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Ophthalmology

Ophthalmology and the ophthalmic drug development landscape is changing. In recent years, we have seen several new medicines being developed or approved for ophthalmological conditions—particularly for patients with a significant unmet need.

This includes conditions such as:

  • geographic atrophy, and
  • age-related macular degeneration.

Rare ocular conditions and retinal diseases are also areas of unmet need being actively addressed by clinical trials, including:

  • retinitis pigmentosa, and
  • leber congenital amaurosis.

Improvements in the range of modalities are also noticeable in ophthalmic drug development; this is especially true for cell or gene therapies and biologics.

We have worked on several ophthalmological products—particularly cell and gene therapy products. Therefore, we understand how to engage with regulatory agencies when seeking advice and approval on the design of clinical trials for registration. For instance, we understand how to propose and justify the most appropriate endpoints to capture clinical benefits.

We have also developed and submitted a plethora of regulatory applications from early-stage clinical development through to approval, including orphan designation applications, paediatric investigation plans and marketing authorization applications.

Questions you may have about ophthalmology drug development

  • What are the requirements and important parameters of ophthalmic formulation?
  • How do controlled drug release mechanisms factor into ophthalmology drug development?
  • How do we demonstrate the comparative effectiveness of our ophthalmology product against existing treatments in clinical trials?
  • What are the regulatory requirements for conducting paediatric clinical trials in ophthalmology and how do they differ from adult trials?
  • How can we ensure safety and efficacy in our paediatric clinical trials, particularly in terms of administration via intravitreal injection?
  • What are the regulatory requirements and regulatory pathways for developing gene therapies for inherited retinal diseases or other ocular genetic disorders?
  • How do we mitigate environmental risk assessments and genetically modified organism applications specific to ophthalmic cell and gene therapies?
  • What evidence do we need to provide to highlight the economic value of our ophthalmology product in terms of reimbursement and market access specific to ophthalmology treatments?