ABSTRACT
Presently, Gulf Cooperation Council countries are lagging in the generation of real-world data and use of real-world evidence for patient-centered care compared with the global average. In a collaborative effort, experts from multiple domains of the healthcare environment from the Gulf Cooperation Council countries came together to present their views and recommended key action points for the generation of robust real-world data and leveraging real-world evidence in the countries. The opinions of the experts are presented, along with existing barriers to the effective generation of real-world evidence in the countries. The Gulf Cooperation Council countries are undergoing transformative changes paving the way for improved healthcare measures; however, the challenges in generating reliable, robust, accessible, and secure real-world evidence are persistent. Hence, ongoing public-private engagements, as well as collaborations between regulators, policymakers, healthcare professionals, insurance and pharmaceutical companies, and patients, are warranted. A few notable examples of real-world evidence studies highlighting the benefits of real-world evidence for gaining valuable insights into patient-centric decision making are also discussed. The actionable steps identified for successful real-world evidence generation would provide long-term, real-world evidence-based patient-centric benefits for the countries.
ABSTRACT
The recent unprecedented emergence of the monkeypox (MPX) outbreak rang an alarm bell creating a burden on global health. The virus historically spread in endemic regions of sub-Saharan Africa by animal-human and human-human modes of transmission with an unknown modality. Currently, cases are occurring worldwide while risk factors, transmission, and outcomes of infection are still poorly defined. In addition, the likely genotypic mutation of MPX is an area of concern and requires further assessment. While licensed smallpox vaccinations are currently considered for early prevention after recent exposure, and antivirals are recommended for treating severe cases, there are no treatments available to treat MPX. This review provides general disease awareness and highlights recommendations for the containment of the virus.
ABSTRACT
BACKGROUND: Since the underlying cause of idiopathic short stature can indeed be undiagnosed mucopolysaccharidosis type I, it is critical to identify patients with mucopolysaccharidosis type I among screened patients with idiopathic short stature. OBJECTIVE: The primary objective of this study is to determine the prevalence of mucopolysaccharidosis type I disease in a high-risk group (ie, patients with idiopathic short stature). METHODS: We plan to perform a multicenter, cross-sectional screening study to primarily assess the prevalence of mucopolysaccharidosis type I disease in patients with idiopathic short stature. All eligible patients will be tested after obtaining written informed consent from their parents and guardians. Eligible patients will be recruited over 18 months from specialty care centers for pediatrics and genetics. RESULTS: This protocol was approved by the Institutional Review Board of King Fahd Medical City and funded by Sanofi Genzyme Saudi Arabia. We expect to collect data from ≥800 patients, as determined by our sample size calculation. CONCLUSIONS: Saudi Arabia is the largest country in the Arabian Peninsula; it has a population of more than 28 million people. To date, there are no reliable data regarding the incidence and prevalence of mucopolysaccharidosis type I in Saudi Arabia; therefore, future multicenter studies will be needed. Further, the prevalence of an attenuated form of mucopolysaccharidosis type I is largely underestimated in Saudi Arabia due to the absence of an effective newborn screening program. Therefore, the implementation of a nationwide newborn screening program is essential for the accurate estimation of the burden of mucopolysaccharidosis and the early diagnosis of patients. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): PRR1-10.2196/28619.
ABSTRACT
Severe acute respiratory syndrome coronavirus (SARS-CoV-2), a novel corona virus, causing COVID-19 with Flu-like symptoms is the first alarming pandemic of the third millennium. SARS-CoV-2 belongs to beta coronavirus as Middle East respiratory syndrome coronavirus (MERS-CoV). Pandemic COVID-19 owes devastating mortality and destructively exceptional consequences on Socio-Economics life around the world. Therefore, the current review is redirected to the scientific community to owe comprehensive visualization about SARS-CoV-2 to tackle the current pandemic. As systematically shown through the current review, it indexes unmet medical problem of COVID-19 in view of public health and vaccination discovery for the infectious SARS-CoV-2; it is currently under-investigational therapeutic protocols, and next possible vaccines. Furthermore, the review extensively reports the precautionary measures to achieve" COVID-19/Flatten the curve". It is concluded that vaccines formulation within exceptional no time in this pandemic is highly recommended, via following the same protocols of previous pandemics; MERS-CoV and SARS-CoV, and excluding some initial steps of vaccination development process.
