared vision.”
Price’s main hope is for a more flexible approach to HTA in the future.
“At the moment, decision-making frameworks can be quite rigid. If you don’t show certain endpoints immediately, the clinical rating by HTAs is often downgraded.
“Within oncology, we see different tumour types acting and responding in different ways. Perhaps we need to have alternate types of outcome measures depending on the specific cancer. That will enable us to make better decisions for patients earlier on.”
He adds that one of his main frustrations is with the large amounts of variability across countries in how their systems work.
Price says that generally HTAs seem open to this kind of dialogue.
“Some newer technologies like CAR-T are causing them to look at these issues and think about how they can do things differently. Generally, regulators have been much more agile in how they assess the benefits of new treatments than payers have.
“If we could have more opportunities to talk under safe harbour conditions about some of these challenges, that would be of great benefit to everyone.”
Martin Price is vice president of health economics, market access and reimbursement in Europe, Middle East and Africa at the Janssen Pharmaceutical Companies of Johnson & Johnson, a role he has held for the past six years. In this capacity, Martin leads the teams responsible for achieving optimal and accelerated market access, at a fair and value-based price, for Janssen’s new products and indications. Prior to this, Martin worked in Janssen’s UK affiliate, latterly as external affairs director, where he was responsible for Market Access, Communications and Government Affairs. Martin joined Janssen in 2001 from GlaxoSmithKline, where he began his career as senior health outcomes manager.
George Underwood is a senior member of the pharmaphorum editorial team, having previously worked at PharmaTimes and prior to this at Pharmafocus. He is a trained journalist, with a degree from Bournemouth University and current specialisms that include R&D, digital and M&A.