Health technology assessment (HTA) in England, France and Germany: What do matched drug pairs tell us about recommendations by national HTA agencies?
J Comp Eff Res (2021). DOI: 10.2217/cer-2021-0047 (Epub ahead of print).
[available on request]
To explore health technology assessment (HTA) outcomes of matched drug pairs by national agencies in Germany (Gemeinsamer Bundesausschuss, GBA), France (Haute Autorité de Santé, HAS) and England and Wales (NICE).
We considered published GBA decisions, HAS reports and NICE guidance from January 2011 to June 2018. HTAs of matched pairs were compared overall, and for non-cancer and cancer drugs separately. We further analyzed the role of additional attributes related to cancer therapies.
Matched pairs show higher concordance for GBA/HAS than for GBA/NICE and HAS/NICE. Overall, NICE evaluated technologies more favorably than GBA and HAS. GBA appraisals of cancer drugs, however, tended to be more positive than cancer-related recommendations by NICE and HAS.
The findings indicate substantial variations in HTAs, although cancer-related outcomes seem to diverge less than non-cancer results.
Resource allocation in the Covid-19 health crisis: are Covid-19 preventive measures consistent with the Rule of Rescue?
Medicine, Health Care and Philosophy (2021). DOI: 10.1007/s11019-021-10045-0 (Epub ahead of print).
The Covid-19 pandemic has led to a health crisis of a scale unprecedented in post-war Europe. In response, a large amount of healthcare resources have been redirected to Covid-19 preventive measures, for instance population-wide vaccination campaigns, large-scale SARS-CoV-2 testing, and the large-scale distribution of protective equipment (e.g., N95 respirators) to high-risk groups and hospitals and nursing homes. Despite the importance of these measures in epidemiological and economic terms, health economists and medical ethicists have been relatively silent about the ethical rationales underlying the large-scale allocation of healthcare resources to these measures. The present paper seeks to encourage this debate by demonstrating how the resource allocation to Covid-19 preventive measures can be understood through the paradigm of the Rule of Rescue, without claiming that the Rule of Rescue is the sole rationale of resource allocation in the Covid-19 pandemic.
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How Much Does It Cost to Research and Develop a New Drug? A Systematic Review and Assessment.
PharmacoEconomics (2021). DOI: 10.1007/s40273-021-01065-y (Epub ahead of print).
Debate over the viability of the current commercial research and development (R&D) model is ongoing. A controversial theme is the cost of bringing a new molecular entity (NME) to market.
Our aim was to evaluate the range and suitability of published R&D cost estimates as to the degree to which they represent the actual costs of industry.
We provided a systematic literature review based on articles found in the Pubmed, Embase and EconLit electronic databases, and in a previously published review. Articles published before March 2020 that estimated the total R&D costs were included (22 articles with 45 unique cost estimates). We included only literature in which the methods used to collect the information and to estimate the R&D costs were clearly described; therefore, three reports were excluded. We extracted average pre-launch R&D costs per NME and converted the values to 2019 US dollars (US$) using the gross domestic product (GDP) price deflator. We appraised the suitability of the R&D estimated costs by using a scoring system that captures three domains: (1) how success rates and development time used for cost estimation were obtained; (2) whether the study considered potential sources contributing to the variation in R&D costs; and (3) what the components of the cost estimation were.
Estimates of total average capitalized pre-launch R&D costs varied widely, ranging from $161 million to $4.54 billion (2019 US$). Therapeutic area-specific estimates were highest for anticancer drugs (between $944 million and $4.54 billion). Our analysis identified a trend of increasing R&D costs per NME over time but did not reveal a relation between cost estimates and study ranking when the suitability scores were assessed. We found no evidence of an increase in suitability scores over time.
There is no universally correct answer regarding how much it costs, on average, to research and develop an NME. Future studies should explicitly address previously neglected variables, which likely explain some variability in estimates, and consider the trade-off between the transparency and public accessibility of data and their specificity. Use of our proposed suitability scoring system may assist in addressing such issues.
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Do certified cancer centers provide more cost-effective care? A health economic analysis of colon cancer care in Germany using administrative data.
International Journal of Cancer (2021). DOI: 10.1002/ijc.33728 (Epub ahead of print).
