DISCUSSION: Traditional clinical trial designs, validation, and evaluation methodologies used for nonorphan drugs often prove unsuitable for orphan drugs, given the small patient populations, sometimes fewer than 1000 cases. There is an increasing need for accessible therapies and both regulators as well as industry are trying to develop affordable and effective drugs to address this need. Despite several steps that have been taken, the timely development of drugs remains a challenge. One of the reasons behind the long development timeline is the recruitment, retention, and conduct of rare disease trials. To optimize the development timelines of orphan drugs in the EU, it is important to ensure that the safety and efficacy of the product is not compromised. Industry and regulatory agencies must implement innovative trial designs, devise flexible policies, and incorporate real-world data for assessing clinical outcomes.
CONCLUSION: Collaboration among academic institutions, pharmaceutical companies (both small and major), patient groups, and health authorities is crucial in overcoming obstacles related to clinical trials and providing assistance and creative ideas. The ultimate objective of granting rare disease patients timely and affordable access to medications with a positive balance between benefits and risks is to be met.
METHODS: Published literature, information from statistical bureaus, clinician surveys and extrapolation of selected data from the European Union were used to determine the socioeconomic costs of AR and urticaria.
RESULTS: Many patients in Asia suffer from perennial allergies and experience symptoms of AR and urticaria for up to 298 days per year. An estimate of the indirect costs of patients insufficiently treated for AR and urticaria amounts to USD 105.4 billion a year, which equates to USD 1,137-2,195 per patient due to absenteeism and presenteeism. Adherence to guideline-approved treatment can lead to estimated savings of up to USD 104 billion.
CONCLUSIONS: The current study suggests that within Asia, the socioeconomic impact of AR and urticaria is similar to that seen in the European Union in spite of the lower wages in Asia. This is due to the mainly perennial allergens prevailing in Asia, whereas the sensitization patterns observed in the European Union are dominated by seasonal exposure to pollen. These results underline the need for governmental initiatives to increase public awareness on the prevention and treatment of these and other allergic diseases as well as greater research funding and large-scale studies to reduce their growing socioeconomic burden in coming years.
HYPOTHESIS: The One-World Medicine concept attempts to merge the best of traditional medicine from developing countries and conventional Western medicine for the sake of patients around the globe.
STUDY DESIGN: Based on literature searches in several databases, a concept paper has been written. Legislative initiatives of the United Nations culminated in the Nagoya protocol aim to protect traditional knowledge and regulate benefit-sharing with indigenous communities. The European community adopted the Nagoya protocol, and the corresponding regulations will be implemented into national legislation among the member states. Despite pleasing progress, infrastructural problems of the health care systems in developing countries still remain. Current approaches to secure primary health care offer only fragmentary solutions at best. Conventional medicine from industrialized countries cannot be afforded by the impoverished population in the Third World. Confronted with exploding costs, even health systems in Western countries are endangered to burst. Complementary and alternative medicine (CAM) is popular among the general public in industrialized countries, although the efficacy is not sufficiently proven according to the standards of evidence-based medicine. CAM is often available without prescription as over-the-counter products with non-calculated risks concerning erroneous self-medication and safety/toxicity issues. The concept of integrative medicine attempts to combine holistic CAM approaches with evidence-based principles of conventional medicine.
CONCLUSION: To realize the concept of One-World Medicine, a number of standards have to be set to assure safety, efficacy and applicability of traditional medicine, e.g. sustainable production and quality control of herbal products, performance of placebo-controlled, double-blind, randomized clinical trials, phytovigilance, as well as education of health professionals and patients.