RESUMO
BACKGROUND: The recent approval of innovative therapies for spinal muscular atrophy (SMA), such as nusinersen, has brought hope to patients and their families. OBJECTIVE: The aims of this study were to compare the characteristics and HRQoL of SMA patients treated with nusinersen and those treated with the standard of care. METHODS: This was a cross-sectional, interviewer-administered telephone questionnaire, which used a purposive sampling of SMA patients through a social support network. EuroQol five-dimensions-3-level (EQ-5D-3L) and the visual analog scale (VAS) have been used to assess the HRQoL. Different descriptive and inferential tests have been performed to compare the characteristics, EQ-5D responses, and mean scores of EQ-VAS between patients on nusinersen and the standard of care. RESULTS: Eleven out of 36 SMA patients (30.55%) have been treated with nusinersen. Patients with type I SMA represented 54% of those treated with nusinersen (P=0.012). Only 12.5% of SMA patients living in the Mecca region are treated with nusinersen in comparison to 50% of patients living in the Riyadh region (P=0.029). No difference was noticed in the proxy-responses for the five domains of the EQ-5D or the mean VAS scores for patients on nusinersen and the standard of care despite controlling for the SMA type and the ability to breathe independently (ß= 1.39, 95% CI= - 5.15-7.93, P=0.667). However, the mean VAS score for patients who are unable to breathe independently was significantly lower than their counterparts who are able to breathe independently even after controlling for the SMA type and nusinersen treatment (ß= -31.61, 95% CI= - 51.59 - -11.63, P=0.003). CONCLUSION: The results of this study highlight the uncertainty about the impact of nusinersen on SMA patients' HRQoL. Therefore, the impact of nusinersen on HRQoL should be examined using more robust study designs.
RESUMO
Drug shortages are a multifaceted problem that has been recurring in Saudi Arabia over the past decade with its significant negative impact on patient care. However, there is a dearth of evidence about possible domestic reasons, if any, behind this recurring problem. Recently, the Pharmacy Education Unit at King Saud University College of Pharmacy has called for a meeting with multiple stakeholders from academia, pharmaceutical care, pharmaceutical industry, purchasing and planning, and regulatory bodies to unveil the root domestic causes of the drug shortages in the Kingdom. Four major topics were used to guide the discussion in this meeting, including: current situation of drug shortages in Saudi Arabia, major factors contributing to drug shortages, challenges and obstacles to improve drug supply, and stakeholders' recommendations to manage drug shortages. The meeting was audio-recorded and transcribed into verbatim by five authors. The text was then reviewed and analyzed to identify different themes by the first and third authors. Multiple causes were identified and several recommendations were proposed. The main domestic causes of drug shortages that were explored in this study included poor medication supply chain management, lack of government regulation that mandates early notification of drug shortages, a government procurement policy that does not keep pace with the changes in the pharmaceutical market, low profit margins of some essential drugs, weak and ineffective law-violation penalties against pharmaceutical companies and licensed drug importers and distributors, and overdependence on drug imports. The participants have also proposed multiple recommendations to address drug shortages. Policy makers should consider these factors that contribute to drug shortages in Saudi Arabia as well as the recommendations when designing future initiatives and interventions to prevent drug shortages.
