Modern medicine is stepping into an exciting and promising renaissance with a wave of small-batch cell, gene and curative therapies receiving regulatory approvals. Sixteen months ago, it was Luxturna, an eye disease treatment from Spark Therapeutics. A few weeks ago, Novartis announced the world’s most costly treatment to date: Zolgensma for Spinal Muscular Atrophy. Then Bluebird Bio received the European regulatory green light for a beta-thalassemia therapy targeting a fatal hemoglobin disorder.
All three of these instances herald a departure from the approaches of pharma’s yesterday as targeted therapies continue the march toward personalized medicine. They are also driving significant changes in drug development and manufacturing with regards to both methods and costs. Historically, pharma has focused on producing large volumes of drugs for large populations of patients. With small-batch therapies, this changes. For example, Zolgensma targets a condition that affects approximately 400 kids a year in the U.S. Extrapolate the numbers out and the price for the drug makes sense ($2.125 million); it’s limited by its potential application. On the other hand, examine a more familiar drug, like cholesterol drug, and find an almost unlimited user base.
This modus operandi of drug development and manufacturing has been able to withstand lower efficiencies since costs have been absorbed by the higher margins available in producing drugs for mass consumption, built to function on a system of chronic treatments. But how will the ramifications of drug products produced to target and cure smaller populations of patients – or even just a few patients in the most extreme cases – change the current paradigm of drug manufacturing?
Leaning in to a Targeted Scale
The recent news of the Zolgensma approval represents a significant advancement for the promise of curative therapies based on gene therapy. It demonstrates that the science and technology underlying these products holds tremendous potential for patient outcomes.
A smaller, targeted scale brings new challenges since manufacturing processes for customized platforms are significantly more complicated. Lower volumes of production will force biotech companies to become more efficient in the development of their manufacturing processes. Adoption of the latest technology solutions to manage process development and utilization large amounts of data will drive unprecedented efficiencies while ensuring high product quality.
Cost of a Cure
For so long, treatments have been approached from a chronic perspective with an associated annual cost of a drug. Now, gene therapies are gearing up to cure disease with one to two doses. Already a debate rages debating the pricing of these solutions. How should a cure be priced relative to a chronic treatment?
The insurance industry is currently built to match outgoing cash flows (payments) to incoming cash flows (premiums and returns on invested capital). This structure matches the system based on chronic treatments, where the total costs are spread out over many years. However, the imbalances created by large lump-sum payments for cures as projected with Zolgensma is likely to force changes to the underlying structure of the insurance system. How this will play out and the level of disruption to the industry is yet to unfold.
Transforming the Pharma Life Cycle Management
It’s an exciting environment and a great time to be at the forefront of cutting edge changes in pharma and manufacturing. With the current political climate and concerns about overall health care costs taking center stage in the national discourse, the industry can expect pricing pressures in the future. To answer this challenge, pharma companies need to adopt new technologies to drive higher efficiencies and smarter manufacturing which can help the industry preserve margins while ensuring high product quality. Knowledge management around design, risk, control, and production during the manufacturing lifecycle is essential to driving these efficiencies, and QbDVision plays a central role in these activities for our customers.