For a parent who has been told that there are no further treatment options and their child is going to die, and then seen that young girl thriving and cancer-free seven years later, the value of the new era of cell and gene therapies is clear.
According to the Alliance for Regenerative Medicine there are nearly 900 companies worldwide conducting over 1,000 clinical trials, two thirds of which are late-stage clinical trials. In 2018, the global industry attracted $13bn of investment and saw $19bn worth of mergers and acquisitions. This investment comes with the expectation that the healthcare system will pay a fair price for these life-changing medicines. Many of the first generation of successful advanced therapeutics share a common set of characteristics; they have high efficacy, they are expected to have a long duration of effect resulting from a short period of treatment and they are expensive to manufacture and provide. With healthcare systems increasingly making payment decisions based upon the value delivered by an intervention, these new drugs can justify a very high price, if they deliver on their promise.
The question of a willingness to pay breaks down into a sub-set. Firstly, should we pay a higher price today to get access to drugs which might become cheaper or better in the future? Secondly, can even the reduced price that they might reach in the future be justified? Thirdly, because we are being asked to pay up front today for many years of healthcare benefit in the future, how do we afford the total cost and what happens if they don’t work as expected? There is another, perhaps less valid, but no less real, issue that comes in the same discussion: that we have a government and healthcare system built on the close short-term link between payment for drugs and their benefits.
In order to accommodate paying for long-term benefit over the longer term we would need to make some high-cost and complex changes to the healthcare operating model. As an illustration of this, consider a hypothetical one-off treatment for haemophilia. Current healthcare costs are in the region of $150k per annum per patient which would add up to $6m over 40 years of treatment. A therapeutic which cost half of that $6m but removed the annual costs could still leave substantial savings for the healthcare provider in the long term. The key issues are: how you would pay the up front costs for the 6,000 patients in the UK ($18bn), and how do you manage the systems so that you are paying for benefits that are actually received?
At the Cell and Gene Therapy Catapult we are concerned with prompting and facilitating the industry (which includes the NHS) in bringing about this change. The details and blueprint are yet to be determined but from our work to date we can see some clear considerations for how the change should be approached and how its success should be measured.
Clarity of intent
In the centre of the discussion are patients, healthcare providers and manufacturers. The most basic solution, which will only work with a small number of therapeutics, is an agreed price which reflects good value today compared to today’s other healthcare priorities. This would not stimulate new investment, bring on the economies of scale or scope that could lead to lower prices in the future. At the other end of the spectrum we have the interest of society as a whole in the generation of an industry which creates jobs and wealth. We need to be very clear about what we are trying to achieve.
The aspiration should be to engineer a change in the system which is applicable to all novel medicines and advanced therapeutics. It should not be something that just works for cancer such as the Cancer Drugs Fund (CDF) or in rare diseases because, by virtue of being rare, the cost can be easily absorbed.
If we are to re-engineer the relationship between innovators and payers, let’s do it in a way that accommodates future innovation. Advanced therapeutics have their own challenges and we understand them. Patient-specific medicines and the genomic data revolution will bring a whole new set of challenges that we should try to anticipate.
Any changes should not be a zero-sum-gain. Simply moving risk from one party to another party, which is more willing to take it, can create economic value. But our aspiration should not stop there. There is value in the NHS that can be released by using it to foster innovation. There is value to society in the jobs and productivity that arise from a growing industry.
Any solution that looks good on paper but ignores behaviours and borders will not be effective. If there is not self-interest in the solution stakeholders will not engage. Nor should anyone be given tasks and expectations that are beyond their mandate. It is not the role of the NHS to stimulate economic growth. Economic growth and particularly growth in productivity is important and other key stakeholders (BEIS, HMRC) need to have their role recognised and defined.
We shouldn’t produce a solution that has no relevance to the rest of the world. It does not need to be a new global standard, but it does need to be understandable, and adoptable.
The tendency of the system and people to revert to what they are familiar with is a danger here. The change needs to be big enough to breakaway from the gravitational pull of today’s bureaucracy. Fortunately, the benefits of these new therapeutics are strong enough to have a pull of their own towards a new way of operating.
As we embark on creating a new relationship between innovators, healthcare providers, payers and patients, continuously calibrating and testing our proposals against these criteria will increase our chances of sustainable success. As we progress we also need to bring people with us. Change of this nature requires political support and for politicians to respond to their constituents, and the voice and support of patients.
We should consider the cost of not redefining this relationship. Firstly, patients may not have access to the life changing medicines, making them second class citizens in global healthcare. Secondly, we fail, again, to harness the power of the NHS to foster and stimulate innovation and thirdly, we miss the opportunity to capitalise upon the world leading expertise of our own researchers, clinicians and companies and build a £10bn industry creating 18,000 new high-value jobs in the UK. The Cell and Gene Therapy Catapult is part of a strong ecosystem of advanced therapy stakeholders who are determined that the benefits of these life-changing medicines reach those who need them.
Matthew Durdy is chief business officer at the Cell and Gene Therapy Catapult .