3 Essential Capabilities to Develop Gene Therapies

Revenue opportunities for health systems and hospitals include manufacturing gene therapies on their own or in partnership with biotechnology companies.

After biotechnology companies or healthcare organizations receive approval for gene therapies, there are three key capabilities to bring the therapies to market, a new PwC Health Research Institute report says.

Gene therapies modify patient genes or cells to treat or cure disease. Over the next decade, the number of patients who will have received gene therapies is expected to reach 500,000.

Health systems and hospitals are playing a crucial role in the development of gene therapies, including autologous therapies that take cells from patients that are manipulated then reintroduced to the body, the PwC Health Research Institute report says.

"In contrast to traditional manufacturing, clinicians and health systems are often an essential part of gene therapy production. For, autologous therapies they participate in the first step of the production process—collecting the cells—and the last one—administering the altered gene therapy product. They are, in essence, extensions of the biopharmaceutical company."

Treatment facilities are pivotal in ensuring patient safety and maintaining quality, the report says. "Companies may need to expand their views of the 'production facility' to include treatment centers, since improper collection, handling or administration during the process could put patient safety at risk and undermine quality. Already, some organizations like the Foundation for the Accreditation of Cellular Therapy are working to set standards for these organizations to ensure consistency of quality standards."

After a gene therapy has received regulatory approval, the three crucial capabilities for biotechnology companies and their healthcare organization partners are advanced manufacturing, responsive supply chains, and tailored commercialization and reimbursement models.

1. Advanced manufacturing
 

Unlike traditional medications, most gene therapies are personalized treatments designed for an individual patient or manufactured in small batches, the PwC report says.

"Traditional medical products are made for many patients to take, with differences in doses, release mechanisms, or coatings allowing a regimen to be more personalized to the patients' needs. Manufacturers of these products have long relied on post-approval scale-up activities to quickly meet market demand by producing millions, and even billions, of doses of product per year."

For gene therapies, the inability to manufacture at large scale has three implications for biotechnology companies and their healthcare organization partners:

• Competition for manufacturing capacity is expected to make acquisitions and partnerships attractive options.
   
• Training staff will be essential to avoid production bottlenecks. "Due to the novel techniques and technologies used in manufacturing gene therapy products—and the small number of approved gene therapy products—few prospective employees have ready-to-hire experience in gene therapy," the PwC report says.
   
• Manufacturers will have to focus on time-to-patient (TTP), which is the amount of time between when a treatment is prescribed and when it is received by a patient. Unlike traditional treatments, which often have TTP measured in hours, TTP timelines for gene therapies can be lengthy, the report says. "TTP can be weeks after accounting for doctor's visits, insurance approvals, manufacturing, and the treatment's time in transit. Decreasing this time will help increase patient and provider satisfaction, and potentially lead to better outcomes." Solutions include having multiple manufacturing sites across the country or manufacturing at the site of care.

2. Responsive supply chain
 

"Gene therapy companies often must rely on a robust supply chain with advanced capabilities, from collection of the cells from a patient to administration of the treatment. Key among these capabilities is a 'cold chain' ensuring products are stored at the right temperature and handled properly from manufacturer to patient or vein-to-vein. A single temperature failure in the supply chain could render the product useless, even dangerous," the PwC report says.

There are two implications from the need for a responsive supply chain:

• Gene therapy manufacturers should consider personalized engagement of patients similar to apps offered by companies such as Domino's Pizza. "Gene therapy companies could borrow this approach, showing patients where their cells are, how far along they are in the production process, the status of delivery, when they need to prepare for treatment, and more. Companies also could include educational, payment information and other support tools in these applications," the report says.
   
• Contracting with distributors and payers should account for the possibility of returned products. "Companies should consider what the 'return' process would look like in practice and how they might structure contracts with payers to account for this possibility. Companies could, for example, require the payer to cover the manufacturing costs of the product under certain circumstances beyond the biopharmaceutical companies' control," the report says.

3: Tailored commercialization and reimbursement models
 

Gene therapies can have high costs because they are not produced at large scale, so reimbursement models should be crafted to assure payers and patients that the therapies have value.

There are already examples of innovative pricing models, the PwC report says. "AveXis Inc., a subsidiary of Novartis Pharmaceuticals Corp., is offering payers a pay-over-time option for its new gene therapy treatment for spinal muscular atrophy in pediatric patients, a genetic disorder that causes muscles to atrophy. Under the plan, insurers would have up to five years to pay for the one-time therapy."

Opportunity for health systems and hospitals
 

Health systems and hospitals can generate revenue opportunities from gene therapy manufacturing, Karen Young, pharmaceutical and life sciences leader at PwC, told HealthLeaders.

"As gene therapy develops into the commercial space, health systems and hospitals may start to see gene therapy as a greater revenue opportunity. Some hospitals already discover, test, and administer gene therapies, which are key aspects to the overall process. Already, some academic medical centers have started investing in gene therapy manufacturing facilities and regulatory capacities, which could help them to control all aspects of the process. Even providers who aren't interested in making or developing gene therapies may wish to partner with companies that do, which could allow them to share in the costs—and rewards—of this space."