Great expectations and great expense – new gene and cell therapies bring hope and controversy in equal measure.
I have lived through enough paradigm shifts that turned out to be paradigmatic in name only to view use of this cliché with some scepticism. However, if it is good enough for the commissioner of the FDA then who am I to argue? Indeed, I confess to using this term myself in a recent presentation on Gene and Cell Therapy (one of NetworkPharma’s excellent ‘brunch club’ series – available on NetworkPharma.tv).
“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases. In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer,”
said FDA commissioner Scott Gottlieb
So what development has led to dusting off this hackneyed old bit of business jargon and, more to the point, is it justifiable? Well this time I believe it really is an appropriate use of the term. It may have taken us 40 years to get here but 2017 has seen major new treatments in gene and cell therapy finally making it to market. For certain haematological malignancies Gilead’s Yescarta (well Kite’s really) has just been approved, hot the heels of Novartis’ Kymriah (prompting the FDA quote). So, not for the first time in pharma launches, it looks like we have ‘London bus syndrome’ in CAR T cell therapy. Spark Therapeutics has just had a positive recommendation from an FDA advisory panel for its ophthalmological gene therapy Luxturna – presaging a likely launch in January 2018.
As you can see by viewing just a small part of the Gene and Cell therapy webcast hyperlinked above the road to these successful launches has been a bumpy one. A few gene therapy products have been approved and launched prior to the recent arrivals but the main two, Glybera from UniCure and Strimveils from GSK, have failed to find a viable market. Their price tags, of €1m and €660k respectively, have been instrumental in this commercial failure but supply chain and patient management have also been challenging. The fact that both therapies are for rare diseases has also contributed to the poor uptake. Despite these issues the three patients who have been treated have been completely cured. And therein lies the paradox for gene/cell therapy – massive cost and massive hassle but spectacular, often curative, outcomes.
The two recently launch CAR T products are not only less expensive (a bit!) but have far bigger potential treatment populations (for diffuse b cell lymphoma and acute lymphoblastic leukaemia) than previous gene therapies. It is reasonable to expect therefore that they will not suffer the fate of Glybera and Strimvelis. It has been a rocky road however and headlines like this ensure the cost controversy is not going away.
In conclusion, and returning to one of the main themes from the webcast, plus the start point for this piece – I believe we absolutely are in the midst of a paradigm shift. To my view however this shift is not just a scientific or medical practice shift but a wholesale change in the value chain and commercial transaction between developers and medical providers. The many SMEs and their big pharma partners bringing gene therapies to patients need to start a dialogue on how to manage and prepare for this new paradigm, with all interested parties at the table. This dialogue needs to start with an open and honest discussion of novel pricing and reimbursement strategies and other issues around access. Newer gene therapies will be moving into therapy areas with far more patients than lymphoma and leukaemia so we need embrace the challenge and try to offset the controversy before it starts.
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