Development and Use of Gene Therapy Orphan Drugs—Ethical Needs for a Broader Cooperation Between the Pharmaceutical Industry and Society

Frontiers in Medicine

article

Sandor Kerpel-Fronius¹  Kotone Matsuyama²  Shehla Naseem³  Johanna Schenk⁴  Honorio Silva⁵  Varvara Baroutsou⁶  Sander Becker⁷  Roberto Carlesi⁸  Luis Collia⁹  Brigitte Franke-Bray^1,10  Peter Kleist^1,11  Chieko Kurihara^1,12  Luis Filipe Laranjeira^1,13 

[1] Department of Pharmacology and Pharmacotherapy, Semmelweis University;Nippon Medical School;Ferozsons Laboratories Ltd.;PPH Plus GmbH & Co. KG;United States;Independent Medical Consultant & Pharmaceutical Medicine Consultant;Consultants in Pharmaceutical Medicine;Independent Researcher;Craveri Pharma;Independent Consultant;Cantonal Ethics Committee;Quality Assurance and Audit Office, Quantum Medical Science Directorate, National Institutes for Quantum and Radiological Science and Technology;Eli Lilly & Co.

关键词: gene therapy;    rare diseases;    orphan drugs;    ethics;    accelerated approval;    health care;    spinal muscular atrophy;    drug pricing;   

DOI  :  10.3389/fmed.2020.608249

学科分类：社会科学、人文和艺术（综合）

来源: Frontiers

【 摘 要 】

Gene therapy orphan medicinal products constitute a unique group of new drugs which in case of hereditary diseases are usually administered only once at an early age, in the hope to provide sufficient gene product to last for the entire life of the patients. The combination of an exceptionally large single payment and the life-long clinical follow-up needed for understanding the long-term benefits and safety of gene therapy, represent new types of scientific, financial, social and ethical challenges for the pharmaceutical industry, regulators and society. With special consideration of the uniqueness and importance of gene therapy, the authors propose a three points plan for a close cooperation between the pharmaceutical industry and society to develop orphan gene therapy. (1) In fully transparent health technology negotiations a close and long-lasting, contractually fixed cooperation should be established between the manufacturers and local health-care stakeholders for sharing the medical and scientific benefits, the financial risks as well as the burdens of the post-authorization clinical and regulatory development. (2) The parties should agree on a fair, locally affordable drug price without the usually very high premium price calculated to compensate for the low number of patients. In case of high manufacturing costs, the companies should offer prolonged, 15–20 years long payment by installment with risk-sharing, especially considering that the late outcome of the treatment is unknown. Society should assist scientifically and financially organizing a specific patient registry, treatment in specialized hospitals and adequate long-term follow-up of patients, the coordinated management of financial transactions related to the risk sharing program. (3) The post-authorization treatment and prolonged observation of additional new cases coordinated by society should provide real world data needed for the modern complex regulatory evaluation of gene therapy products by the competent authorities. We assume that fair sharing of the benefits and risks as well as a well-organized cooperation of society with the industry in collecting real world evidence might result in better drug evaluation and improved accessibility due to lower prices. The outlined concept might support gene therapy more efficiently than the presently requested outstandingly high prices.

【 授权许可】

CC BY