Hospital certification has become an important measure to improve cancer care quality, with the potential effect of prolonging patient survival and reducing medical spending. However, yet to be explored is the cost-effectiveness of cancer care provided in certified hospitals, considering significant additional costs incurred from certification requirements. We performed a cost-effectiveness analysis (CEA) using two colon cancer populations (N = 1909) treated in different levels of certified hospitals (CHs) vs noncertified hospitals (NCHs) from a healthcare system's perspective. We matched patient-level data of incident colon cancer cases, diagnosed between 2008 and 2013 from a large statutory health insurance in Saxony, Germany, to calculate net treatment costs by phase (initial, continuing and terminal phase). The costs were supplemented with extra costs from 31 additional services required for certification. Effectiveness measure was total survival time in life-years. Outcome of interest was incremental costs per additional life-year. The annualized net colon cancer treatment costs by phase showed a U shape with high costs in the initial (mean €26 855; 95% CI €25 058-€28 652) and the terminal phases (mean €30 096; 95% CI €26 199-€33 993). The base-case CEA results and all sensitivity analyses consistently demonstrated longer survival and lower costs for the colon cancer cohort treated in CHs vs NCHs. To conclude, we used administrative data to derive the first cost-effectiveness evidence supporting that colon cancer care delivered in the certified cancer centers in Germany improves survival outcomes and saves costs from a healthcare system's perspective. Generalization of the study results should be exercised with caution.
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Orphan Medicine Incentives:
How to address the unmet needs of disease patients by transforming the European OMP landscape?
Copenhagen Economics (May 2021), Booklet.
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Income loss after a cancer diagnosis in Germany: An analysis based on the socio-economic panel survey.
Cancer Medicine (2021), 10(11): 3726–3740.
Cancer treatments often require intensive use of healthcare services and limit patients' ability to work, potentially causing them to become financially vulnerable. The present study is the first attempt to measure, on the German national level, the magnitude of absolute income loss after a cancer diagnosis.
This study analyzes data from the Socio-Economic Panel (SOEP) survey, one of the largest and most comprehensive household surveys in Germany, consisting of approximately 20,000 individuals, who are traced annually. The empirical strategy consists of ordinary least squares (OLS) and multinomial logistic estimators to measure changes in job income, work status, working hours, and pension as a result of reporting a cancer diagnosis for the period between 2009 and 2015. Sample consistency checks were conducted to limit measurement error biases.
Our results show that job incomes dropped between 26% and 28% within the year a cancer diagnosis was reported. The effect persisted for two years after the diagnosis and was no longer observable after four years. The finding was linked to an increased likelihood of unemployment and a reduction of working hours by 24%. Pension levels, on the other hand, were not affected by a cancer diagnosis.
These findings suggest that many cancer patients are exposed to financial hardship in Germany, particularly when the cancer diagnosis occurs during their working age and before requirements to obtain a pension are met. Further research seems warranted to identify particularly vulnerable patient groups.
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Cost-effectiveness of risk-based breast cancer screening: A systematic review.
International Journal of Cancer (2021). DOI: 10.1002/ijc.33593 (Epub ahead of print).
To analyse published evidence on the economic evaluation of risk-based screening (RBS), a full systematic literature review was conducted. After a quality appraisal, we compared the cost-effectiveness of risk-based strategies (low-risk, medium-risk and high-risk) with no screening and age-based screening. Studies were also analysed for modelling, risk stratification methods, input parameters, data sources and harms and benefits. The 10 modelling papers analysed were based on screening performance of film-based mammography (FBM) (three); digital mammography (DM) and FBM (two); DM alone (three); DM, ultrasound (US) and magnetic resonance imaging (one) and DM and US (one). Seven studies did not include the cost of risk-stratification, and one did not consider the cost of diagnosis. Disutility was incorporated in only six studies (one for screening and five for diagnosis). None of the studies reported disutility of risk-stratification (being considered as high-risk). Risk-stratification methods varied from only breast density (BD) to the combination of familial risk, genetic susceptibility, lifestyle, previous biopsies, Jewish ancestry and reproductive history. Less or no screening in low-risk women and more frequent mammography screening in high-risk women was more cost-effective compared to no screening and age-based screening. High-risk women screened annually yielded a higher mortality rate reduction and more quality-adjusted life years at the expense of higher cost and false positives. RBS can be cost effective compared to the alternatives. However, heterogeneity among risk-stratification methods, input parameters, and weaknesses in the methodologies hinder the derivation of robust conclusions. Therefore, further studies are warranted to assess newer technologies and innovative risk-stratification methods.
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PRO ("patient-reported outcomes") und Lebensqualität in der Onkologie.
[Patient-reported outcomes (PRO) and quality of life in oncology.]
Forum (2020), 35: 382-390.
[available on request]
It is widely accepted that with patient-centered care, attempting to meet the patient’s individual health needs and desired outcomes should be a primary objective of healthcare provision. In order to treat patients not only from a biological and clinical perspective but to also take into account the mental, psychosocial and financial dimensions of cancer and cancer care, the expectations and experience of patients need to be understood. This requires research into patient-reported outcomes (PRO), including but not limited to (self-reported) health-related quality of life (HRQoL). This article provides a brief overview of some of the recurring issues that arise when preference-based instruments are used to measure the HRQoL of cancer patients. Despite massive research efforts, substantial progress, and considerable interest, these instruments have not yet been used to their full potential. This observation is particularly striking in the context of approval of new cancer drugs. There is also a need for more research into PROs beyond HRQoL, which include the potentially far-reaching psychological, social, and financial consequences of cancer. It is therefore suggested to prioritize socioeconomic impact analyses from the perspective of patients and their relatives, including both caregivers and dependents (e.g. children), with a view towards generating robust evidence on the full dimension of the issue and towards identifying particularly vulnerable patient groups.
