ABSTRACT
INTRODUCTION: Free-of-charge (FoC) medicine schemes are increasingly available and allow access to investigational treatments outside clinical trials or in advance of licensing or NHS commissioning. METHODS: We retrospectively reviewed FoC medicine schemes evaluated between 2013 and 2019 by a single NHS trust and a regional drug and therapeutics committee (DTC). The details of each locally reviewed FoC scheme, and any nationally available Medicines and Healthcare products Regulatory Agency Early Access to Medicines Scheme (MHRA EAMS) in the same period, were recorded and categorised. RESULTS: Most FoC schemes (95%) allowed access to medicines intended to address an unmet clinical need. Over 7 years, 90% were company-FoC schemes and 10% were MHRA EAMS that were locally reviewed. Phase 3 clinical trial data were available for 44% of FoC schemes, 37% had phase 2 data and 19% were supported only by phase 1 data, retrospective observational studies or preclinical data. Utilisation of company-FoC schemes increased on average by 50% per year, while MHRA EAMS schemes showed little growth. CONCLUSION: Company-FoC medicine schemes are increasingly common. This may indicate a preference for pharmaceutical companies to independently co-ordinate schemes. Motivations for company-FoC schemes remain unclear and many provide access to treatments that are yet to be evaluated in appropriately conducted clinical trials, and whose efficacy and risk of harm remain uncertain. There is no standardisation of this practice and there is no regulatory oversight. Moreover, no standardised data collection framework is in place that could demonstrate the utility of such programmes in addressing unmet clinical need or to allow generation of further evidence.
Subject(s)
Delivery of Health Care , State Medicine , Humans , Retrospective StudiesABSTRACT
A 42-year-old man reported to our service with a 1-week history of vague cervical neck pain on a background history of a gelastic seizure disorder. Radiological imaging confirmed a type II hangman's fracture through the C2 pedicle. A CT angiogram of carotid and vertebral arteries was normal, and the patient was managed with an occipitocervical fusion. The patient had no neurological insult and was discharged on day 5 with a Miami-J collar for 6 weeks. Although there are case report evidence of lumbar and thoracic vertebral fractures secondary to seizures, this is the first report of a spine injury resulting from a gelastic seizure.
Subject(s)
Cervical Vertebrae , Epilepsies, Partial/complications , Neck Pain/diagnosis , Spinal Fractures , Spinal Fusion/methods , Adult , Cervical Vertebrae/diagnostic imaging , Cervical Vertebrae/injuries , Epilepsies, Partial/diagnosis , Humans , Male , Neck Pain/etiology , Neurologic Examination/methods , Spinal Fractures/diagnosis , Spinal Fractures/etiology , Spinal Fractures/physiopathology , Tomography, X-Ray Computed/methods , Treatment OutcomeABSTRACT
The current trend in hand surgery has streamlined the treatment of acute hand trauma to the modern-day surgery unit. As the volume of hand trauma caseloads continues to increase, it is becoming increasingly difficult to schedule patients for theater on the day of injury. It, therefore, becomes paramount to adequately triage patients in accordance with best clinical evidence and predictors of poor clinical outcomes. Animal models suggest that the earlier flexor tendons are repaired, the better the patient functional outcome. The largest study to date examining the timing of injury to functional post-operative outcome also recognizes that the faster these injuries are repaired, the better the patient outcome. Age-related changes to tendon biomechanics and structure are well-documented. However, no conclusive evidence exists specific to the degenerative changes and mechanical properties of flexor tendons in humans. The animal model strongly suggests that increasing age is associated with local architectural and biological changes that directly affect the tendon repair functional outcome. Although retrospective analyses to date suggest that smoking is a negative outcome predictor for functional tendon outcome, no prospective large-scale studies exist. A large, single-center prospective study specifically examining the positive and negative outcome predictors of flexor tendon repairs and functional post-operative outcome is warranted. The negative predictive model of patient care may enable us to further council patients preoperatively and stratify patients according to clinical need.
