Measuring quality of urology care using a qualified clinical data registry.

PURPOSE OF REVIEW: Qualified clinical data registries (QCDRs) serve as a framework for quality improvement efforts, clinical research endeavors, and participation in reimbursement incentive programs. However, the measurement of quality and the recommendations to guide QCDRs in developing new quality measures is a complex process. In this review, we highlight the government policies that lead to the creation of QCDRs, how QCDR quality measures are developed, and the current QCDRs that focus on urological care. RECENT FINDINGS: QCDRs facilitate participation in the merit-based incentive payment system for reimbursement adjustments. Most QCDRs leverage existing clinical guidelines in the development of new quality measures. In 2018, there are four urology QCDRs with quality measures for many urological conditions. These QCDRs form the infrastructure for quality improvement and provide new resources for research endeavors. SUMMARY: Quality measurement within QCDRs will allow urologists to focus improvement efforts to deliver high-quality urological care while also facilitating reimbursement incentives and creating novel research datasets.

[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.

[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.

[Significance of the German Act on the Reform of the Market for Medicinal Products for psychopharmacotherapy]. / Bedeutung des Arzneimittelmarktneuordnungsgesetzes für die Psychopharmakotherapie.

The German Act on the Reform of the Market for Medicinal Products (AMNOG) will lead to rapid disappearance of many new psychotropic drugs from the market in Germany over the next few years or their not being introduced in the first place. This article lists the reasons and discusses possible solutions. In the long term, the AMNOG could not only lead to an improvement of psychopharmacology but also contribute to the development of psychiatry as a whole, especially if its standards become an international reference.

[Impact of early benefit assessment on patients with epilepsy in Germany: Current healthcare provision and therapeutic needs]. / Auswirkungen der frühen Nutzenbewertung auf Patienten mit Epilepsie in Deutschland : Aktuelle Versorgungsrealität und therapeutischer Bedarf.

Epilepsy is one of the most common chronic neurological diseases and represents a significant burden for patients, their families and society. In more than 75 % of patients anticonvulsant therapy consists of valproate, carbamazepine, lamotrigine or levetiracetam. There is a need for polytherapy in drug-refractory patients and they suffer from negative effects on quality of life and employment that is associated with high indirect costs. To allow a comprehensive treatment in this patient group, access to new anticonvulsants with novel modes of action is needed; however, all applications for new antiepileptic drugs failed to prove added benefits during the Pharmaceutical Market Restructuring Act (AMNOG) in Germany. One of the main reasons is the mandatory definition of a standard comparative therapy. It remains unclear whether there will be studies in the future which will fulfill the requirements of the current version of AMNOG. Observational studies after approval and marketing of new antiepileptic drugs could be better alternatives to prove added benefits for individual patients in the current German healthcare system.

[The new German drug market law AMNOG from a child and adolescent psychiatry perspective]. / Das Arzneimittelmarktneuordnungsgesetz aus kinder- und jugendpsychiatrischer Perspektive.

BACKGROUND: The European Union (EU) regulation 1901/2006 plus the implementation of pediatric investigational plans by the European Medicines Agency (EMA) have contributed to more clinical studies in pediatric psychopharmacology. A new drug market law (AMNOG) has been in force in Germany since 2011 that requires an additional process of assessment of benefits of newly authorized medications by the Federal Joint Committee (Gemeinsamer Bundesausschuss, G­BA), which also holds for medications licensed for pediatric populations. OBJECTIVES: Summary of early assessments of benefits for newly registered compounds in the treatment of psychiatric disorders and critical discussion from the perspective of child and adolescent psychiatry. MATERIAL AND METHODS: Application and critical review of documents and written statements by various institutions and stakeholders related to assessment procedures and respective decisions by the G­BA for these medications. RESULTS AND CONCLUSION: Clearly differing requirements for study designs and outcome parameters characterize the conditions for market authorization and for the assessment of benefits. Further adjustments to the regulations in implementing the AMNOG appear to be essential, integrating agencies involved so far, complimented by expertise from regulatory agencies and medical scientific societies.