1. Comments on "Cost of decentralized CAR T-cell production in an academic nonprofit setting" by Schmitt M et al.
2. Reply to: Comments on "Cost of decentralized CAR T-cell production in an academic nonprofit setting" by Ran T et al.
International Journal of Cancer (2020).
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Cost of decentralized CAR T-cell production in an academic nonprofit setting.
International Journal of Cancer (2020), 147(12): 3438-3445.
Chimeric antigen receptor (CAR) T‐cell therapy is a promising immunotherapy with high acquisition costs, and it has raised concerns about affordability and sustainability in many countries. Furthermore, the current centralized production paradigm for the T cells is less than satisfactory. Therefore, several countries are exploring alternative T‐cell production modes. Our study is based on the T‐cell production experience in a nonprofit setting in Germany. We first identified the work steps and main activities in the production process. Then we determined the fixed costs and variable costs. Main cost components included personnel and technician salaries, expenditure on equipment, a clean room, as well as production materials. All costs were calculated in 2018 euros and converted into U.S. dollars. For a clean room with one machine for closed and automated manufacturing installed, annual fixed costs summed up to approximately €438 098 ($584 131). The variable cost per production was roughly €34 798 ($46 397). At the maximum capacity of one machine, total cost per product would be close to €60 000 ($78 849). As shown in the scenario analysis, if three machines were to be installed in the clean room, per production cost could be as low as €45 000 (roughly $59905). If a cheaper alternative to lentivirus was used, per production total cost could be further reduced to approximately €33 000 (roughly $44309). Decentralized T‐cell production might be a less costly and more efficient alternative to the current centralized production mode that requires a high acquisition cost.
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Allen Patienten gerecht werden - Gedanken eines Gesundheitsökonomen zur Covid-19-Krise.
Newspaper Article / Frankfurter Allgemeine Zeitung / No. 124, p. 20 (2020/05/29).
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HTA agencies need evidence-informed deliberative processes:
Comment on “Use of evidence-informed deliberative processes by health technology assessment agencies around the globe".
Int J Health Policy Manag (2020). DOI: 10.34172/ijhpm.2020.22 (Epub ahead of print).
There are at least two reasons why health technology assessment (HTA) agencies need to seek process-based solutions to support the legitimacy of healthcare resource allocation, ie, (i) in pluralistic societies, the existence of often conflicting and incommensurable claims (ie, the “fragmentation of value”) and the lack of a broadly accepted, ethically defensible analytical framework, and (ii) the well-documented loopholes of the conventional logic of cost-effectiveness (CE) with its reductionist concept of allocative efficiency, which fails to reflect the distributive dimension of resource allocation decisions in collectively financed health schemes.
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Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicine Agency for hereditary metabolic diseases in the MetabERN network.
Orphanet Journal of Rare Diseases, (2020) Jan 6; 15(1): 3.
The European Medicine Agency granted marketing approval to 164 orphan medicinal products for rare diseases, among which 28 products intended for the treatment of hereditary metabolic diseases. Taking advantage of its privileged connection with 69 healthcare centres of excellence in this field, MetabERN, the European Reference Network for hereditary metabolic diseases, performed a survey asking health care providers from 18 European countries whether these products are available on the market, reimbursed and therefore accessible for prescription, and actually delivered in their centre.
Responses received from 52 centres (75%) concerned the design of treatment plans, the access to marketed products, and the barriers to delivery. Treatment options are always discussed with patients, who are often involved in their treatment plan. Most products (26/28) are available in most countries (15/18). Among the 15 broadly accessible products (88.5% of the centres), 9 are delivered to most patients (mean 70.1%), and the others to only few (16.5%). Among the 10 less accessible products (40.2% of the centres), 6 are delivered to many patients (66.7%), and 4 are rarely used (6.3%). Information was missing for 3 products. Delay between prescription and delivery is on average one month. Beside the lack of availability or accessibility, the most frequent reasons for not prescribing a treatment are patients’ clinical status, characteristic, and personal choice.
Data collected from health care providers in the MetabERN network indicate that two-third of the orphan medicines approved by EMA for the treatment of hereditary metabolic diseases are accessible to treating patients, although often less than one-half of the patients with the relevant conditions actually received the approved product to treat their disease. Thus, in spite of the remarkable achievement of many products, patients concerned by EMA-approved orphan medicinal products have persistent unmet needs, which deserve consideration. The enormous investments made by the companies to develop products, and the high financial burden for the Member States to purchase these products emphasize the importance of a scrupulous appreciation of treatment value involving all stakeholders at early stage of development, before marketing authorization, and during follow up.
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