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Affordability of in vivo gene therapy. Problems and potential solutions

https://doi.org/10.17749/2070-4909/farmakoekonomika.2020.040

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Abstract

Aim. To review foreign approaches to the evaluation and financing of in vivo gene therapy drugs.

Materials and Methods. The first stage of the review included the systematic search for publications of clinical-economic studies (CES) on in vivo gene therapy drugs registered on the international pharmaceutical market. The analysis of the methodology used in the found CESs was performed based on the given criteria. The second and third stages of the review included the search for the information on the approaches and results of the evaluation of in vivo gene therapy drugs by foreign agencies on the assessment of healthcare technologies (AHT) and the implemented approaches to the financing of expensive medical technologies.

Results. Totally, four in vivo gene therapy drugs registered on the foreign markets were found: talimogene laherparepvec (Imlygic), voretigene neparvovec (Luxturna), onasemnogene abeparvovec (Zolgensma), and alipogene tiparvove (Glybera). The analysis of the methodology of CES on in vivo gene therapy drugs did not reveal general methodological peculiarities. The analysis of the approaches to the reimbursement for the cost of these four drugs in the European countries showed that there were no special procedures intended for gene drugs. However, in the majority of countries, there are some mechanisms of reimbursement for orphan drugs and the majority of the above-mentioned in vivo gene therapy drugs are orphan. It was revealed that the most perspective methods of payment for in vivo gene therapy drugs were installment payments, risk-sharing agreements, and their combinations. Still, none of these payment methods are implemented in Russia because of the lack of regulatory and legal framework.

Conclusion. The design and methodological peculiarities of CES on in vivo gene therapy drugs are similar to orphan drugs (in some countries, there is a special flexible procedure). In the Russian Federation, there is no special procedure of ATH for orphan drugs, which can provide difficulties in the reimbursement for these drugs. Besides, in the Russian Federation, the issue of the implementation of innovative models for drugs provision (risk-sharing, installment payments is acute). The changes in this area would also lead to the optimization of the influence of expensive healthcare technologies on the budget of the system of healthcare.

About the Authors

V. V. Omelyanovskiy
Center for Healthcare Quality Assessment and Control of the Ministry of Healthcare of the Russian Federation; Financial Research Institute of the Ministry of Finance of the Russian Federation; Russian Medical Academy of Postgraduate Education, Moscow
Russian Federation

MD, Dr Sci Med, Professor, General Director, Head of the Center for Healthcare Funding

Researcher ID: P-6911-2018; Scopus Author ID: 6507287753  

10-5 Khokhlovsky per., Moscow 109028, Russia

3-2 Nastasyinsky per., Moscow 127006, Russia

2-1 Barrikadnaya str., Moscow 123995, Russia



N. Z. Musina
Center for Healthcare Quality Assessment and Control of the Ministry of Healthcare of the Russian Federation; Saint Petersburg State Chemical Pharmaceutical University; Russian Presidential Academy of National Economy and Public Administration
Russian Federation

PhD (Pharmaceutical Sciences), Associate Professor at the Department of management; Head of the HTA Research Laboratory of the Institute for Applied Economic Research; Head of Development 

Researcher ID: C-8075-2018  

10-5 Khokhlovsky per., Moscow 109028, Russia

14 let. A Prof. Popov str., Saint Petersburg 197376, Russian Federation

82 Vernadskogo pr., Moscow 119571, Russia



V. A. Lemeshko
Center for Healthcare Quality Assessment and Control of the Ministry of Healthcare of the Russian Federation; Financial Research Institute of the Ministry of Finance of the Russian Federation; Institute of Pharmacy and Translational Medicine, Sechenov University
Russian Federation

Deputy Head of the Department of Methodological Support of Comprehensive Health Technology Assessment; Research assistant at the Center for Healthcare Funding; Post-graduate student at the Department of Pharmacology, Educational Department

Researcher ID: AAD-8959-2019  

10-5 Khokhlovsky per., Moscow 109028, Russia

3-2 Nastasyinsky per., Moscow 127006, Russia

8-2 Trubetskaya str., Moscow 119991, Russia



A. A. Antonov
Center for Healthcare Quality Assessment and Control of the Ministry of Healthcare of the Russian Federation
Russian Federation

Lead Specialist, Department of Methodological Support for Comprehensive HTA

10-5 Khokhlovsky per., Moscow 109028, Russia



F. V. Gorkavenko
Center for Healthcare Quality Assessment and Control of the Ministry of Healthcare of the Russian Federation
Russian Federation

Chief Specialist, Department of Methodological Support for Comprehensive HTA

Researcher ID: A-8805-2017

10-5 Khokhlovsky per., Moscow 109028, Russia



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For citation:


Omelyanovskiy V.V., Musina N.Z., Lemeshko V.A., Antonov A.A., Gorkavenko F.V. Affordability of in vivo gene therapy. Problems and potential solutions. FARMAKOEKONOMIKA. Modern Pharmacoeconomic and Pharmacoepidemiology. 2020;13(2):170-182. (In Russ.) https://doi.org/10.17749/2070-4909/farmakoekonomika.2020.040

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