[Actual medical care situation and therapeutic needs in multiple sclerosis: Impact of the Pharmaceutical Market Restructuring Act (AMNOG)]. / Versorgungsrealität und therapeutischer Bedarf bei Multipler Sklerose : Auswirkungen des AMNOG.

BACKGROUND: The treatment of patients with multiple sclerosis (MS) is associated with constantly rising costs for the healthcare system and pharmaceuticals constitute 60 % of the direct medical costs. The Pharmaceutical Market Restructuring Act (Arzneimittelmarkt-Neuordnungsgesetz, AMNOG) came into force on 1 January 2011 with the aim of limiting the costs of pharmaceuticals by obligating newly approved products to be subjected to an early evaluation of the additional benefits by the Federal Joint Committee (FJC, Gemeinsamer Bundesausschuss, G­BA). The majority of products evaluated up to October 2015 in neurology (5 out of 8) were approved for treatment of MS. OBJECTIVE: Has the AMNOG been able to fulfill the original aims? MATERIAL AND METHODS: Analysis of available information on MS therapies evaluated by the FJC between December 2010 and October 2015. RESULTS: For various reasons an additional benefit could be shown in only 2 out of 5 assessment procedures for MS drugs. It is obvious that some methodological shortcomings of the process have to be improved. Additionally requirements for pivotal clinical trials have to be harmonized with AMNOG requirements taking the best available evidence and real-life data into consideration (e.g. non-interventional studies) and a closer collaboration between the FJC, healthcare providers and the neurological societies is necessary. CONCLUSION: The AMNOG procedure currently only partially fulfills the original aims, which could be the reason why guidelines play a more important role for therapy decision-making than FJC decisions. As the early evaluation procedure is an adaptive process methodological shortcomings might be overcome in the future; however, this requires a much closer collaboration between the FJC and neurological societies.

Medicare Program: Hospital Outpatient Prospective Payment and Ambulatory Surgical Center Payment Systems and Quality Reporting Programs; Organ Procurement Organization Reporting and Communication; Transplant Outcome Measures and Documentation Requirements; Electronic Health Record (EHR) Incentive Programs; Payment to Nonexcepted Off-Campus Provider-Based Department of a Hospital; Hospital Value-Based Purchasing (VBP) Program; Establishment of Payment Rates Under the Medicare Physician Fee Schedule for Nonexcepted Items and Services Furnished by an Off-Campus Provider-Based Department of a Hospital. Final rule with comment period and interim final rule with comment period.

This final rule with comment period revises the Medicare hospital outpatient prospective payment system (OPPS) and the Medicare ambulatory surgical center (ASC) payment system for CY 2017 to implement applicable statutory requirements and changes arising from our continuing experience with these systems. In this final rule with comment period, we describe the changes to the amounts and factors used to determine the payment rates for Medicare services paid under the OPPS and those paid under the ASC payment system. In addition, this final rule with comment period updates and refines the requirements for the Hospital Outpatient Quality Reporting (OQR) Program and the ASC Quality Reporting (ASCQR) Program. Further, in this final rule with comment period, we are making changes to tolerance thresholds for clinical outcomes for solid organ transplant programs; to Organ Procurement Organizations (OPOs) definitions, outcome measures, and organ transport documentation; and to the Medicare and Medicaid Electronic Health Record Incentive Programs. We also are removing the HCAHPS Pain Management dimension from the Hospital Value-Based Purchasing (VBP) Program. In addition, we are implementing section 603 of the Bipartisan Budget Act of 2015 relating to payment for certain items and services furnished by certain off-campus provider-based departments of a provider. In this document, we also are issuing an interim final rule with comment period to establish the Medicare Physician Fee Schedule payment rates for the nonexcepted items and services billed by a nonexcepted off-campus provider-based department of a hospital in accordance with the provisions of section 603.

Craniofacial Surgery and Adverse Outcomes: An Inquiry Into Medical Negligence.

