Coverage of bluebird bio’s withdrawal of Zynteglo for beta thalassaemia from Germany after pricing negotiations collapsed, and the company’s recent announcement that it is winding down its European operations, sent ripples of anxiety through the industry. What can we learn from this to navigate market access challenges and pricing negotiations to enable patients to benefit from breakthrough science?

The promise of science hasn’t always easily translated into timely market access for innovative therapies.

With more companies expecting to seek regulatory approval for cell and gene therapies (CGTs) in the coming years, market access continues to be challenging for these products. The incredible promise of recent scientific advances to develop lifesaving and life-changing treatments is increasingly recognised by health policy makers, who are striving to ensure that they can harness the benefits of research and innovation. But the promise of science hasn’t always easily translated into timely market access for innovative therapies. This is not unique to CGTs but is increasingly concerning if patients are truly going to receive the benefit of these therapies.

Health Technology Assessments (HTA) and reimbursement processes were, of course, not designed with CGTs in mind. Innovative approaches are sometimes needed to address the twin barriers of cost and uncertainty of long-term benefits. There are promising signs that this may be, at least in part, addressed through reformed HTA processes: a joint clinical assessment is now possible at the EU level and national HTA bodies are explicitly recognising the challenge of CGTs, e.g., in the recent consultation on HTA methods by NICE in the UK.

Whilst individual companies may not have the capacity to engage with multiple policy initiatives, trade bodies and coalitions such as the Alliance for Regenerative Medicine (ARM) continue to seek to shape international and national policies in this area.

However, the tension between financial pressures on health systems and innovation is likely to remain a real challenge as more CGTs are approved, particularly if this starts to include treatments for more common conditions. But this is not insurmountable – there have been, and there will continue to be, successes. Individual companies can maximise their chances of a successful launch with well-planned preparations.

Over the last 12 years I have seen companies well-prepared for their HTA and others who have seemed surprised that their product is not immediately welcomed with open arms by HTA bodies and payers. As is so often the case, preparation is key and there are steps that can be taken early to help prepare for the challenges of market access, including:

• Developing a market access strategy that builds on a clear understanding of the perspectives of HTA bodies and payers, as well as patient and clinician needs and expectations of new treatments. A market access strategy should be pragmatic, looking beyond formal routes for engagement with key stakeholders.
• Engaging early and often with payers and HTA agencies in key markets. This allows companies to prepare key decision makers, educating them about the new treatment, the science behind it and the disease it targets as well as gaining early insights into the likely receptiveness of the anticipated value proposition and evidence base. Identifying gaps in the evidence allows companies to address them and minimise the delays and frustrations of discovering too late what is really important to payers and HTA bodies.
• Designing clinical trials with HTA and reimbursement decisions in mind, as well as the demands of the FDA and other regulators, will help ensure a stronger evidence base at the point of launch.
• Generating evidence alongside clinical trials, such as disease level quality of life and burden of illness evidence, particularly for rare or less well-understood diseases, can help define the burden of disease and the value of new treatments.
• Developing innovative approaches to funding and reimbursement that are responsive to the legal, practical and philosophical realities of payers.
• Collaborating with patient groups and other stakeholders on areas that are non-competitive such as disease level data can be an efficient approach to building evidence.

Whilst these preparations may not guarantee success in challenging markets, not preparing will likely guarantee failure.

Hanover supports companies in shaping the external environment to facilitate patient access to innovative therapies. Through our integrated public affairs, patient advocacy and market access approach, we work with organisations to navigate the challenges of bringing new treatments to patients. 

For more information about Hanover and our Market Access offer, please contact Emma Eatwell.