The technology, knowledge and capacity exist to dramatically improve Ribociclib clinical trial global access to CFCs, it is now a moral imperative for governments, payers and industry to rise to the challenge by improving market accessibility, reducing reimbursement barriers, and adopting market-based business solutions to achieve it. Recent experience with a Health Technology Assessment (HTA) in Sweden and advancement of HTAs and similar tools such

as Comparative Effectiveness Research in other countries underscores the importance of outcomes analysis to support the high cost of present day treatment practices. There is an on-going need for additional research, outcomes analysis and evidence. The Swedish HTA concluded, in part, that the scientific evidence is insufficient to determine if there are any differences in effects between different dosing strategies or to determine which dosing strategy, i.e., on-demand or prophylaxis, is the most cost-effective in treating haemophilia [45,46]. Readers should not interpret Selleck Proteasome inhibitor this to mean prophylaxis is not the appropriate clinical decision; rather it means that the level of graded evidence to assess cost-effectiveness is not always of the highest level and, perhaps out of necessity, is often based on best clinical practice. Given the well-documented

outcomes of current clinical practice, randomized controlled studies to obtain additional evidence would be considered unethical in many countries today. Thus, fresh approaches to confront such assessments and to advance care beyond current levels are required. Assessment of treatment interventions for rare diseases such as haemophilia should not be confined to traditional analysis. Ranking haemophilia related interventions with standard interventions of therapeutics and public health in Cost Utility Analysis comparisons is inappropriate. They should be assessed with new methodologies specific to the disease BCKDHB and take into consideration societal willingness to support people with rare diseases [47]. Given bleeding frequency is one of the most important outcome

measures, greater emphasis and understanding of concepts such as the cost savings for a bleed prevented need to be integrated into our analysis. Following from this and the Swedish HTA, a novel cost-utility model for the assessment of the cost-effectiveness of prophylaxis to treat haemophilia has been proposed taking into account other variables in the equation such as reductions in the incidence of inhibitors, co-morbidities other than joint bleeds, and quality of life [48]. Emerging therapeutic advances should not be justified or brought to market based only on the notion that they will be economically more affordable, although that may be the case, but rather more importantly that they will be therapeutically more advantageous.