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Health technology assessment: case study on breast carcinoma treatment in the Czech Republic
Název práce v češtině: Hodnocení zdravotní technologie (HTA):
léčba karcinomu prsu, případová studie ČR
Název v anglickém jazyce: Health technology assessment:
case study on breast carcinoma treatment in the Czech Republic
Klíčová slova: hodnocení zdravotnických technologií, Kaplan-Meierův odhad funkcí přežití, semi-Markovův proces, Coxův model proporcionálních rizik, model zrychleného času, HER2+ metastatická rakovina prsu, pertuzumab, Česká republika
Klíčová slova anglicky: health technology assessment, Kaplan-Meier estimator of survival curves, semi-Markov process, Cox proportional hazard model, accelerated failure time model, HER2+ metastatic breast cancer, pertuzumab, Czech Republic
Akademický rok vypsání: 2016/2017
Typ práce: diplomová práce
Jazyk práce: angličtina
Ústav: Institut ekonomických studií (23-IES)
Vedoucí / školitel: PhDr. Lucie Bryndová
Řešitel: skrytý - zadáno vedoucím/školitelem
Datum přihlášení: 31.05.2017
Datum zadání: 31.05.2017
Datum a čas obhajoby: 16.01.2019 09:00
Místo konání obhajoby: Opletalova - Opletalova 26, O105, Opletalova - místn. č. 105
Datum odevzdání elektronické podoby:04.01.2019
Datum proběhlé obhajoby: 16.01.2019
Oponenti: PhDr. Mgr. Jana Votápková, Ph.D.
 
 
 
Kontrola URKUND:
Seznam odborné literatury
Chicaíza-Becerra, L., García-Molina, M., Gamboa y Carlos Castañeda-Orjuela, O. “ErbB2+metastatic breast cancer treatment after progression on trastuzumab: a cost-effectiveness analysis for a developing country.” Rev. salud public 2014, 16 (March 2014): 270-280. Available at: https://www.ncbi.nlm.nih.gov/pubmed/25383500 (accessed May 12, 2017).

Durkee, B. Y., Qian, Y., Pollom, E. L., King, M. T., Dudley, S. A., Shaffer, J. L., Chang, D. T., Gibbs, I. C., Goldhaber-Fiebert, J. D., Horst, K. C.: “Cost-Effectiveness of Pertuzumab in Human Epidermal: Growth Factor Receptor 2–Positive Metastatic Breast Cancer.” Journal Clinical Oncology 2015, 34 (September 8, 2015): pp. 902-909. Available at: https://www.ncbi.nlm.nih.gov/pubmed/26351332 (accessed May 11, 2017).

Gianni, L., Pienkowski, T., Im, Y.H., Roman, L., Tseng, L.M., Liu, M.C., Lluch, A., Staroslawska, E., de la Haba-Rodriguez, J., Im, S.A., Pedrini, J.L., Poirier, B., Morandi, P., Semiglazov, V., Srimuninnimit, V., Bianchi, G., Szado, T., Ratnayake, J., Ross, G., Valagussa, P. “Efficacy and safety of neoadjuvant pertuzumab and trastuzumab in women with locally advanced, inflammatory, or early HER2-positive breast cancer (NeoSphere): a randomised multicentre, open-label, phase 2 trial.” The Lancet Oncology 2012, 13 (January 2012): pp. 25-32. Available at: http://www.sciencedirect.com/science/article/pii/S1470204511703369 (accessed September 23, 2017).

Sassi, F. “Calculating QALYs, comparing QALY and DALY calculations”. Health policy and planning 2006, 21 (July 2006): pp. 402-8. Available at: https://www.ncbi.nlm.nih.gov/pubmed/16877455 (accessed April 23, 2017).

State Institute for Drug Control. Postup pro posuzování analýzy nákladové efektivity. Prague: State Institute for Drug Control, February 2017. Available at: http://www.sukl.cz/leciva/sp-cau-028 (accessed May 22, 2017).

Wieseler, B., Köhler, M., Haag, S., Biester, K., Brockhaus, A. C., McGauran, N., Grouven, U., Kölsch, H., Seay, U., Hörn, H., Moritz, G., Staeck, K. “Information on new drugs at market entry: retrospective analysis of health technology assessment reports versus regulatory reports, journal publications, and registry reports.” British Medical Journal 2015, 350 (26 February 2015). Available at: http://www.bmj.com/content/350/bmj.h796 (accessed September 23, 2017).

World Health Organization: Using Health Technology Assessment for Universal Health Coverage and Reimbursement Systems. Geneva: World Health Organization, November 2016. Available at: http://www.who.int/health-technology-assessment/HTA_November_meeting_report_Final.pdf (accessed April 23, 2017).
Předběžná náplň práce
Motivation:

