Initiating and defining a sustainable project on the example of rare disease therapy

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 case-studies.