[Biosimilars and the efficiency principle]. / Biosimilars und das Wirtschaftlichkeitsgebot.

Biosimilars are subject to the efficiency evaluation according to section 106 Social Code Book Five (SGB V). The specific evaluation method influences the physician's prescription behaviour. In the case of an individual prescription limit evaluation (Richtgrößenprüfung), the prescription of biosimilars would usually not result in recourse but, as a start, in the initiation of the evaluation proceedings. Starting from 2017, the individual prescription limit evaluation will be cancelled. The active ingredient evaluation (Wirkstoffprüfung) expected instead at a regional level may provide for biosimilar to original drug ratios and result in recourses if these ratios are missed. First agreements on specific evaluation proceedings at a regional level are expected for this year.

The Use of Economic Evaluation to Inform Newborn Screening Policy Decisions: The Washington State Experience.

POLICY POINTS: Newborn screening not only saves lives but can also yield net societal economic benefit, in addition to benefits such as improved quality of life to affected individuals and families. Calculations of net economic benefit from newborn screening include the monetary equivalent of avoided deaths and reductions in costs of care for complications associated with late-diagnosed individuals minus the additional costs of screening, diagnosis, and treatment associated with prompt diagnosis. Since 2001 the Washington State Department of Health has successfully implemented an approach to conducting evidence-based economic evaluations of disorders proposed for addition to the state-mandated newborn screening panel. CONTEXT: Economic evaluations can inform policy decisions on the expansion of newborn screening panels. This article documents the use of cost-benefit models in Washington State as part of the rule-making process that resulted in the implementation of screening for medium-chain acyl-CoA dehydrogenase (MCAD) deficiency and 4 other metabolic disorders in 2004, cystic fibrosis (CF) in 2006, 15 other metabolic disorders in 2008, and severe combined immune deficiency (SCID) in 2014. METHODS: We reviewed Washington State Department of Health internal reports and spreadsheet models of expected net societal benefit of adding disorders to the state newborn screening panel. We summarize the assumptions and findings for 2 models (MCAD and CF) and discuss them in relation to findings in the peer-reviewed literature. FINDINGS: The MCAD model projected a benefit-cost ratio of 3.4 to 1 based on assumptions of a 20.0 percentage point reduction in infant mortality and a 13.9 percentage point reduction in serious developmental disability. The CF model projected a benefit-cost ratio of 4.0-5.4 to 1 for a discount rate of 3%-4% and a plausible range of 1-2 percentage point reductions in deaths up to age 10 years. CONCLUSIONS: The Washington State cost-benefit models of newborn screening were broadly consistent with peer-reviewed literature, and their findings of net benefit appear to be robust to uncertainty in parameters. Public health newborn screening programs can develop their own capacity to project expected costs and benefits of expansion of newborn screening panels, although it would be most efficient if this capacity were shared among programs.

[Requirements for drug approval and additional benefits assessment: Regulatory aspects and experiences]. / Anforderungen an Zulassung und Zusatznutzenbewertung von Arzneimitteln : Regulatorische Aspekte und Erfahrungen.

The early assessment of benefits of newly approved drugs with novel active substances or new applications, which came into force on 1 January 2011 still represents a challenge to all parties involved. This article highlights the definitions, regulatory requirements and interaction between drug marketing approval and early assessment of benefits in Germany. The constellation of an extensively harmonized European and even international drug authorization process with a predominantly national regulation of drug reimbursement situation inevitably causes friction, which could be markedly reduced through early joint advisory discussions during the planning phase for pivotal clinical trials. During the year 2015 the Federal Institute for Drugs and Medical Devices (BfArM) carried out 300 scientific advice procedures of which 34 were concerned with applications in the field of indications for the central nervous system (CNS). In comparison 98 advisory meetings were held by the Federal Joint Committee (G-BA) of which the BfArM provided advice in 12 instances and in 2 cases on CNS indications. Study design, endpoints and appropriate comparative therapies are the key issues in exchanges and discussions between the BfArM, the G­BA and applicants. Under these aspects the BfArM and G­BA promote an early and consistent involvement in early advice procedures regarding the prerequisites for drug approval and assessment of additional benefits.

