Browsing by Subject "rare disease"

Orphan medicinal products (OMPs) are pharmaceuticals, that are utilized in the treatment of rare diseases. Rare diseases are diseases with a prevalence of at most five individuals out of 10 000. Clinical trials with statistically robust clinical data are challenging to conduct with rare diseases, since patient populations are small and the amount of trial subjects enrolling into these trials are usually scarce. Rare diseases also represent a variety of different diseases with divergent properties (5000-8000 identified). This presents challenges in health technology assessment (HTA) when reimbursements for these treatments are assessed and decided, especially when these treatments are usually considerably expensive and burdening to national health care systems. The main objectives for this study and master´s thesis was to research via interviews with experienced professionals from pharmaceutical industry and officials as how to define, monitor and assess the clinical effectiveness of OMP treatments, how to enhance their market access, and how to develop the current conditional reimbursement system in Finland. The interviewees (n=12) all represented from their respective backgrounds and introduced opinions from their own occupational positions and frameworks based on their professional experience. The study was executed as a qualitative study utilizing semi-structured interviews with predetermined questions and themes between 6th of April 2023 and 8th of June 2023. The interviewees were initially contacted via email and phone by one of this thesis supervisors from a professional pool of individuals identified using purposive sampling. The interview transcriptions were examined and analyzed using content analysis, and they were coded and grouped into themes. When inquired, the most common opinions regarding how to define and monitor clinical effectiveness of OMP treatments, the most common answers emphasized individual clinical assessment, real world data (RWD) collection, consideration of symptom control and overall quality of life, economic effectiveness, and clinical expert assessment. Market access of OMPs could be enhanced with more flexible and bold applications for negotiations and agreements, and a need for uniform, predictable MEA procedures, parameters for treatment discontinuation, outcomes-based models, and earlier proactive start for initial negotiations on behalf of the payer (society). The existing conditional reimbursement system might be developed with outcomes-based models, increased dialogue and trust between companies and officials within the realm of negotiations, uniform and predictable MEA procedures, and already established MEA negotiation frameworks to fast tract OMP market access. Development of the existing conditional reimbursement scheme, as well as objective to enhance market access environment in Finland could be accomplished by novel, flexible, patient specific, holistic and bold systems with an emphasis on systematic collection of RWD. Uniform and predictable MEA procedures with predetermined negotiation frameworks could bring value through faster market access and valuable predictability for pharmaceutical companies in their operations. Rapid market access of OMPs could be beneficial via clinical effectiveness of the treatments, as well as through collecting valuable clinical data from the medicinal products.