By Javier Vega, Senior Director, IPG Health Consulting
IPG Health Consulting has been supporting the development and commercialization of cell and gene therapy (CGT) since its infancy; from working with academia on product development, to advising governments on national strategies, to working directly with pharma players spearheading commercialization efforts. Below we highlight some of the key dynamics we have observed over the past decade in this fast-paced roller-coaster of a category, from which we will subsequently publish deeper analyses focusing on various parts of the CGT ecosystem, its pain points, as well as the critical success factors. Most importantly, we will showcase successes and failures, leaders and laggers, and draw from these ambitious pioneers some lessons for others to learn, assimilate and adapt.
The road to success: Unlocking the potential for a brighter future in CGT
As an industry, we’ve watched the first CGT product entrants make scientific waves, seen industry player strategies converging into archetypes, and brought in new reimbursement models to cope with steep prices. Although a strong belief exists that CGT will provide a long-term solution to genetic diseases, does this innovation momentum across the industry have enough time to demonstrate commercially viable models that deliver successful ROIs and maintain investment to fully unlock its potential?
Looking back, IPG Health Consulting sees the potential of CGT hinging on a myriad of interconnected moving parts, but true success for this category can be condensed down to the following four key elements:
- Unlocking scientific possibilities of next-level CGT technologies: Broadening the repertoire of platforms and targets will give access to new reaches for CGT, outside traditional rare diseases where they seem to still be niched. Innovations across AAV vectors (e.g., capsid proteins, gene expression promoter engineering), variations of CAR-T (e.g., CAR-M, CAR-NK, TCR-T) and proof of concept of CRISPR technologies are all looking to significantly broaden the applicability of CGT.
- Optimizing the end-to-end manufacturing process: Players are now focusing their energy on moving beyond established in-house, batch, small-scale manufacturing into a world of larger processing, or even setting up specialist network sites for outsourcing to ultimately reduce turnaround times. The transition from autologous (patient-derived) to allogeneic (patient-agnostic) cell sourcing could even provide off-the-shelf treatment and remove the costly, burdensome and time-intensive part of the process.
- Establishing useable reimbursement models: Given the short-term clinical trial data in small, rare populations (often in single-arm trials), current players have had a tough challenge to convince healthcare systems to pay for these high-priced therapies. We now see several different models emerging from individual patient-data-dependent (e.g., SP, IT) to HTA-reassessment-dependent (e.g., DE, FR, UK), but there remains room for significant improvements.
- Building the CGT brand: Along with all the access hurdles described, once launched to the market, players must focus on the product uptake and overcome the complexities of patient and HCP experiences.
Demonstrating ROI for continued investment winners and losers will emerge Across Big Pharma, we have seen some interesting entry strategies into CGT. We have witnessed some "optimistic embracers" investing broadly with belief in the long-term despite the obvious hurdles, the "single committers" that are making targeted but monumental investments and the "strategic selectors" that are much pickier about which specific types of platforms they develop. Together with these are the biotechs, or "New Pharma," that are truly carrying the development risk. Here we see some "new kids on the block" coming up with innovations in contrast to the "established specialists" that bring years of legacy and partnerships.
Despite the flurry of investment activity in the last five to ten years, the "money momentum" started to wane over the course of 2021, likely driven by nervousness at the lack of ROI demonstrated by successful launches. The fate of the market climate may rest on the shoulders of current pioneers in this space’s ability to continue to push product uptake in current indications, successfully expand into new indications and pave the way to reduced market barriers for others to follow.
But who is doing it right? Who will succeed, and who will fail? What does it take to win? We will be delving into these critical areas in deeper analyses to expand on each, respectively.