Cannabis for medicinal use in patients with rare diseases

Abstract

Patients with Rare Diseases (RDs) present with chronic and debilitating symptoms such as pain, anxiety and epileptic seizures. Symptoms can be unresponsive to conventional treatment and may lead to a decreased Quality of Life for patients. Cannabinoids have been reported to be efficacious against chronic pain refractory to conventional analgesics, anxiety and seizures. OBJECTIVE: Identification of RDs for which Medicinal Cannabis (MC) can be used and identification of issues related to RDs and perceptions on the use of MC in patients with RDs. METHODS: Study was divided into 2 phases. Phase 1: Literature Review to identify RDs in which cannabis or cannabinoids are used Phase 2: Development, validation and dissemination of 2 questionnaires for: (i) Health Care Professionals (HCPs) and (ii) RD patients. RESULTS: Cannabinoids were described as possible therapeutic agents in 20 RDs. The questionnaires were completed by 101 HCPs and 38 RD patients. Thirty-three HCPs had no experience on use of MC but would consider using it in their practice for management of RDs. Most patients (n = 29) did not have experience with use of MC and 20 patients would consider using MC to treat their condition or relieve symptoms of their disease. CONCLUSION: The study helps identify the potential of MC use in RDs associated with chronic symptoms such as pain, muscle spasticity, seizures and anxiety.