How did one of the most innovative drugs, Glybera, which heralded the dawn of gene therapy and whose efficacy allowed it to overcome a severe pathology, ultimately fail at its launch?
Gene therapy: a medical paradigm shift
Before going further, let’s recall the principle of gene therapy: it involves inserting corrective genes (healthy DNA) into cells to repair deficient genes causing pathologies. Gene therapy has the advantage of curing the patient for life: the effects of the treatment are not temporary.
Glybera: a drug for a rare disease
Glybera is a drug developed by the Dutch biotech company UNIQURE in partnership with the Chiesi laboratory (Italy). This drug was developed for a narrow indication, namely patients suffering from familial lipoprotein lipase deficiency, a rare genetic disease affecting 150 to 200 people in Europe. The effects of this pathology are severely debilitating and painful: the inability to break down triglycerides leads to an accumulation of fat in the patients’ blood, causing various clinical manifestations (acute pancreatitis).
A record price and major prescription constraints
Around fifty injections are necessary per patient, depending on their weight. On average, for a 70 kg patient, the treatment cost is 1.2 million Euros, making Glybera the most expensive drug on the planet, at least in the year of its launch in 2012.
Beyond the price, which led to a wave of public disapproval, the prescription of Glybera came with significant administrative complications. The Berlin doctor Elisabeth Steinhagen-Thiessen was thus forced to complete documentation “thicker than a thesis” to administer it to a patient. As for the astronomical cost of the treatment, it was ultimately covered by the German insurer DAK.
A therapeutic success… but a commercial failure
As a consequence of the prohibitive price and prescription difficulties, Glybera was administered only once in Germany… yet with a result exceeding expectations, achieving 100% cure… for life!
What does the history of Glybera teach us about sales?
- Innovative technology alone does not justify the price: more than any other, the pharmaceutical sector is subject to strong ethical constraints, due to the particular status of drugs, which are not products like any other. The question “Is there a fair price for medicine?” is a classic debate frequently raised in the public sphere. Price setting must result from a reflection that considers the cost of research, the cost of the effects of the pathology (i.e., the treatments to follow without resorting to Glybera – for the case of the single patient treated, this corresponded to the cost of 40 hospitalizations). Once these elements are taken into account, the “social price” accepted by public opinion and governments still needs to be studied.
- Reputation is quickly tarnished: not taking into account the “social price” threatens communication and brand image. Regarding Glybera, it was simply perceived as “the $1 million drug,” even though its revolutionary technology marked a turning point for research and patient care. Not to mention gene therapy and Glybera, other remedies (for example, against hepatitis C) raised strong protests in 2018 due to their commercialization price.
- All stakeholders must be considered: for the pharmaceutical sector, national health systems are key, especially in contexts of budget restrictions and healthcare spending limitations. Generally, current health systems are not adapted to cover drugs like those used in gene therapy. France, for its part, declared that it would never pay for Glybera; Germany stated that its benefits were not quantifiable. The complexity of prescribing (and more generally, distributing a product) Glybera thus constituted a real hindrance, with the need for case-by-case reimbursement.
- Markets considered unprofitable can become profitable due to regulatory changes: this is the case for orphan diseases, with strong incentives to encourage laboratories to focus on these types of pathologies. For example, laboratories benefit from 10 years of exclusive marketing rights for drugs dedicated to orphan diseases. However, these have long been the “unloved” pathologies of laboratories, due to the too-small number of patients and the significant R&D costs associated with them.
Conclusion
The Glybera case shows that scientific innovation, however remarkable, does not guarantee commercial success. Without an economically, regulatory, and socially acceptable model, this revolutionary drug failed to find its place in the market.
This study reminds us that the launch of a product, including in the pharmaceutical sector, must integrate all stakeholders and go beyond mere technical performance. Innovation is not enough: without an adapted pricing, communication, and distribution strategy, even a major breakthrough can fail.