[Benefit assessment of medical services in German health service - legal framework, historical and international perspective]. / Nutzenbewertung medizinischer Leistungen im deutschen Gesundheitswesen - rechtlicher Rahmen, historische und internationale Perspektive.

The term benefit describes the (positive) causal, patient-relevant consequences of medical interventions, whether diagnostic or therapeutic. Benefit assessments form the basis of rational decision-making within a health care system. They are based on clinical trials that are able to provide valid answers to the question regarding the relevant benefit or harm that can be caused by an intervention. In Germany, evidence-based benefit assessments are fixed by law, i.e., the Social Code Book V. The application and the practical impact of these assessments could be improved.

[Benefit assessment of drugs]. / Nutzenbewertung von Arzneimitteln.

In Germany, new drugs are subject to a benefit assessment at the time of their market access. This "early benefit assessment" is the method primarily used for the benefit assessment of pharmaceuticals in Germany. While for the authorization of a drug a positive risk-benefit ratio is sufficient, early benefit assessment examines whether the new drug has an added benefit compared with other therapies, and thus differs significantly from authorization. For the evaluation, the manufacturer is required to submit a dossier, which must contain all the relevant studies. Early benefit assessment is very transparent in international comparisons, because all the relevant data and the evaluation report will be published. The assessment is carried out with regard to the evidence-based standard of care (the "appropriate comparator"). If the new drug is found to have an additional benefit, the extent of this added benefit is assessed. In addition, groups of patients should be identified with the particular extent of the added benefit. Therefore, subgroup analyses have to be carried out frequently. Often, for new drugs, only registration studies are available. General requirements for such studies (e.g., placebo comparison, endpoints) and decisions regarding the approval process (e.g., dosage regimens) can affect the level of confidence of these studies in the benefit assessment. Joint scientific advice by regulatory authorities and HTA (health technology assessment) agencies are provided to solve this problem. However, this is not possible without additional expense for the pharmaceutical companies.

[Benefit assessment of medical devices]. / Nutzenbewertung von Medizinprodukten.

Medical devices play an important role in both the diagnostic and therapeutic care of patients. The hope is that particularly innovative medical devices can contribute to the improvement of patient care. However, there is no mandatory need to conduct clinical studies with medical devices that allow an assessment of their benefit within the framework of EU market access or on the way to reimbursement by the statutory health insurance (SHI) in Germany. Numerous examples show that the existing legal framework for market access and for reimbursement in the SHI system is insufficient for providing patients with only those examination and treatment methods, i. e., medical devices, that comply with the benefit requirement and the imperative for quality stipulated in the Social Code Book V. However, it is possible to conduct meaningful clinical trials, i. e., randomized controlled trials, with medical devices as well. Hence, regular, indication-related benefit assessment of medical devices with a higher risk class as a prerequisite for reimbursement for a specific medical device is not only necessary, but also feasible. The 2014 report of the Advisory Council on the Assessment of Developments in the Healthcare System contains a promising recommendation for implementing this. A regulatory framework as described in the report would allow patients the fastest possible access to safe and effective medical device innovations, while increasing planning reliability for the development and marketing of new products, which has often been criticized as insufficient by manufacturers.

[The German Pharmaceutical Market Reorganization Act (AMNOG) from an economic point of view]. / Das Arzneimittelmarktneuordnungsgesetz (AMNOG) aus ökonomischer Sicht.

Since the introduction of early benefit assessments in Germany, prices for new medicinal products are set in accordance with the "degree of additional benefit." The major aim of the present work is to point out the economic rationale for the definition of a regulatory price for patent-protected drugs. With regard to the economic objectives of efficient allocation of resources, reducing information asymmetries, and promoting high-value innovation, the applied benefit assessments represent major progress in the German health care sector. In addition to the multifaceted criticism of procedural details, there is a general risk that the institutions involved are lagging behind societal preferences. In this case, early benefit assessments may lead to suboptimal results. The pharmaceutical industry's ability to innovate, on the other hand, may be seen to be a result of the interaction between national benefit assessments and the research activities of internationally oriented drug manufacturers. Accordingly, recent trends toward the implementation of national early benefit assessments in combination with international reference pricing may be seen to be critical; however, Germany is merely following the trend of other countries.

