Cell and gene therapy: The vital role of logistics
By 2030, the cell and gene therapy market is projected to be worth £76.03bn, with a substantial compound annual growth rate of 44%. This major growth not only represents a market opportunity but also a logistical challenge unlike any other in healthcare. With investments in the billions of dollars, how will the industry overcome the bottlenecks associated with transportation and regulatory compliance that could potentially threaten this potential?
Cell and gene therapies (CGT) offer hope to patients living with life-threatening conditions where limited treatment options may be available – and logistics is one of the most important aspects.
Treatments have the potential to significantly improve the lives of patients with a diverse range of diseases and conditions, from cancers and blood disorders to neurodegenerative and autoimmune conditions.
Some clinical trials have found that cell and gene therapies can be administered as a first-line treatment, without requiring any follow-up – even curing conditions entirely in certain cases. This could transform the current drug pricing model, shifting from regular doses over time to a single treatment.
However, the success or failure of treatments can all rest on logistics. Furthermore, the highly sensitive nature of materials and personalisation of treatments can require bespoke logistics solutions.
The projected growth of the cell and gene therapy market
GlobalData estimates that the cell and gene therapy market will achieve global sales of $76.03bn by 2030, up from $5.88bn in 2023, with a compound annual growth rate (CAGR) of 44% over the seven years.
Oncology already dominates the space, which is expected to continue to 2030 and account for 51.6% of the global market, valued at $39.3bn. Metabolic disorders will comprise an estimated 10.6% of the market at $8.1bn by 2030, followed by haematological disorders worth $5.4bn at 7.1% of the global sales total.
Within oncology, most use cases are for blood cancer, but there are promising trials underway targeting solid tumours. Other potential treatments are in development for haematology, neurodegenerative diseases – such as Alzheimer’s, Parkinson’s, and multiple scoliosis – as well as the autoimmune space.
The majority of the T-cell immunotherapy (TCI) landscape comprises chimeric antigen receptor T-cell (CAR-T) immunotherapy products, accounting for 73% of all active TCIs, according to GlobalData. In addition, the TCI market is projected for significant growth, rising from $13m in 2017 to $30bn in 2030, says GlobalData.
Autologous therapies make up 76% of CGT treatments in development, compared with allogeneic cell therapies at 24% in oncology.
However, there has been an increase in the development of allogeneic therapies for areas such as blood cancers. As such products would effectively be off-the-shelf treatments, this could transform patient access to therapies and even bring down prices.
Cost barriers to cell and gene therapy treatments
A major barrier to patients for treatments is the high costs involved. In Europe, the cost of CAR-T cell treatment varies by country, with the average ranging from $55,000 to $450,000. Prices in the US typically start from $370,000, with $100,000 in Israel, and $55,000 in China.
For gene therapy in the US, there are eight FDA-approved oncologic treatments, with prices ranging from $65,000 to $475,000. However, costs can soar even higher. The world’s most expensive drug is a gene therapy treatment for metachromatic leukodystrophy (MLD). Lenmeldy, or Libmeldy in other markets, is a one-time treatment with a reported cost of $4.25m.
With treatment costs so high, even the smallest logistical failure could cost not just time and money – but also lives. In the future of healthcare, speed and precision are of the essence.
Transportation of cell and gene therapy products
Logistical complexities for these types of advanced treatments require personalised processing and close coordination between healthcare providers and logistics teams.
As cell and gene therapies contain living materials, the end-to-end supply chain contains far greater complexities than standard pharmaceuticals. Given the sensitivity of cell and gene therapy products, the capacity to offer a variety of storage and transportation options is imperative to meet specific requirements.
Precision is required at every stage, along with coordination and clarity between different stakeholders. Stringent compliance is also necessary given the time-critical and personalised treatments. The first step is selecting a donor for the starting material, which is from the patient in autologous therapies. Cell collection occurs from a procedure such as apheresis or a biopsy.
