What challenges does the advanced therapies sector face? 

Miami skyline

Over the three days of Advanced Therapies Week (ATW) 2024 in Miami, Florida, US, 16-19 January 2024, DDW’s Megan Thomas heard from attendees about the obstacles the sector needs to overcome to succeed.  

Viral vector manufacturing  

At the Advanced Therapies Awards ceremony, Dr Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy, was awarded a Lifetime Achievement Award in recognition of ‘extraordinary contributions to gene therapy’, a field he has helped shape through his pioneering work on lentiviral vectors. Lentivirus strategies are one of the most widely used tools in biomedical research and clinical testing, says Stella Vnook, CEO, Likarda. “Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said.  

Natalia Elizalde, Chief Business Development Officer, VIVEbiotech, says that Dr Naldini’s award represents the significant benefits and demonstrated safety of the therapies developed using lentiviral vectors that are being used for more and more clinical trials and more prevalent diseases. She said: “In order to make these therapies more accessible for a broader population, the manufacturing processes must be considering since the beginning cost-effectiveness to be able to treat a broader population and scalability up to commercial scales.” 

Pricing and approval of advanced therapies 

Philip Wills, PhD, Chief Commercial Officer, Catalent Cell & Gene Therapy, says that it is already clear that pricing of advanced therapies requires innovative thinking. He said: “The industry, as a whole, needs to carefully consider the true value of curing lifelong genetic or degenerative diseases, particularly when evaluating novel therapeutics that present unique treatment opportunities.”

Joel Eichmann, Co-Founder and Managing Director, Green Elephant Biotech, agrees that prices need to come down significantly for CGT to be widely available. He said: “Increasing the level of automation and standardisation is not only the way to reduce manufacturing costs, but also paves the way for follow-on approvals once a platform technology is part of an approval. To achieve this, we need greater collaboration between academia, therapy developers and manufacturing equipment developers.” 

Stella Vnook , CEO, Likarda, notes that as more regenerative therapy options come to the market, patients will demand access to the treatments, often using the court system. She said: “Payment options will be devised to absorb the costs. One option is that manufacturers will need to provide a product guarantee or refund the cost in a pay-for-performance model. This possibility is a slippery slope that will be more costly for the manufacturer both in the refunds when the desired outcomes are not achieved and in litigation costs over whether the product claims were met or not.” 

According to Matthew Hewitt, PhD, VP, Technical Officer Cell & Gene Therapy & Biologics, Charles River, the FDA may approve 10 or more CGT products in 2024 following eight US approvals in 2023. He said: “This is something to celebrate as patients will have additional therapeutic options available to them, in some cases the therapies represent the first therapeutic option for patients. Pricing and reimbursement continues to be a challenge we address as a field. I don’t think anyone disagrees that we need to bring down pricing to increase patient adoption and take strain off healthcare reimbursement systems. While there are multiple methods to address these challenges such as increased automation, optimised cell lines, and downstream processing methods to name a few, it will be an iterative process to collectively address challenges. However, I believe we should focus foremost on getting approved therapies to the market so patients have options.” 

Heather Purvis, Director of Clinical Operations, Title21 Health Solutions, said that as advanced therapies and approaches to patient care continue to evolve, the need for standardised and turnkey approaches grows. She said: “The emerging therapies of recent years, and the near future, have been long sought-after as breakthrough treatments for otherwise incurable diseases, which benefit millions globally yet are only attainable by a handful, in part because the onus of the dollar ultimately lands on the shoulders of the patient. Even as more therapies become commercially available, costs remain high and insurer knowledge needs to keep pace. Better, more intentional data and forums for cross-functional knowledge building across sectors are the best opportunities to increase price transparency, pace to market, and ultimately, access to therapies for patients.” 

Josh Ludwig, Global Director, Commercial Operations, ScaleReady, adds: “The recent approvals in the CGT field are a testament to the innovation and progress we’re witnessing.” 

Manufacturing, development and regulation 

Priya Baraniak, Chief Business Officer, OrganaBio, says that CGTs offer “revolutionary” treatment options, but their high cost presents a major hurdle to wider accessibility. She said that several key challenges contribute to this issue, which include:


  • Scalability: Current processes often involve manual labor and small-batch production, driving up costs. Automating and scaling up manufacturing is crucial for cost reduction. 
  • High-cost materials: Specialised equipment, reagents, and cell culture media significantly contribute to the overall cost. Developing more efficient and affordable alternatives is key. 
  • Vector production: Viral vectors used in some CGTs are expensive to produce due to complex manufacturing processes and limited yields. Optimising these processes and exploring alternative delivery methods can bring down costs. 

Development and Regulatory: 

  • Clinical trials: Long and complex clinical trials with stringent regulatory requirements are necessary for safety and efficacy evaluation, but they also contribute significantly to the overall cost. Streamlining trial designs and regulatory pathways can help. 
  • Reimbursement models: Current healthcare systems may not have appropriate reimbursement models for the one-time, high-cost nature of CGTs. Developing value-based payment models that consider long-term benefits could improve access. 
  • Intellectual property: High patent royalties and licensing fees for key technologies can inflate treatment costs. Fostering open innovation and collaboration could help address this issue. 

Other Challenges: 

  • Limited insurance coverage: Many insurance companies are hesitant to cover CGTs due to their high price tags and limited long-term data. Increased clinical data and evidence of cost-effectiveness can improve coverage. 
  • Patient access and equity: High costs disproportionately impact access for low-income patients, creating ethical and equity concerns. Developing financial assistance programs and exploring alternative funding models are important. 

Baraniak concludes that addressing these challenges requires a multi-pronged approach involving collaboration between researchers, manufacturers, policymakers, healthcare providers, and patient advocacy groups. She said: “By optimising manufacturing processes, streamlining development and regulatory pathways, and developing innovative pricing and reimbursement models, we can bring these life-changing therapies within reach of more patients.” 

Marlin Frechette, Chief Quality & Compliance Officer (CQO) at FUJIFILM Irvine Scientific, said: “The CGT sector is at an inflection point today – a changing regulatory environment, new drug therapies, and new patient needs. As industry leaders, we have a shared purpose to collaborate and find solutions for today’s demands; this conference provides the platform to do just that. Because we share best practices on how to meet regulatory challenges head on and accelerate more therapies with standard CGT workflows for clinical trials or scale-up demands and more, together with our customers we are able to move the industry forward to the next level of patient access.” 

Dr Edwin Stone, CEO at Cellular Origins, said: “Cell therapies are transformative but also complex given all of the different stakeholders needed to deliver. From biology, through manufacture, supply chain, reimbursement, ATW brings all these voices which is vital as we grow this industry and get therapies to more patients.” 

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