Novel approach to decision making for orphan drugs

J 2023

Novel approach to decision making for orphan drugs

DECKER, Barbora, Tomáš MLČOCH, Anastasie PUSTOVALOVA and Tomáš DOLEŽAL

Basic information

Original name

Novel approach to decision making for orphan drugs

Authors

DECKER, Barbora (203 Czech Republic, guarantor, belonging to the institution), Tomáš MLČOCH (203 Czech Republic), Anastasie PUSTOVALOVA (203 Czech Republic) and Tomáš DOLEŽAL (203 Czech Republic, belonging to the institution)

Edition

International Journal of Technology Assessment in Health Care, NEW YORK, CAMBRIDGE UNIV PRESS, 2023, 0266-4623

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30104 Pharmacology and pharmacy

Country of publisher

United States of America

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 3.200 in 2022

RIV identification code

RIV/00216224:14110/23:00130319

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1017/S0266462323000053

UT WoS

000924890900001

Keywords in English

orphan drugs; rare diseases; reimbursement; health policy; health technology assessment

Abstract

V originále

Background Out of 185 orphan medicinal products (OMPs) registered in 2015–2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this proportion has steadily decreased over time. After years of public debate induced by this unsatisfactory OMP patient access, the national viewpoint shifted toward creating a special pathway for the reimbursement of OMP. Thus, a rigorous pricing and reimbursement procedure with strict timelines and elaborated methodology has been recently adopted in Czechia. Methodology The innovative legislation follows the recommendations for value assessment and funding processes for rare diseases and incorporates additional elements of value, such as the societal perspective. First, the application with clinical evidence, cost-effectiveness, and budget impact analyses is submitted to the governmental health technology assessment (HTA) agency by the Marketing Authorization Holder or a Health Insurance Fund. Moreover, professional associations and patients’ organizations are rightful participants in the proceeding, providing evidence and comments. Then, the HTA agency performs the assessment/appraisal of the evidence. It subsequently publishes the assessment report summarizing available information. The report is then forwarded to the Ministry of Health and its advisory body consisting of patients, clinical experts, health insurance funds, and the State. They critically evaluate the documents and issue a binding opinion following prespecified decision-making criteria. Based on this binding opinion, the decision is issued by the HTA agency. Thus, the role of the advisory body in this process is crucial. Conclusion We believe that this novel approach may offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of “value-based” decision making. Background Out of 185 orphan medicinal products (OMPs) registered in 2015–2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this proportion has steadily decreased over time. After years of public debate induced by this unsatisfactory OMP patient access, the national viewpoint shifted toward creating a special pathway for the reimbursement of OMP. Thus, a rigorous pricing and reimbursement procedure with strict timelines and elaborated methodology has been recently adopted in Czechia. Methodology The innovative legislation follows the recommendations for value assessment and funding processes for rare diseases and incorporates additional elements of value, such as the societal perspective. First, the application with clinical evidence, cost-effectiveness, and budget impact analyses is submitted to the governmental health technology assessment (HTA) agency by the Marketing Authorization Holder or a Health Insurance Fund. Moreover, professional associations and patients’ organizations are rightful participants in the proceeding, providing evidence and comments. Then, the HTA agency performs the assessment/appraisal of the evidence. It subsequently publishes the assessment report summarizing available information. The report is then forwarded to the Ministry of Health and its advisory body consisting of patients, clinical experts, health insurance funds, and the State. They critically evaluate the documents and issue a binding opinion following prespecified decision-making criteria. Based on this binding opinion, the decision is issued by the HTA agency. Thus, the role of the advisory body in this process is crucial. Conclusion We believe that this novel approach may offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of “value-based” decision making.

Links

MUNI/A/1342/2022, interní kód MU

Name: Preklinický a klinický výzkum v oblasti farmakokinetiky, neuropsychofarmakologie a personalizované farmakoterapie v onkologii

Investor: Masaryk University, Preclinical and clinical research in pharmacokinetics, neuropsychopharmacology and personalized pharmacotherapy in oncology

Citovat

DECKER, Barbora, Tomáš MLČOCH, Anastasie PUSTOVALOVA and Tomáš DOLEŽAL. Novel approach to decision making for orphan drugs. International Journal of Technology Assessment in Health Care. NEW YORK: CAMBRIDGE UNIV PRESS, 2023, vol. 39, No 1, p. 1-6. ISSN 0266-4623. Available from: https://dx.doi.org/10.1017/S0266462323000053.

