OBJECTIVES: OBC’s simple designs to date have limited applicability to cell and gene therapies and other treatments for rare disease. To keep pace with increasingly complex therapies, OBCs have begun to evolve including agreements with innovative design. We explored OBCs within rare diseases to identify how new methodologies and tech platforms could advance practice for cell and gene, and other rare disease, therapies.

METHODS: Publicly available information describing all rare disease-related OBCs, pipeline products, and commercialized treatments in the US and EU was collected. Analysis of marketed and pipeline products captured and assessed the complexities of each deal and quantified their market impact. Current VBA terms were analyzed to consider outcome measures, duration, data collection methods, and applicability to combination products or indication expansions.

RESULTS: In the US, 8%, or 6, of public OBCs are in the rare disease space, while the EU has a higher number at 31, or 19%. All rare disease OBCs within the US were executed post 2017 and were signed within a year of the drug’s launch. In the EU, such contracts have been executed since the early 2000s. In the EU, 77% of the OBCs are within rare oncological indications. Within the EU in 2018, 2 contracts were executed within stem cell & gene therapy, while in the US, 50% of the rare disease OBCs were within stem cell & gene therapy.

CONCLUSIONS: As personalized and gene therapies become more prevalent, OBCs will become an increasingly impactful tool for pricing and access if they evolve to capture the complexities inherent to these therapies. As deals evolve to address treatment, population, and supply chain nuances particular to rare diseases, creating deal standards to help companies better collaborate with payers, by leveraging technology platforms for data collection and sharing, will be critical to support this evolution and activate adoption.