The aim of this master’s thesis is to demonstrate the contribution of Health technology assessment (HTA) when using appropriate data that is individual-patients data and economic conditions of a particular country while deciding on drugs reimbursement. Health technology assessment is a research-based evaluation of all available information relevant to use of a drug (or other health technologies) aiming to affect administrative, clinical and political decision-makers. Conducting of HTAs has been promoted since 1970s when interest in the economic aspects of health technologies started to grow in reaction to a rapid growth of new medical technologies and limited health budgets. Nevertheless, creating HTAs is not as common as one would suppose since it is considered to be overly time- and money-consuming.
A health technology assessment will be conducted in this master’s thesis on case study on breast carcinoma treatment in the Czech Republic; in particular, Perjeta is the drug under examination. The State Institute for Drug Control (SIDC) represents the main Czech authority in decision-making in the area of the amounts and conditions of reimbursements for medicinal products. The process of negotiations based on pharmacoeconomic analyses is conducted in administrative procedures individually for each medicinal product. It is reviewed in particular on the basis of safety, cost efficiency, cost benefit, efficacy and budget impact.
Eligibility of Perjeta for state permanent reimbursement in the Czech Republic will be examined using techniques of HTA with the aim to examine whether using data on Czech patients and Czech health and economic conditions leads to significantly different results from studies using data on foreign patients and conditions. These findings will lead to the conclusion whether conducting a demanding HTA should become a standard for new drugs.
The HTA will be carried out in cooperation with the Second Faculty of Medicine of the Charles University that will provide expert medical consultations and access to medical databases.
Breast cancer is the most common cancer in women worldwide and the second most common cancer overall. In the Czech Republic, more than 6500 patients are diagnosed with breast cancer yearly and the incidence rate is continuously growing. The pharmaceutical industry has been successfully developing new drugs targeting various carcinoma types, nevertheless these drugs are generally very expensive and budget rationing has to come to the fore.
Perjeta (active substance pertuzumab) is a recently developed drug approved for use for patients who have HER2-positive breast cancer (approximately 13% of breast cancer patients in the Czech Republic) that has spread to different parts of their body and who have not received anti-HER2 therapy or chemotherapy so far. International clinical studies have been conducted demonstrating significant positive effects on health status of the patients; however an agreement on its reimbursement from statutory health insurance in the CR has not been reached yet between the pharmaceutical company (F. Hoffmann-La Roche Ltd) on one hand and the Institute and health insurance companies (HICs) on the other hand.
The last administrative procedure so far has started on May 12, 2017. Perjeta as a highly innovative medicinal product has already been twice approved before for temporary reimbursement for limited time periods, but has not been approved for permanent reimbursement from social health insurance for the time being. As a consequence, Perjeta is not currently prescribed for treatments in the Czech Republic.


Hypotheses:

Hypothesis #1: Perjeta is cost-effective at stated willingness to pay (SIDC, HICs) to be accepted for permanent reimbursement.
Hypothesis #2: The limits for permanent reimbursement set by the Institute and insurance companies are not adequate in terms of Czech conditions.
Hypothesis #3: Using data on Czech patients and Czech health and economic conditions for conducting HTAs leads to significantly different results in comparison to using only proxies from foreign studies.


Methodology:

Health technology assessment is a research-based evaluation of all available information relevant to use of a drug (or other health technologies) aiming to affect administrative, clinical and political decision-makers. It consists on one hand of profound review of clinical studies, foreign HTAs, regulations of national health care authorities and possibly other sources, and on the other hand consists of an empirical research. Apart from clinical effectiveness, economic effectiveness is assessed based on available data on costs and benefits linked to the newly launched drug.
Cost-effectiveness should be assessed based on direct costs, namely costs of doctor’s examination and diagnosing, pharmaceutical costs, costs of symptomatic treatment and treatment of undesirable effects, costs of hospitalization and costs of after-progression treatment. Benefits of Perjeta (pertuzumab) have been demonstrated by clinical studies in particular in combination with Herceptin (trastuzumab) and chemotherapy (docetaxel). Thus, I will clarify the relationship between discounted costs and consequences of use of the combination of pertuzumab, trastuzumab plus docetaxel in comparison with currently used alternative for the first line of treatment, a combination of trastuzumab plus docetaxel. Both these alternatives are used as treatment for a patient until progression of the disease, unmanageable toxicity, or death.
This usage-oriented pharmacoeconomic analysis employs cost-effectiveness analysis where consequences of the launched drug are assessed in natural units, e.g. years of life gained. Rather than years of life gained another measurement unit that combines duration and quality of life is being promoted lately in health oriented research, quality-adjusted life years (QALYs). Cost-utility analysis then differs from cost-effectiveness analysis in that the consequences are measured in the form of QALYs. Both these analyses will be conducted in this HTA.
Empirical analysis using econometric tools will employ clinical data on Czech patients treated by pertuzumab obtained both from the Czech National Cancer Registry, Czech Society for Oncology and directly from oncology centres (to date over 200 patients treated by Perjeta in the Czech Republic). Some data, especially on QALYs, will be taken from foreign studies. Markov model evaluating the cost effectiveness through transition probabilities will be employed using data on patients’ health states (stable disease, progressing disease and death). Flows of patients between these three states will be quantified together with transition probabilities.
Cost-effectiveness will be given by incremental cost-effectiveness ratio as a commonly acknowledged measure representing the average incremental cost associated with an additional unit of the measure of effect. The results will be verified both by deterministic and probabilistic sensitivity analyses probing the effects of uncertainty in the assumptions about treatment costs and utilities. Budget impact of the reimbursement for health insurance companies will then be estimated and compared with current state of non-reimbursement.


Expected contribution:

This applied microeconomic research of clinically effective drug Perjeta is expected to have a direct impact on admission of this drug for permanent reimbursement from statutory health insurance in the CR. It will broaden the small number of health technology assessments that are being done in the Czech Republic with data on Czech patients. The value added of health technology assessments with data on Czech patients in general will be assessed in contrast to mere adopting of foreign results that represents a common practice today.


Outline:

1. Motivation: I will assess the value added of health technology assessments with data on Czech patients in contrast to adopting of foreign results. I will conduct a usage-oriented pharmacoeconomic analysis aiming to affect administrative, clinical and political decision-makers.
2. Literature review: I will present the most influencing studies on breast carcinoma treatment and foreign HTAs with their methods and results.
3. Data: I will summarize obtained data on Czech patients and Czech health and economic conditions and present basic statistics.
4. Methods: I will explain and employ methods used in HTAs, including cost-effectiveness and cost-utility analysis verifying the results using sensitivity analysis.
5. Results: I will discuss the results and compare these results with other studies.
6. Concluding remarks: I will summarize my findings and their implications for policy decisions and future research.
 
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