@article{2254723,
   author = {Decker, Barbora and Mlčoch, Tomáš and Pustovalova, Anastasie and Doležal, Tomáš},
   article_location = {NEW YORK},
   article_number = {1},
   doi = {http://dx.doi.org/10.1017/S0266462323000053},
   keywords = {orphan drugs; rare diseases; reimbursement; health policy; health technology assessment},
   language = {eng},
   issn = {0266-4623},
   journal = {International Journal of Technology Assessment in Health Care},
   title = {Novel approach to decision making for orphan drugs},
   url = {https://www.cambridge.org/core/journals/international-journal-of-technology-assessment-in-health-care/article/novel-approach-to-decision-making-for-orphan-drugs/B040F9FED7D2CCDEEB83D33080AF5E15},
   volume = {39},
   year = {2023}
}

TY  - JOUR
ID  - 2254723
AU  - Decker, Barbora - Mlčoch, Tomáš - Pustovalova, Anastasie - Doležal, Tomáš
PY  - 2023
TI  - Novel approach to decision making for orphan drugs
JF  - International Journal of Technology Assessment in Health Care
VL  - 39
IS  - 1
SP  - 1-6
EP  - 1-6
PB  - CAMBRIDGE UNIV PRESS
SN  - 02664623
KW  - orphan drugs
KW  - rare diseases
KW  - reimbursement
KW  - health policy
KW  - health technology assessment
UR  - https://www.cambridge.org/core/journals/international-journal-of-technology-assessment-in-health-care/article/novel-approach-to-decision-making-for-orphan-drugs/B040F9FED7D2CCDEEB83D33080AF5E15
N2  - Background Out of 185 orphan medicinal products (OMPs) registered in 2015–2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this proportion has steadily decreased over time. After years of public debate induced by this unsatisfactory OMP patient access, the national viewpoint shifted toward creating a special pathway for the reimbursement of OMP. Thus, a rigorous pricing and reimbursement procedure with strict timelines and elaborated methodology has been recently adopted in Czechia. Methodology The innovative legislation follows the recommendations for value assessment and funding processes for rare diseases and incorporates additional elements of value, such as the societal perspective. First, the application with clinical evidence, cost-effectiveness, and budget impact analyses is submitted to the governmental health technology assessment (HTA) agency by the Marketing Authorization Holder or a Health Insurance Fund. Moreover, professional associations and patients’ organizations are rightful participants in the proceeding, providing evidence and comments. Then, the HTA agency performs the assessment/appraisal of the evidence. It subsequently publishes the assessment report summarizing available information. The report is then forwarded to the Ministry of Health and its advisory body consisting of patients, clinical experts, health insurance funds, and the State. They critically evaluate the documents and issue a binding opinion following prespecified decision-making criteria. Based on this binding opinion, the decision is issued by the HTA agency. Thus, the role of the advisory body in this process is crucial. Conclusion We believe that this novel approach may offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of “value-based” decision making. Background Out of 185 orphan medicinal products (OMPs) registered in 2015–2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this proportion has steadily decreased over time. After years of public debate induced by this unsatisfactory OMP patient access, the national viewpoint shifted toward creating a special pathway for the reimbursement of OMP. Thus, a rigorous pricing and reimbursement procedure with strict timelines and elaborated methodology has been recently adopted in Czechia. Methodology The innovative legislation follows the recommendations for value assessment and funding processes for rare diseases and incorporates additional elements of value, such as the societal perspective. First, the application with clinical evidence, cost-effectiveness, and budget impact analyses is submitted to the governmental health technology assessment (HTA) agency by the Marketing Authorization Holder or a Health Insurance Fund. Moreover, professional associations and patients’ organizations are rightful participants in the proceeding, providing evidence and comments. Then, the HTA agency performs the assessment/appraisal of the evidence. It subsequently publishes the assessment report summarizing available information. The report is then forwarded to the Ministry of Health and its advisory body consisting of patients, clinical experts, health insurance funds, and the State. They critically evaluate the documents and issue a binding opinion following prespecified decision-making criteria. Based on this binding opinion, the decision is issued by the HTA agency. Thus, the role of the advisory body in this process is crucial. Conclusion We believe that this novel approach may offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of “value-based” decision making.
ER  -

DECKER, Barbora, Tomáš MLČOCH, Anastasie PUSTOVALOVA and Tomáš DOLEŽAL. Novel approach to decision making for orphan drugs. \textit{International Journal of Technology Assessment in Health Care}. NEW YORK: CAMBRIDGE UNIV PRESS, 2023, vol.~39, No~1, p.~1-6. ISSN~0266-4623. Available from: https://dx.doi.org/10.1017/S0266462323000053.

Detailed Information on Publication Record