Publication on Orphan Gene Therapy

The advent of orphan gene therapy products represents a new era in medicine development. The extraordinary high price requested for a single treatment in life without knowing the length of the therapeutic effects raises the question of how long society will be able and willing to finance these treatments. In their recently published paper (Frontiers | Development and Use of Gene Therapy Orphan Drugs—Ethical Needs for a Broader Cooperation Between the Pharmaceutical Industry and Society | Medicine (, members of the IFAPP Ethics Working Group recommend close cooperation between the pharmaceutical industry and society for sharing the scientific and financial burdens of the development of these medicinal products coupled with the abandonment of the premium price usually requested by the producers to compensate for the expected limited use of the drug by the small number of patients suffering from rare diseases.