Joe Cappello, Senior Vice President of Global Specialty & Branded Product Strategy & Access, AmerisourceBergen

In 2018, the FDA approved a record number of drugs that treat rare diseases.

 These medications can help extend the lives and alleviate pain and other medical concerns of many patients living with a rare or orphan disease. However, as the FDA continues to increase the number of specialty therapies it reviews and approves, manufacturers must ensure that patients have access to these medicines.

Manufacturers must consider the various interdependencies that will be required, such as the most convenient point of care for patients, while working with stakeholders to keep costs at an affordable rate. Therapy owners should look for a partner that can offer them the integrated support required to bring these complex medicines to market and with the access patients need to improve their lives.

Time and resources are among the most important yet often limited considerations that manufacturers must focus on throughout the commercialization process. Given the interdependencies, manufacturers should seek support that allows them to focus on what they do best—develop novel therapies that improve patient lives. Collaborating with an expert that has a proven history of navigating the commercialization process will ensure a manufacturer does not miss any steps that might prohibit patient access.

A trusted partner such as a distributor can offer a variety of best practices, from reaching the small patient populations who may need a treatment option to providing a stakeholder education strategy to inform reimbursement decisions for rare disease states. Distributors uniquely offer the flexibility to comprehensively address needs at all stages of the therapy life cycle with a single point of contact who makes accessing different services easier.

Although it is never too late in the commercialization process for manufacturers to engage an expert, earlier collaboration will best position a therapy for market success. A partnership that begins 10 to 12 months prior to the anticipated product approval can add the greatest value to manufacturers. For specialty manufacturers that may not have worked with collaborators in the past, this article outlines the instances leading up to the approval date in which partners can offer vital assistance.

Given that many specialty medicines treat patient populations in the thousands rather than the hundreds of thousands, access will be instrumental to the performance of a therapy. If a product does not make it into the hands of the patients who need it most, its efficacy is irrelevant. Hand in hand with the logistics strategy, a well-planned distribution strategy in the early stages of the product life cycle can make a significant difference in its viability. Most important to this strategy is an understanding of where a patient receives the therapy so that those providers are considered key stakeholders throughout the commercialization process. Distribution experts can work with stakeholders to ensure they have the capability to manage the inventory of these products and dispense the right drug to the right patient at the right time, which is a requirement for rare and orphan products.

To secure the viability of many products that treat rare diseases, temperature sensitivity is of the utmost importance, which is why manufacturers must have an established logistics program. Whether a therapy will reach patients in the United States or across the globe, manufacturers should maintain flexibility to support their entire network, providing end-to-end visibility of a product.

For example, a logistics strategy is critical for cell therapies that require a high-touch and a centralized, integrated model to navigate the patient, provider, and manufacturer through a very complex routine in which 1 missed appointment, 1 missed shipment, and 1 missed treatment can have significant consequences for the patient. Beginning this work now will allow a logistics manager to investigate various solutions that can increase supply chain efficiency, maximize a manufacturer’s return on investment, and improve the care of the patients the manufacturer serves.

The complexity of specialty therapies requires a manufacturer to have a strong understanding of how various stakeholders understand and value a product. Distributors work across the health care continuum and can offer insights through their daily interaction with these parties. Payer insights are critical to understanding their willingness to reimburse certain products and to developing a reimbursement plan that provides patients with the greatest access to the therapy.

Insights in the form of research and data on the patient population will also be important to collect. Understanding that smaller patient populations are being served, partners can use their expertise to understand their patient journey, informing the development of patient assistance programs and therapy delivery. Regularly monitoring the full reimbursement, competitive, regulatory, and legislative landscape will provide manufacturers with the latest information to consider as they approach their launch date.

At Least 9 to 6 Months From Approval Date 

Rare and specialty products can be increasingly expensive; however, when costs increase, manufacturers need to take steps to also increase access so that no patient is left behind. Experts can work with a manufacturer to develop a custom support service that can interact directly with patients to address access barriers and support them as they navigate their health. Another factor that emphasizes the need for a patient support service is the therapy itself. The therapy may require special handling, could be complicated to take, or might have serious adverse effects that require additional support for the patients who take the medicine. The right partner can help a manufacturer map the patient journey, build a program that supports patients and propels product success, and determine the right distribution strategy to establish appropriate access.

A partner can provide value to a manufacturer, whether that partner is engaged 12 months or even just a few months prior to a product launch. As a manufacturer approaches its approval date, a distributor can work across the supply chain to ensure programs such as logistics, inventory management, payer and provider engagement, reimbursement strategy, and patient support services are integrated, operating efficiently, and ready to initiate. Support during this time can be invaluable as manufacturers work to final- ize remaining details and communicate with their stakeholders. Knowing that a similar-minded contact with expertise in launching specialty products is working with a manufacturer’s goals in mind can ease uncertainty and empower the manufacturer in these final months.

Although bringing a therapy for a rare or orphan disease to market can be complex, it is critically important to patients who may not have had another treatment option in the past. For many first-time manufacturers, navigating the commercialization process can seem daunting. However, engaging with an expert with an array of integrated offerings and experience in bringing these therapies to market can make all the difference. A strong partnership can support the successful commercialization of a product, position the therapy to meet a manufacturer’s business goals, and provide patients with an option to improve their health and quality of life.

1. Maynard J, Furia-Helms A. FDA is working to bridge gaps and meet needs for rare disease product development. FDA website. www.fda.gov/NewsEvents/Newsroom/FDAVoices/ucm632217.htm. Updated February 19, 2019.

Articles

A strong partnership can support the successful commercialization of a product, position the therapy to meet a manufacturerâ€™s business goals, and provide patients with an option to improve their health and quality of life.