[Disease management programs: Difficulties in the analysis of benefit]. / Disease-Management-Programme : Schwierigkeiten bei der Nutzenbewertung.

After an introduction to the theme with an overview of the implementation of the Disease Management Programs (DMP), accompanying documentation, present utilization and costs of the programs, the present article is primarily devoted to the issue of the analysis of the benefits of DMP. Following an assessment of the legally specified evaluation requirements, in the absence of a prospective, randomized, controlled trial (RCT), the results of three studies are first summarized, which, with the application of propensity score matching, utilize the routine data of the statutory health insurance schemes to form a control group. The overview concludes with a look at the planned changes in evaluation and the intended expansion of the DMP to programs for other chronic illnesses.

[Integrated care in pain therapy: innovations and coordinated treatment offers from the "Techniker Krankenkasse"]. / Integrierte Versorgung in der Schmerztherapie : Innovationen und koordinierte Versorgungsangebote der Techniker Krankenkasse.

Sectorally segregated healthcare structures are seen as a major reason for deficits in quality and efficiency. With the introduction of §§ 140 a ff. SGB V, the sectoral splitting into outpatient, inpatient and rehabilitative services will be eliminated. This is especially true for the requirements of state-of-the-art multidisciplinary and comprehensive pain therapy. Through this form of contract competition and competition for the best idea, incentives shall be created for economic behaviour of market participants, above all the efficient use of limited resources and allocation of healthcare resources based on need. Selective contracts are essential competition parameters for continued innovative development of the healthcare system. They enable statutory healthcare providers to offer their insurants innovative treatments which are not available in standard care. Agreements can be made concerning higher levels of quality and healthcare services, incentives for economical behaviour and success-based payment models. The key idea is the orientation on the needs of the insurant. The successful realisation of innovation in pain therapy is described using a practical example. Professional contractual partners, high quality information and communication, the taking over of responsibility for treatment and cost effectiveness are factors essential the success of innovative treatment concepts.

[Cochlear implant treatment in Germany]. / Cochleaimplantatversorgung in Deutschland.

INTRODUCTION: Restoration of impaired auditory function through cochlear implant is possible, with high reliably and great success. Nevertheless, there are regular disputes between patients and insurance companies due to high costs. In Germany, approx. 1.9 Mio. people are severely hearing impaired. It can be estimated that for adequate hearing rehabilitation about 30,000 cochlear implants/year are necessary. Currently, less than 10% of those affected are offered cochlear implant. DISCUSSION: A handicap is defined if there is deviation from normal hearing for more than 6 months. This sets a time frame for the supply with cochlear implant after sudden deafness. The professional code requires to advice all medical options to a person seeking help for hearing loss. This includes benefit-risk consideration. At this point, the economic aspect plays no role. The indication for medical treatment is only subject to the treating physician and should not be modified by non-physicians or organizations. It should be noted that a supply of hearing aids is qualitatively different to the help from a cochlear implant, which provides a restoration of lost function. In social law (SGB V and IX) doctors are requested to advise and recommend all measures which contribute to normal hearing (both sides). This indicates that doctors may be prosecuted for not offering help when medically possible, just because health insurance employees did not approve the cost balance. CONCLUSION: The current situation, with insufficient medical care for the hearing impaired, needs clarifying. To do this, patients, health insurance companies, the political institutions, legislation and professional societies need to accept their responsibilities.

Objectivity of reimbursement decisions - does it always has to pay off?

UNLABELLED: Rising health care needs impose increased pressure on limited budgets of health care systems around the world. Not only life expectancy is improved, but also increases awareness of patients on modern treatments. It, as a result, leads to the constant search for ways to rationalize health services better attuned to the ability to pay. THE PURPOSE OF THE STUDY: Analysis of the pricing and reimbersement criteria included in the Polish law. MATERIALS AND METHODS: Based on a literature review to specify criteria for the allocation of resources in the health sector and to compare them to those included in the "Act on reimbursement of medicines, foodstuffs intended for particular nutritional and medical devices". RESULTS: Economic criteria dominate the rules governing the reimbursement process of pharmacotherapy in Poland. Referring to the principle of equity they focus on the ability to improve health. Two aspects are repeated frequently: cost-effectiveness and impact on payer's budget. CONCLUSION: Selection of the allocation criteria was carried out in the Polish law to a limited extend, which may give rise to difficulties in making objective reimbursement decisions.

