Adri Steenhoek

Orphan drugs expenditure in the Netherlands in the period 2006–2012

BackgroundThe relatively low budget impact of orphan drugs is often used as an argument in reimbursement decisions. However, overall, the budget impact of orphan drugs can still be substantial. In this study, we assess the uptake and budget impact of orphan drugs in the Netherlands.MethodsWe examined the number of orphan drugs, the number of patients and budget impact of orphan drugs in the Netherlands in the period 2006 to 2012, both for inpatient and outpatient orphan drugs. Budget impact was provided in absolute numbers and relative to total pharmaceutical spending.ResultsThe number of orphan drugs and patients treated increased substantially over the period studied. Overall, budget impact increased substantially over a period of six years, both in absolute terms (326% increase) as well as relative to total pharmaceutical spending (278% increase). Growth rates decreased over time. In 2012, 17% of available drugs had an individual budget impact of more than €10 million per year.ConclusionsIndividual budget impact of orphan drugs is often limited, although exceptions exist. However, in total, the budget impact of orphan drugs is considerable and has grown substantially over the years. This could potentially influence reimbursement decisions for orphan drugs in the future.

Productivity and quality of Dutch hospitals during system reform

This study addresses the productivity of Dutch hospitals since the start of the health systems reform in 2005. We consider DEA based measures, which include efficiency and quality for the complete set of Dutch hospitals and present cross-sectional and longitudinal analysis. In particular, we consider how hospital efficiency has developed. As the reform created an environment of regulated competition, we pay special attention to relative efficiency. Our results suggest that the differences in efficiency among hospitals have become larger. In the years 2009–2010, the number of hospitals identified as (close to) efficient by DEA analysis decreased.

Practical feasibility of outcomes research in oncology: lessons learned in assessing drug use and cost-effectiveness in The Netherlands.

OBJECTIVE To investigate the practical feasibility to develop evidence on drug use and cost-effectiveness in oncology practice. PATIENTS AND METHODS Feasibility was examined using three Dutch case studies. Each case study investigated the degree of appropriate drug use and its incremental cost-effectiveness. Detailed data were retrospectively collected from hospital records. In total, 391, 316 and 139 patients with stage III colon cancer, metastatic colorectal cancer and multiple myeloma were included in 19, 29 and 42 hospitals, respectively. RESULTS The methods used in the case studies were feasible to develop evidence on some aspects of drug use including types of treatments used, dosages, dose modifications and healthcare costs. Aspects such as baseline patient characteristics, reasons to start or stop a treatment and treatment effects were less feasible because of missing values. Despite difficulties to correct for confounding by indication, it was possible to estimate incremental cost-effectiveness by synthesising evidence in two of the three case studies. CONCLUSION It is possible to generate evidence about drug use and cost-effectiveness in oncology practice to facilitate informed decision-making by both payers and physicians. This can improve quality of care and enhance the efficient allocation of resources. However, the optimal approach differs between drugs and their indications. Generating high-quality evidence requires active interdisciplinary collaboration. Patient registries can facilitate data collection but cannot resolve all issues. In most circumstances it is inevitable to use data-synthesis to obtain valid incremental cost-effectiveness estimates, but for some indications it will not be feasible to derive a valid and precise estimate.

Health economic evaluations in reimbursement decision making in the Netherlands: Time to take it seriously?

Health technology assessment already informed Dutch policymaking in the early 1980s. Evidence of health economic evaluations is, however, only systematically used in drug reimbursement decision making. Outpatient drugs with an added therapeutic value and expensive specialist drugs require evidence from an economic evaluation. Due to many exemptions, however, the availability of evidence of health economic evaluations remains rather low. Although the Dutch reimbursement agency suggested a cost-effectiveness threshold range depending on the severity of the disease (i.e., €10,000 - 80,000 per Quality Adjusted Life Year), it was never confirmed nor endorsed by the Ministry of Health. It is highly questionable whether health economic evaluations currently play a role in actual Dutch reimbursement decision making. Although the requirements exist in policy procedures, recent cases show that Dutch policymakers experience great difficulties in putting restrictions on reimbursement based on evidence from health economic evaluations. The near future will show whether the need will increase to base decisions on societal value for money, and whether Dutch policymakers show the courage to take health economic evaluations seriously.

The economics of improved cancer survival rates: better outcomes, higher costs.

In 2006, over three million new cases of cancer were diagnosed in Europe. This number will increase in the coming years as a result of an aging population and population growth. Advances in the diagnosis and treatment of cancer have resulted in increased survival rates. Simultaneously, increasing costs of screening, diagnosis and the treatment of cancer could threaten the ability to ensure high-quality care and provide access to care for all patients. New genetic tests and biomarkers may help to identify those subtypes of patients that would be most likely to benefit from new cancer drugs. In our opinion, there is still much to gain in cancer diagnosis and treatment but these gains should be worth the costs.

The Drug Reimbursement Decision-Making System in Iran

BACKGROUND Previous studies of health policies in Iran have not focused exclusively on the drug reimbursement process. OBJECTIVE The aim of this study was to describe the entire drug reimbursement process and the stakeholders, and discuss issues faced by policymakers. METHODS Review of documents describing the administrative rules and directives of stakeholders, supplemented by published statistics and interviews with experts and policymakers. RESULTS Iran has a systematic process for the assessment, appraisal, and judgment of drug reimbursements. The two most important organizations in this process are the Food and Drug Organization, which considers clinical effectiveness, safety, and economic issues, and the Supreme Council of Health Insurance, which considers various criteria, including budget impact and cost-effectiveness. Ultimately, the Iranian Cabinet approves a drug and recommends its use to all health insurance organizations. Reimbursed drugs account for about 53.5% of all available drugs and 77.3% of drug expenditures. Despite its strengths, the system faces various issues, including conflicting stakeholder aims, lengthy decision-making duration, limited access to decision-making details, and rigidity in the assessment process. CONCLUSIONS The Iranian drug reimbursement system uses decision-making criteria and a structured approach similar to those in other countries. Important shortcomings in the system include out-of-pocket contributions due to lengthy decision making, lack of transparency, and conflicting interests among stakeholders. Iranian policymakers should consider a number of ways to remedy these problems, such as case studies of individual drugs and closer examination of experiences in other countries.

Uitkomstenonderzoek: een definitie is dringend gewenst

SamenvattingHet doet ons genoegen dat collega Krabbe het met ons eens is dat er op dit moment geen eenduidigheid bestaat met betrekking tot het begrip ‘uitkomstenonderzoek’ en dat het noodzakelijk is om hieraan via een heldere definitie een eind aan te maken. Tevens bevestigt hij de grote actualiteit van het onderwerp, mede in het licht van de ook door hem geciteerde en door ons al genoemde maatschappijbrede ‘outcomes movement’.

Uitkomstenonderzoek: hoogste tijd voor een definitie!

Outcomes research: it’s the moment to set up definitionsIn the Dutch society the financing of services as health care and education is in a collective way. More than in the past providers of these services have to explain the relation between the costs and the quality of the services. It’s the era of an ‘outcomes movement’. Health care also must be prepared for these effectiveness questions. At this moment methods are developed to implement the results of outcomes research with (expensive) drugs in health care policy and in the society. Because the developments are rather new, clear and broadly accepted definitions of some basic terms are lacking. It’s the moment to set up these definitions now, also for outcomes research itself. Outcomes research is driven by objectives and is done in a specific context or perspective. The results support decisions on a macro, meso and/or micro level.