Patient expectations and regulatory needs in the risk-based drug development of cystic fibrosis

Abstract

Aims

Cystic Fibrosis (CF) is a rare, chronic, progressive, multi-organ disease. It needs life-long, complex therapy with many difficulties for patients to follow. New therapy development also has difficulties owing to regulatory and economic challenges.

Methods

This study evaluates the intervention possibilities of therapy management in CF, taking into consideration the aspects of patient adherence and Quality of Life (QoL), regulatory and economic backgrounds. It presents a Quality by Design (QbD)-based knowledge and Risk Assessment (RA)-based evaluation method.

Results

The RA-based evaluation showed that the most highly critical intervention points in the CF therapy-management with greatest positive estimated impact are: increasing of the social support, increasing of the level of patient education and improvement of the potentials of therapy monitoring. The degree of adherence in CF depends mainly on the degree of progression and the complexity of the therapy. New drug combination and new medicine development were found as they have lower critical effect of the patient’s QoL.

Conclusion

The risk-based exploration of the therapy interventions can help in finding the factors which have the highest effect on the therapy success and patient adherence. They can be the alternative of a time and cost consuming new medicinal research and development.