Abstract
Universal access to effective, safe and affordable medicines is the ultimate goal of health systems worldwide. The rapid evolution of medical science in previous decades has led to significant improvement in the diagnosis and treatment of diseases, including those that are rare and/or life threatening. A more rigorous approach in drug approval processes, triggered initially by the thalidomide tragedy in the late fifties, has raised the safety profile of new drugs in order for them to be on the market. The availability of effective drugs widely used in the treatment of diseases with high prevalence such as hypertension, high cholesterol and ischemic heart disease has improved the life expectancy and quality of life in very large populations of patients. The introduction of innovative drugs, mainly in oncology and virology, albeit raising questions about the sustainability of health systems, is leading to tangible improvements in the prognosis of previously incurable diseases.
Yet it is estimated that three quarters of the world's population, an estimated 5.5bn people, have low or non-existent access to medicines. Furthermore, the regulatory process for innovative drugs is becoming so cumbersome that practising clinicians are often facing the need to identify unlicensed alternatives when approved drugs fail or are unavailable, a problem that is particularly relevant for patients with rare or ultra-rare diseases. This in itself, together with greater patient knowledge, has triggered the search by patients for cheaper alternatives to expensive or sometimes unaffordable drugs which, coupled with the availability of counterfeit and falsified drugs over the internet, is becoming a serious threat to patient safety. Finally, drug shortage, due to a variety of causes, including manufacturing problems, parallel export, and lack of economic value for pharma companies, may affect the availability of fully approved and effective drugs.
The high and ever increasing global unmet need for access to medicines, together with the growing challenges and complexity involved, requires a coordinated presentation and discussion of this very relevant subject. For this reason, we are launching a new journal specifically focused on these topics. Medicine Access @ Point of Care is an open access, multi-disciplinary, peer-reviewed publication dealing with access to medicine in the broadest sense, including expanded access/compassionate use of medicines, global access, early access, treatment of rare diseases, and any situation where an unmet clinical need requires treatment which is not, or not yet, licensed or available. The journal also deals with regulatory compliance, counterfeit and falsified medicines, shortage of medicines and responding to patient demand outside of traditional access routes, regardless of where in the world the patient is. It is our aim to publish reviews, point of views, position papers, research articles and case studies. For these case studies, the focus will be not on the clinical case itself, but the challenges faced when sourcing unlicensed, unavailable or hard-to-access medicines, and how the issue of access to treatment was solved. The name Medicine Access @ Point of Care has been chosen to emphasize the journal focus on medicine access, and on the implications that this has where it really matters, i.e. at the point of care, with its direct impact on patients receiving the most appropriate and safe treatment available, exploring licensed and (when needed) unlicensed options.
We do expect hospital pharmacists, hospital specialists, drug safety experts, market access experts, decision makers, payers and patients' associations to be particularly interested in the Journal, but we hope that the content will reach the broadest possible audience, given the critical importance of its content for all health systems. The Editorial Board of the Journal, including several prominent researchers, clinicians, pharmacists, and regulatory authorities active in the field, will grow with the Journal and will oversee the review of the manuscripts, whilst steering its development.
We hope that Medicine Access @ Point of Care will be able to attract valuable content for its readers worldwide and will contribute to a better knowledge both within and outside the scientific community of a timely, effective and safe access to medicines.