Gene therapy market access Q&A

What do you see as the big barriers to adoption of novel payment mechanisms in Europe?

Historically, there’s been a fair amount of reluctance to adopt novel payment mechanisms like outcomes-based reimbursement schemes, often because of concerns around the administrative burden of implementing them (combined with past examples that did not work out quite as intended).

Thankfully, I think there is a shift in attitudes towards this, but there are a couple of big fish still needing to be fried, like ensuring that an appropriate data collection infrastructure is in place, tackling the narrow budget cycle focus of both payers and manufacturers (revenue recognition legislation plays a part here), and silo budgeting between health and social care. There are other important issues relating to this also, like making sure that there are enough data managers in place, and that everyone benefits and learns from the data that is collected, as well as recognising the importance of patient-reported outcomes, so there are plenty of factors that can contribute to improving the situation.

Could Europe steal a march on the US if it gets cell and gene market access right?

I tend not to think about this as a competition, but more in terms of how different countries and healthcare systems have evolved differently over time to present different opportunities and challenges.

The US differs greatly from the European countries in that 1) typically there is a higher willingness to pay, and that 2) there are many more payers. In most European countries, manufacturers deal with one public body acting as the insurer and payer, which increases the bargaining power of the insurers and makes the reimbursement issue almost binary (hit/miss).

In the US, there are more potential avenues (ie, insurers) for getting your product used in patients, which means that failure to get coverage with one insurer does not mean ‘game over’ for companies.

On the other hand, if you succeed in getting reimbursed, for example, in France, you have much greater reach in that country, as there is one central health insurer, so there are pros and cons.

The very first pioneers in gene therapy in Europe hit profitability problems – and even the current generation aren’t getting returns yet. What’s going to unlock the long-term sustainability of the field – evolution in cell and gene manufacturing platforms or novel payment methods?

I think both will be vital to the long-term success of this relatively new field of science. Essentially anything that reduces the cost of getting new products to patients and improves payers’ confidence that money is spent wisely will help.

Some of the earlier cell and gene therapy launches were hampered by less than paradigm- shifting efficacy profiles, which made it hard to justify step-changes in price/cost. There’s a lesson in that, to take reimbursement considerations seriously from an early stage, because no one is going to fork out large sums for minor improvements. And frankly, as a UK taxpayer, I’m glad that that’s the case.