Initiating and Defining a Sustainable Project on the Example of Rare Disease Therapy

Abstract

Purpose:The main aim of the article was to present the possibilities of improving the definition of orphan therapy implementation projects using the P5 GPM model in the context of reimbursement decisions. Design/Methodology/Approach: Theoretical research aimed at verifying the applicability of the P5 model in the practice of managing the implementation of orphan therapies. In the first part, the authors presented the essence of defining the project. Then, the background for the implementation of orphan therapies has been presented. Findings: The article presents the long-term impact relating to the limitation of the development of innovation and cooperation, as well as competences of the medical staff and the medical environment, exclusion of the patient and his family from the labour market, the increase in logistics costs related to attempts to reach the treatment, which has an impact on the natural environment. All of this results in a reduction in national welfare. The authors indicated that the use of the P5 model allows for a comprehensive analysis of the environment of orphan therapy implementation projects both in the short and long term, linking them with the goals of sustainable development, which in turn will ensure the correct definition of the project and making investment decisions. Practical Implications: Possibility to use the concept in the health technology assessment process for the purposes of the reimbursement decision, taking into account the sustainable development goals. The P5 model can be helpful in defining these types of projects. Originality/Value: A look at the logic of intervention in case of reimbursement of rare disease therapies from the perspective of the SDGs. Indication of the broad long-term impact of the project from the point of view of all its stakeholders. The considerations presented in the article constitute an introduction to empirical research, primarily in the form of casestudies.

Literature Review on Health Emigration in Rare Diseases—A Machine Learning Perspective

“…Many are under the auspices of EURORDIS (European Organization for Rare Diseases), which aims to increase the number of new therapies for rare diseases annually by 3 to 5 times by 2025 while lowering their cost by 3 to 5 times [397]. The UN, the WHO, and IRDiRC are moving in the same direction-rare diseases are an essential link in the chain of sustainable development [5,388,390,391]. A recent Rare 2030 trends study shows that increasing strains on healthcare budgets will force the emergence of new models of care delivery while inequalities in access to treatment and care continue to grow across Europe among people with rare diseases [389].…”

“…Moreover, since these drugs-being innovative and produced on a small scale, usually with orphan drug status-most often tend to be horrendously expensive, their financing is problematic for limited public budgets and unattainable for the patient alone. As a result, because rare diseases mainly affect children, families choose to emigrate, with all its consequences [5].…”