Changes in the drug development paradigm; keep dreaming?

Published 21 February 2017

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We all know the current paradigm in drug development is far from perfect and does not always suit patients’ needs. More specifically, new treatments without sufficient earning potential are not likely to be developed into new products, despite the potential benefits for patients. We all accept this; it is the price we pay for the system designed to protect that same patient. And of course, patients need to be protected and the public needs to be able to trust that new treatments are indeed safe and working. So, we developed a system in which huge amounts of cash are needed to put a new product on the market. As a result, prices for new treatments are sky-rocketing and -even more bothersome– many high potential treatments will never reach patients at all. Indeed: the price we pay.

At EATRIS, our focus is translational research. How can we increase the efficiency of the drug development, and ensure that patients are reached faster and with better treatments? One clear bottleneck is the gap between academic researchers, funders and regulators, and recently I came across a very nice showcase in which exactly this gap was overcome.

Prof. Jolanda de Vries from Radboudumc has been working on dendritic cell based vaccines for many years. This is a promising and emerging approach, especially in the treatment of (metastasized) melanoma. The problem is that the treatment is complicated and hence expensive; it is an autologus cell therapy which means it requires harvesting and treatment of the patient’s own dendritic cells. Moreover, just as with regular vaccinations a single set of treatments is sufficient to boost the patient’s own immune response. This might be nice for the patient, businesswise it is less attractive. So, end of story? By no means!

Prof. De Vries successfully applied for a specific conditional approval program in the Netherlands. Under the arrangements of this program, costs of this experimental treatment are actually reimbursed by the Dutch health insurance. As a result, a nationwide clinical trial has been initiated (EudraCT Number 2015-005322-19), which is co-sponsored by the supplier of the kits needed to treat the cells.

There are multiple reasons I like this so much. First and foremost, of course, it is great that these patients now have a treatment option they would have missed out on otherwise. But I also like the way the various stakeholders are working together; the academic who initiated and is leading the trial, the participating hospitals with their physicians and patients, the government, which granted the conditional approval, the insurance companies (even though they did not have much of a choice after the approval had been granted, they do reap the fruits of successful treatments), and even the company that is co-sponsoring the trial in the hope to generate market share in consumables once the treatment has proved to be effective.

Now, would this just be a black swan, or is this a glimpse on what may become a new paradigm for drug development? Let’s hope for the latter. We should be able to reinvent the system; in fact, we owe this to ourselves. One way to accomplish this is to limit the expenses needed to get new treatments to patients. This can be achieved by granting conditional approvals once the safety of a new treatment has been established. In this way, treatments will become available earlier, at lower costs, and efficacy data will be based on real-world results rather than on results from strictly controlled phase III trials (which often proves not to be too predictive for real-world results). This is even more relevant in the era of personalized medicines.

Let us return to the trial of Prof. De Vries for a moment. After all, it is only for the dedication of Prof. De Vries and her team that this dendritic cell therapy has reached the patient. It is exactly this dedication we will need. In the end, translational research is not a goal in itself; treating patients is the goal.