For traditional drugs, Time-to-Patient (TTP)—the time between when a patient is prescribed a treatment and when they are able to receive it from a pharmacist—can be measured in hours. In the gene therapy space, 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 as well—a benefit for companies entering into outcomes-based contracts with insurers.
Manufacturers can generate consistent TTP by developing standards for the burgeoning gene therapy industry. Reducing this time could require new models of distributed production, in which a product is produced in several sites across the US, or even at the site of care. As compared with scaling up, companies will need to consider how to “scale out.”
Companies also may need flexibility in their production capacity. Since some gene therapies are intended to cure conditions affecting relatively small populations of patients, companies should consider how to scale in a manner that is consistent with long-term sustainability, such as through contract manufacturing agreements.
As an increasing number of companies enter the gene therapy space, competition for contract manufacturing organization production capacity will increase, potentially raising costs or limiting its usefulness as an option.
The advanced therapies field is expected to grow rapidly over the next few years. Greater competition for existing manufacturing capacity likely will result in higher costs or scarcer supplies for customers, and companies may need to make investments into manufacturing technologies—or purchases of companies for their manufacturing expertise—to ensure they are able to compete or gain an edge over other players. For some traditional biopharmaceutical companies, investing in gene therapies is meant to ensure that a company is not missing out on the potential to cure a disease it now treats.
Another complexity may well be the stock return process, in which supply of a drug is returned from a distributor or provider to the manufacturer for a return of payment. Under such circumstances, companies could, for example, require the payer to cover the manufacturing costs of the product under certain circumstances beyond the biopharmaceutical companies’ control.
For citations, implications and insights, please read our full report, Gene therapies require advanced capabilities to succeed after approval.