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What important lessons can we take from the development process of Glybera?

May 10 2016

Are you or your company currently in the process of developing a novel gene therapy? Perhaps you are also trying to acquire funding for it? Then continue reading and take the relevant lessons ttopstart picked up from a recent MIT technology report1, describing the difficult market introduction of the world’s only approved gene therapy so far; Glybera developed by UniQure. 

The first gene therapy approved in the Western world, Glybera to treat lipoprotein lipase deficiency, costs $1 million per treatment and has been used just once. Those are not the numbers you are dreaming of when you are developing a new drug. However, every cloud can have a silver lining and better numbers are certainly feasible, if you consider the following carefully:

Set-up of your clinical trials
One of the most important steps in your development process is the set-up of your clinical trial as such that the result will be solid and convincing. In case of Glybera, “the drug was given to just 27 people in three “open-label” experimental studies, meaning no patient got a placebo. Those tests never showed a lasting change.” Going back to clinical trials (“last fall UniQure chose to scrap its plans to sell the drug in the U.S. after the Food and Drug Administration said new, expensive trials would be needed”) is a waste of resources and time. This should be prevented by proper designing of your plan as early as the preclinical phase.

Dialogue with regulators and insurers
“By 2012, the company had failed twice to convince European regulators, (…). Nor has Glybera convinced the national regulators in Europe who decide what drugs get reimbursed. Last year, French authorities said they would not pay for the drug. Germany judged Glybera’s benefits “non-quantifiable.” This underscores the urgency that should be given to the dialogue with regulators and insurers early in the development process (also illustrated in our design for reimbursement strategy). They can also provide feedback to point one. They might have useful tips for the design of the clinical trials, which can later ease the approval process. Moreover, a proper dialogue can also contribute to the following: “with nearly 670 gene-therapy trials under way, and 68 in the later stages, known as Phase III, it’s becoming urgent to understand how these therapies will be paid for.”

Pricing and sales
It is fairly straightforward, a 1-million-dollar price tag scares off anybody. “The decision to charge around $1 million for Glybera (the exact amount depends on the patient’s weight) does raise questions. Some people think gene therapies should be paid for in yearly installments, and only so long as they keep working.” From the beginning, this should be kept in mind, a price that is too high will undermine your success. Thus use your resources carefully, to prevent unnecessary increase of your price. In addition, UniCure “has turned over European sales to the Italian drug maker Chiesi Farmaceutici, which calls selling the drug challenging." So far they have failed to do their job.  This has amongst others to do with their limited experience in the area of complex therapies. Determining your commercialization strategy and identifying barriers on the route-to-market -early on- can prevent running into problems such as these.

It is obvious from these lessons that they do not only apply to gene therapy but also to drug development in general. The lessons are aligned with the principles that ttopstart offers in its services (FFWD, traffic light model). As such, ttopstart is perfectly equipped to support you in developing a new gene therapy or other drugs by identifying relevant funding and strategic advice in all different development stages.



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Andree Schram, PhD
+31 (0)30 7370779