Gene therapy commercial challenges: Are we ready?
Gene and cell therapies
Nothing is more exciting in medicine today than cell and gene therapy (CGT). Not that long ago, it was considered science fiction that a patient’s own cells could actually be modified to fight disease. This is now possible and destined to radically alter a disease course. Being such a new therapy, there are a multitude of issues to grapple with. One is the cost, which was addressed in a previous article where we discussed“ one and done” therapies. Another unknown is on the supply side. In this post we examine whether the various stakeholders are adequately resourced to produce new gene therapies on a scale to meet market demand. This includes industry, hospitals, governments and contract manufactures. This question was posed to us by several companies that were trying to understand the landscape and the specific commercial challenges. There is a sense of urgency given the potential tidal wave of new gene therapies on the horizon.
According to the FDA there are currently 17 approved CGTs in the United States. The number of manufacturers of these products has ballooned from 69 in 2014 to 255 by 2018 according the Alliance for Regenerative Medicine (ARM). The number of investigational products as of 2018 was 657 in Phase I, 509 in Phase I/II, and 455 in Phase II. As for later development, 89 have reached Phase III, 32 are in Phase II/III, and 28 in Phase IV as of 2018.
How gene and cell therapies are different from standard pharma products
There are several factors that make cell and gene therapies unique. First, they offer the potential of actually curing the disease which will be incredibly disruptive to the industry. As previously noted, this has significant implication for the pricing model versus the annuity model currently in existence for treating chronic conditions. For example, we have heard discussions of charging for the treatment over a number of years for one-time administration given the level of costs. There are other considerations though such as the regulatory framework across multiple markets which is still evolving given the newness of this therapy. Even more important is that the enormous level of complexity in manufacturing these potential cures. A traditional pharma product is manufactured once and sold to potentially millions of patients. Alternatively, CTGs are specific to each patient and must undergo multiple iterations and logistical steps.
Case in point is the chimeric antigen receptor (CAR) T cell therapy, which uses a patient’s T cells (a type of immune system cell) to attack cancer cells. With CAR T therapies, a patient’s blood is drawn and T cells are removed. Then the gene for a special receptor that binds to a certain protein on the patient’s cancer cells is added in the laboratory. A large number of CAR T cells are grown in the laboratory and then infused back into the patient. The patient’s unique “living drug” then fights the cancer.
While pretty amazing, it can result in a logistical nightmare if not done correctly and thus a potential shortage of critically needed treatments. Processes on how to best understand and meet demand are still in their infancy. This is due to the time-consuming nature and complexity related to engineering CGTs. There are multiple aspects that will affect commercial planning: drug approvals, patient populations, technology, clinical acceptance and uptake, to name a few. Others include the capabilities and capacity afforded by such entities as academic centers, contract and government manufacturing.
Considerations for gene and cell therapy commercial planning
CGT has the potential to rapidly improve human health. However, a company needs to effectively plan for the commercial launch of a new gene cell therapy and effectively assess and meet the demand. Considerations to factor into the analysis include:
1) Capacity for the CGT industry to supply the expected demand.
2) How many resources will be competing for the same manufacturing technology.
3) The addressable patient population.
4) If a company should build in-house manufacturing capacity or license it out.
5) What role government will play in manufacturing capacity.
6) If Medicaid will enable gene therapies to be an option for indigent (will affect demand).
7) What key partnerships need to be defined now.
A basic question to ask is what happens if a given gene therapy can cure patients, will the industry be ready to meet that challenge? At this point there are more questions than answers. However, the use of thoughtful analysis will help the industry to determine the true demand and put in place the most effective processes to ensure the widest accessibility of these groundbreaking treatments.
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