To maintain the integrity of cells through transit and storage, materials may be frozen through cryopreservation to avoid degradation. In addition, strict controls must be in place to prevent contamination. Such logistics requires specialists in cold chain logistics, which need to maintain a full chain of custody and ensure traceability.
When a starting material arrives at the modification site, bioservices teams will need to handle the thawing and initial processing. In addition, documentation of the material is required, along with clearly defined next steps for modification and expansion.
To optimise timeframes and resources, expert consultants may oversee processes and ensure compliance with regulatory and GMP requirements.
Once the modification process is complete, the enhanced therapy is sent to the clinical site where the patient will be treated. Given the small window for delivery, the process must be strictly controlled – with any last-mile logistics considerations in place.
Once the therapy arrives at the treatment site, final quality checks are undertaken before preparation and then administration to the patient.
Precision logistics in biopharma
With the condition of the product and timing crucial for successful treatment, the precision of logistics is critical. Any delays can cause treatments to fail. Time is of the essence, as the patient may not always be able to wait for treatments. Furthermore, if a shipment does not reach the patient in time, it may be necessary to start the cell extraction process all over again. This can extend treatment timelines, raise costs, and put the patient at risk of their condition worsening.
A further complexity is in the regulations. There are compliance considerations around the difference between autologous and allogenic therapies regarding approvals and regulatory requirements.
As the industry adapts, the existing regulatory framework may not align cohesively with innovative treatments. Consequently, it is essential to be certain of regulatory requirements – especially if operating across borders.
One option for autologous therapies to navigate regulatory paths is via a compassionate basis. Allogenic therapies require more stringent regulatory scrutiny due to their potential to be mass-produced and distributed across multiple patient populations. Nevertheless, both present logistical challenges when operating across different regulatory environments.
The technologies enhancing logistics for the future of cell and gene therapy
When every second counts, tracking and real-time monitoring are vital throughout logistics to determine exactly where a shipment is. Technologies such as Internet of Things sensors can monitor shipments with precision, sending alerts when there are any deviations in temperature.
The deployment of artificial intelligence (AI) and machine learning can optimise transformation routes and avoid any obstacles. In addition, AI can run multiple scenarios and predict possible outcomes to stay ahead of the curve with unforeseen events such as roadworks and poor weather conditions.
On top of this, the use of blockchain can provide a robust and reliable audit of each stage of logistics to provide transparency throughout the supply chain. In addition, blockchain can deliver greater efficiencies in ensuring regulatory compliance and reducing human error.
Specialist services in cell and gene logistics
Transporting cell and gene therapies requires specialists in logistics. Oximio is an experienced provider of cold chain logistics with state-of-the-art facilities at its warehouses, a courier fleet, and experts in regulations across all major market regions. The company also works globally with trusted partners to ensure shipments arrive on time and in the necessary conditions.
A cell and gene therapy sponsor approached the company with precise end-to-end logistics support requirements. The biological materials were highly sensitive and required two precise temperature ranges. The first required conditions of -70°C for biosamples such as whole blood and tissues. The second needed temperature of approximately -196°C for an investigational medical product.
Drawing on extensive experience, Oximio developed a bespoke logistics plan with robust quality controls in place. Close collaboration with teams on the ground and stakeholders was essential.
For materials requiring ultra-low temperature storage, the company developed insulated containers filled with dry ice, along with real-time temperature monitoring to deliver continuous cold chain integrity.
The result was that all materials arrived in the necessary conditions, with the temperature successfully maintained and validated throughout. Operating across borders, all regulatory and safety standards were met.
With cell and gene therapy projected for major growth in the future of healthcare, specialists in logistics are pivotal to realising the potential for patients.
Cell and Gene Therapy Logistics from Oximio
For further information on how we can support your cell and gene therapy logistic requirements, please contact us. We’d welcome the opportunity to discuss your needs.