Exploring Facets of the Cell and Gene Therapy Market

Advancements in Cell and Gene Therapy  

Throughout 2023, there were seven new cell and gene therapies approved by the United States FDA, including the following:  

• CASGEVY for sickle cell disease (SCD) 
• ELEVIDYS for Duchenne muscular dystrophy (DMD) 
• LANTIDRA for type 1 diabetes  
• LYFGENIA for SCD 
• OMISIRGE for hematological malignancies  
• ROCTAVIAN for hemophilia A 
• VYJUVEK for dystrophic epidermolysis bullosa 

Among these approved therapies were many remarkable advancements, including the first CRISPR-based gene therapy for sickle cell disease.  

Acknowledging the critical role each of these treatments has and will continue to play in patient care, LifeSciencesIntelligence asked Klarer to identify the most significant advancements throughout the past year. 

“They're all significant,” she stressed. “This is just the beginning of overhauling how we approach disease treatment.” 

Although Klarer maintains that every advancement in the cell and gene therapy space is vital, as they impact different areas, she identified a few exciting developments. Previously, most cell and gene therapies were autologous cell-based therapies for blood-based cancers or adeno-associated viral vector (AAV)-based therapies for rare diseases; however, throughout the past year, more exploration has focused on other cell and gene therapy modalities.  

“There are a couple of new modalities that will increasingly become prevalent in the market,” she noted. “Ex vivo gene therapies have come out as well. It's again an exciting and new type of modality that [experts] expect to see more of in the near future.” 

Beyond that, Klarer expressed excitement over the growing research base surrounding cell and gene therapies for solid tumors. 

“[The industry is] getting our hands dirty in rare disease, blood-based cancers. But solid tumors will be another big frontier with much promise. It's just a bit more difficult to treat. So hopefully, we can make those types of strides in the next few years,” she added.  

Predictions  

As she spoke of the exciting changes expected in cell and gene therapies for solid tumors, Klarer continued to explore the anticipated expansion of cell and gene therapies that address underlying diseases.  

“There is so much there that can be addressed. Scientifically, there are reservations about what else comes from adjusting how the body works, but those types of trade-offs are part of the conversation in many disease areas that are yet to be addressed. And it's important to see how quickly we can push forward in some areas with such significant unmet need.” 

Cell and Gene Therapy Pricing  

Beyond the therapeutic value of new cell and gene therapies, Klarer discussed the financial component. As more cell and gene therapies emerge, the price tag continues to grow, posing questions about treatment affordability and whether the costs are justified.  

Necessary Costs  

“This might be a contradictory opinion, but there's been a lot of focus on the price of these products when they've been challenging to invent and make for many patients,” began Klarer.  

She noted that a lot of the cell and gene therapies that are being approved are manufactured on a case-by-case basis through extensive procedures. Besides the resources allocated to developing, testing, and validating these treatments, the cost per patient to manufacture the products is significant, requiring a sizeable investment to treat a small patient population.  

“To generate more funding in the field and the ability to propagate additional development, [the pricing] is necessary to continue for the near future,” noted Klarer. “Even raw materials for these products are quite expensive aside from the physical, manual manufacturing process and inability to produce the product at scale just yet.” 

Patient Care Costs 

From an insurance perspective, the cost of treatment is a critical access barrier; however, looking at the broader scope, it is not the only barrier to care. For patients, the process is lengthy and expensive, irrespective of the cost of treatment. Typically, a patient must see a specialist that offers that treatment. In most cases, that means traveling and staying near a treatment center for an extended time. During this time, patients undergo assessments, testing, confirmatory testing, and more before they even begin the treatment process. For autologous cell treatments, providers must collect and process cells, and the patient must return for infusion and monitoring.  

“The approach to treatment is quite different than other types of products where maybe [patients] can visit for a few hours every month or every two weeks or take a pill at home. Those make it a little bit more accessible as well. The patient journey associated with these products can be their own financial challenge, aside from insurance companies covering the cost of the product.” 

Personnel Expertise and Equipment 

“One of the other components of why these products have to be so expensive at this time is the amount of work it takes for a pharmaceutical company to activate hospitals to offer some of these products,” added Klarer.  

Companies spend time educating multiple departments across different specialties, including cell collection, product transportation, handling, patient monitoring, side effect mitigation, and more.  

They must have a comprehensive and robust commercialization infrastructure in place to get the products to the clinical sites. For smaller companies launching their first marketed asset, deploying their product across multiple clinical sites is difficult.  

“The number of sites that [companies] can activate sometimes is more limited in the beginning because it takes a lot of time [and resources] to set up all the contracts and get everybody trained and comfortable with a new type of product like this,” she noted.  

Beyond that, companies must consider the treatment's complexity and how that impacts deployment. All these factors play into pricing. Without an effective and inexpensive way to scale these treatments, the costs will remain high. 

Scaling and Manufacturing  

LifeSciencesIntelligence asked Klarer to expand on the difficulties associated with scaling and manufacturing.  

“[The cell and gene therapy field] just needs more talent in making products so we can help scale them,” she noted.  

As students enter the biology, chemistry, and engineering industries, many are unaware of the potential career paths in cell and gene therapy. However, with the proper education in these fields, the industry can recruit more talent to help with the scaling process.  

Beyond research and manufacturing, more medical training needs to be focused on cell and gene therapies. According to Klarer, access to cell and gene therapy is limited to providers with experience prescribing and administering these medications, which is a small subset of healthcare professionals. However, as more cell and gene therapies come to market, provider training must expand to accommodate the growing number of patients.  

“This is the infancy of the industry, and [healthcare] may get to a point where products are a lot easier to administer, receive, and store, or types of storage capabilities are expanded at hospital visits because it's worth allocating additional resources as [they] have an increased product count, or there are more allogeneic products available so they can stock up if there are large enough indications,” she stated hopefully.  

As the industry continues to evolve, healthcare professionals must monitor cell and gene therapy changes. Continued education and scientific exploration are critical to advancing healthcare.