When a patient gets access to a drug that changes the trajectory of their disease, it’s a beautiful thing. As a mother of a child with a rare disease, I’ve dedicated my career to connecting patients with emerging therapies. But there are so many barriers that stand in the way of patient access, especially in rare disease. Patients may be widely dispersed among countries with different regulatory processes, payer models and languages. HCPs need to be connected and to be supported in delivering new therapies. The raison d’être behind RareiTi is the development of a plan that navigates these barriers so that the pathway to access and commercialization is opened.
RareiTi deploys managed access programs as a key strategy to opening pathways. Managed access is a broad term for several regulatory routes that are used to provide access to unapproved therapies. It first came into broad use after the AIDS epidemic with people desperate to get life-saving therapies and now is (or should be) a consideration for any company developing a novel therapy. Managed access goes beyond moving an unlicensed drug from point A to point B backed by a strong program of pharmacovigilance. At RareiTi, we apply global expertise in designing and implementing managed access programs, and we tackle the complex tasks of engaging with regulators, payers, HCPs, patients and caregivers during enrollment and throughout the life of the program, including the transition to commercialization. Our proprietary technology provides sponsoring drug companies with anonymized Real World Data collected from patients on the program, while providing HCPs with information on adherence, allowing future clinical trials to be better targeted and opening the pathway to better outcomes.
The pathway to access for rare disease therapies can be difficult to navigate, filled as it is with regulatory, reimbursement, and logistics challenges. Having an experienced guide armed with the right tools can lead to a smoother road and better outcomes for drug developers, HCPs and patients.