What a manufacturer should consider when bringing a gene therapy to market

Complex, costly, revolutionary cures

There are a few certainties in launching a gene therapy. First, these therapies are revolutionizing the field of medicine by offering potentially curative treatments for rare, ultra-rare and complex diseases, many of which were once considered untreatable.

Second, the launch of any drug is a formidable challenge that demands considerable time, resources and logistical expertise; however, the complexity of gene therapies places added pressure on an already strained manufacturer’s capacity.

Last, this landscape is only intensifying as the number of therapies in development grows. More than 1,000 cell and gene therapies are currently in development, and 75 are expected to be approved in the U.S. by 2030

Key considerations for manufacturers when bringing a gene therapy to market

Tip #1: Think small for your optimal network size

In terms of a specialty pharmacy network, we say “think small.” It is important to consider how large of a network you want.

We recommend a limited, small, or exclusive distribution network with a skilled specialty pharmacy partner. You need a trusted collaborator to manage your complex therapy and deliver on its promises for long-term growth and overall success of your gene therapy. Compassionate patient care and experience with ordering and clearance processes should also be a consideration when selecting a specialty pharmacy. A large network may lead to confusion among prescribers through a varied experience. The therapy and logistics are complex, so it is best to place tight controls through an exclusive specialty pharmacy partner.

Tip #2: Plan early for launch

Start finding a specialty pharmacy partner as early as possible. Be prepared and do not wait until you are ready to launch your therapy or have received FDA-approval before engaging a specialty pharmacy. A longer lead time allows for customization to fit the needs of your therapy.

You should trust that your specialty pharmacy partner has the flexibility to collaborate with you to customize a model specific to the therapy you are launching. To ensure integrity of your product, consider a specialty pharmacy’s ultra-cold shipping capabilities, storage infrastructure, clean rooms and adaptable models that can accommodate both small and large populations. By planning early, you can thoroughly examine all aspects leading to a successful launch.

Tip #3: Consider specialty pharmacies as consultative partners

Your specialty pharmacy should be more than a vendor. Your specialty pharmacy should be a consultative partner that provides insights from prior experience, helping you see things with a distinct and diverse perspective.

A true specialty pharmacy partner will not just add value to your launch strategy, but also it will help provide insight into synergies for market growth. Specialty pharmacies should offer comprehensive management and distribution options, providing white-glove services and highly customizable solutions across multiple distribution channels. These options might include buy and bill, white bagging, and brown bagging. The specialty pharmacy partner you choose should recognize that your therapy is unique and provides individualized solutions to bring your gene therapy to market.

Accredo the right specialty pharmacy partner

In your search for the right specialty pharmacy partner, remember that experience is essential. Seasoned specialty pharmacies have proven best practices for gene therapy launches in collaboration with manufacturers.

Accredo has been supporting gene therapy launches since the FDA approved the first one in the U.S. in 2017. As of 2023, we have five gene therapy launches in our portfolio, with several more on the horizon. In short, we have the experience you need and are here to help you think beyond the clinical aspect associated with a pharmacy. We carefully consider the many complexities across the health care spectrum–for payers, patients, providers and manufacturers.

We have access to 22 exclusive network products, and over 80 limited small network products. Our relationship with CuraScript SD and their access to 30 exclusive distribution products–gives us even more experience and flexibility in serving the unique needs of manufacturers.

With nearly 80 percent of the FDA-approved specialty drugs in 2023 having limited distribution requirements, it’s hugely important that your partner can handle these unique limited and exclusive therapies every day.

If you want to learn more about how Accredo can enhance your gene therapy launch success, Contact Us and a specialist will reach out.