The cellular and gene therapy development pipeline anticipates major transformations with hundreds of products moving forward toward approval. The 40 gene therapies projected to launch by the end of 2025¹ will shape the future treatment landscape for life-threatening disorders as well as common conditions. Navigating the evolving and complex healthcare data, policy, and regulatory requirements provides exciting opportunities and presents new challenges to patients’ access to treatment.
Patients who have or will receive human gene therapy products may need to be followed for up to 15 years to effectively evaluate potential long-term safety effects, due to the product design and how it modifies the host genome². With current gene therapies on the market targeted for rare and orphan diseases, and future approvals from regulators for more common conditions such as osteoarthritis and wet age-related macular degeneration, pressure will be on payers and policymakers to develop innovative payment models that can accommodate the associated high-costs.
Below are some important considerations for gene therapy manufacturers when approaching filing and commercialization.
Leverage an Integrated Registry Model
Biopharmaceutical companies are utilizing integrated registries, involving the assembly and integration of data from existing registries or other real-world data sources, as a means for assessing long-term risks and obtaining a deeper understanding of the natural history of the disease under study. Early in the planning phase of development, these data can also be considered for creating an external comparator group. Robust data collected on patients in real-world settings can be used to meet regulatory objectives, inform indication-specific experts, and support publications. A model for establishing a worldwide registry for patients treated with novel therapies was first seen when the Center for International Blood and Marrow Transplant Research (CIBMTR) outcomes database, originally established to track and share long term follow-up data from bone marrow transplant recipients, expanded to provide a mechanism for long term follow up of new commercial therapies.
Modernized Data Strategy
Organizations are appropriately looking to utilize real-world data to help bridge the evidence gaps for approval as well as to satisfy post-marketing requirements for gene therapies and other treatments. In designing and operationalizing these initiatives comes the challenge of ensuring data consistency, and overall quality and completeness. Aggregating and normalizing disparate data sets from various sources into one platform requires careful planning, a modern and compliant technology infrastructure, and strategic operational know-how.
Anticipate the Need to Provide Expanded Access
After clinical studies are complete, there is often considerable time before the treatment receives approval and before new patients can be treated with approved commercial therapy. An Expanded Access Program (EAP) gives patients access to therapies that are awaiting approval from regulatory bodies. In addition to the standard regulatory and operational considerations for launching EAPs, additional proactively planning is required for gene therapies given the logistical mechanism for determining patient eligibility and treatment administration.
Prepare to Offer Support Throughout the Treatment Journey
Pharmaceutical manufacturers must prepare to support the patient through the product initiation and treatment journey in new ways. The stakes are extremely high due to the potential cost of treatment and logistical requirements to move the product from manufacturing to the actual site of care and ensure that patients are treated as quickly as possible. Additionally, the patient may have to travel to obtain treatment at a trained facility. With the complexity of gene therapy, there must be alignment between multiple stakeholders related to collaborating and communicating for the sake of the patient’s treatment: the prescriber, the lab, the product manufacturer, third-party logistics, and the site of care. The need for a highly experienced hub team as part of a patient support program is vital to help guide the patient, family, and health care provider along the way.
Champion Payer Value, Analytics, and Outcomes
As several biopharmaceutical organizations and payers are implementing value-based contracts to demonstrate the value of their therapies, work does not stop after approval. Patient data and insights become more valuable as a tool for creating product differentiation. Patient support and hub providers should champion the ability to combine claims data and patient data collected from the hub to inform and meet the requirements of innovative payment models.
Looking forward, biopharmaceutical companies have a lot to keep in mind when approaching regulatory filing and launch of their gene therapies. By creating collaborative approaches to long-term safety and outcomes data for patients, manufacturers can help patients ensure they get access to these potentially life-changing therapies.
UBC has extensive experience in helping sponsors bring first-in-class, molecular entities to market. Our expertise spans epidemiology, safety, risk management, and clinical development, as well as the access and adherence challenges cell and gene patients face.
References
- https://business.caremark.com/insights/2021/gene-therapies-pipeline-highlight-variety-conditions-and-anticipated-treatments.html
- https://www.fda.gov/regulatory-information/search-fda-guidance-documents/long-term-follow-after-administration-human-gene-therapy-products