Hospital reimbursement incentives: is there a more effective option?--Part II.

As discussed in Part I of this article, hospital executives in Canada, Germany, and the United States manage their facilities' resources to maximize the incentives inherent in their respective reimbursement system and thereby increase their bottom line. It was also discussed that an additional supply of available hospitals, physicians, and other services will generate increased utilization. Part II discusses how the Patient Protection and Affordable Care Act of 2010 will eventually fail since it neither controls prices nor utilization (e.g., imaging, procedures, ambulatory surgery, discretionary spending). This article concludes with the discussion of the German multipayer approach with universal access and global budgets that might well be a model for U.S. healthcare in the future. Although the German healthcare system has a number of shortfalls, its paradigm could offer the most appropriate compromise when selecting the economic incentives to reduce the percentage of the U.S. gross domestic product expenditure for healthcare from 17.4% to roughly 12.0%.

Early phase clinical trials in pediatric hematology and oncology.

Pediatric oncology is an unrivaled success story in the recent history of medicine. This success is mostly based on a persistent refinement of evidence based therapeutic concepts. With that regard physicians and their staff are highly experience in the conduct of prospective evidence based trials and are therefore competent partners for the pharmaceutical industry. In times of personalized medicine the individual target population is diminishing and the borders of indications are not more disease based. A situation that requires new concepts from the industry. Therefore children with cancer could benefit early from the current developments as well as the pharmaceutical industry could benefit from the legislative incentives through highly recruiting and well conducted prospective trials. Pivotal is a functional platform of communication in order to maintain a close dialogue between academia and pharmaceutical companies.

[Off-label therapy: current problems from the perspective of the Pharmaceutical Commission of the German Medical Profession]. / Off-Label-Therapie: aktuelle Probleme aus Sicht der Arzneimittelkommission der deutschen Ärzteschaft.

The off-label use of approved pharmaceuticals outside the authorized status is implemented in pharmacotherapy of many diseases, especially for rare diseases and in cases of therapy resistance. The German regulations are presented and analyzed and the relative literature is discussed.

The pragmatist's guide to comparative effectiveness research.

Following an acrimonious health care reform debate involving charges of "death panels," in 2010, Congress explicitly forbade the use of cost-effectiveness analysis in government programs of the Patient Protection and Affordable Care Act. In this context, comparative effectiveness research emerged as an alternative strategy to understand better what works in health care. Put simply, comparative effectiveness research compares the efficacy of two or more diagnostic tests, treatments, or health care delivery methods without any explicit consideration of costs. To economists, the omission of costs from an assessment might seem nonsensical, but we argue that comparative effectiveness research still holds promise. First, it sidesteps one problem facing cost-effectiveness analysis--the widespread political resistance to the idea of using prices in health care. Second, there is little or no evidence on comparative effectiveness for a vast array of treatments: for example, we don't know whether proton-beam therapy, a very expensive treatment for prostate cancer (which requires building a cyclotron and a facility the size of a football field) offers any advantage over conventional approaches. Most drug studies compare new drugs to placebos, rather than "head-to-head" with other drugs on the market, leaving a vacuum as to which drug works best. Finally, the comparative effectiveness research can prove a useful first step even in the absence of cost information if it provides key estimates of treatment effects. After all, such effects are typically expensive to determine and require years or even decades of data. Costs are much easier to measure, and can be appended at a later date as financial Armageddon draws closer.

[Reimbursement of new medical therapy]. / Erstattungsfähigkeit neuartiger Arzneimitteltherapien.

Medical progress poses a new financial challenge for the German statutory health insurance. In order to assure the stability of the German statutory health insurance all services have to be economical, but on the other hand the health care system allows access to new therapy in the ambulant and in-patient sector. This article describes in detail how new therapies are reimbursed.