OBJECTIVE: This study aimed to evaluate factors contributing to medical negligence relevant to craniofacial surgery. METHODS: Retrospective analysis of verdict and settlement reports on the Westlaw legal database for outcome, awards, physician defendants, and other specific factors raised in malpractice litigation. RESULTS: Of 42 verdicts and settlement reports included, 52.4% were resolved with either an out-of-court settlement or plaintiff verdict, with aggregate payments totaling $50.1M (in 2013 dollars). Median settlements and jury-awarded damages were $988,000 and $555,000, respectively. Payments in pediatric cases ($1.2M) were significantly higher. Plastic surgeons, oral surgeons, and otolaryngologists were the most commonly named defendants. The most common alleged factors included intraoperative negligence (69.0%), permanent deficits (54.8%), requiring additional surgery (52.4%), missed/delayed diagnosis of a complication (42.9%), disfigurement/scarring (28.6%), postoperative negligence (28.6%), and inadequate informed consent (20.6% of surgical cases). Failure to diagnose a fracture (19.0%) and cleft-reparative procedures (14.3%) were the most frequently litigated entities. CONCLUSION: Medical negligence related to craniofacial surgery involves plaintiffs in a wide age range as well as physician defendants in numerous specialties, and proceedings resolved with settlement and plaintiff verdict involve substantial payments. Cases with death, allegedly permanent injuries, and pediatric plaintiffs had significantly higher payments.

[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.

Jimmo and the improvement standard: implementing Medicare coverage through regulations, policy manuals and other guidance.

In Jimmo v. Sebelius, the plaintiffs alleged that the Centers for Medicare and Medicaid Services (CMS) regularly and improperly denied Medicare reimbursement for outpatient therapy treatment when the beneficiary did not show a likelihood of improvement. These denials, based on policy manuals and other guidance, appear to contradict the government's own regulations, which specifically prohibit coverage denials based solely on the so-called "Improvement Standard." In Jimmo, the United States District Court for the District of Vermont found that CMS' use of the Improvement Standard may have violated the rulemaking provisions of the Administrative Procedure Act (APA) and denied CMS' motion for summary judgment. Subsequently, the parties settled out of court. In the settlement, CMS agreed to revise its policy manuals to clarify that the Improvement Standard was not an acceptable basis on which to deny Medicare coverage. CMS declined to defend its policies even though courts often grant deference to agency interpretations. The settlement implies that the agency feared that it would not have received such deference. It also implies that future Supreme Court decisions may give less deference to agency interpretations.

Regulation, reimbursement, and the long road of implementation of personalized medicine--a perspective from the United States.

There is undisputed evidence that personalized medicine, that is, a more precise assessment of which medical intervention might best serve an individual patient on the basis of novel technology, such as molecular profiling, can have a significant impact on clinical outcomes. The field, however, is still new, and the demonstration of improved effectiveness compared with standard of care comes at a cost. How can we be sure that personalized medicine indeed provides a measurable clinical benefit, that we will be able to afford it, and that we can provide adequate access? The risk-benefit evaluation that accompanies each medical decision requires not only good clinical data but also an assessment of cost and infrastructure needed to provide access to technology. Several examples from the last decade illustrate which types of personalized medicines and diagnostic tests are easily being taken up in clinical practice and which types are more difficult to introduce. And as regulators and payers in the United States and elsewhere are taking on personalized medicine, an interesting convergence can be observed: better, more complete information for both approval and coverage decisions could be gained from a coordination of regulatory and reimbursement questions. Health economics and outcomes research (HEOR) emerges as an approach that can satisfy both needs. Although HEOR represents a well-established approach to demonstrate the effectiveness of interventions in many areas of medical practice, few HEOR studies exist in the field of personalized medicine today. It is reasonable to expect that this will change over the next few years.

Patient Reported Outcome (PRO) assessment in epilepsy: a review of epilepsy-specific PROs according to the Food and Drug Administration (FDA) regulatory requirements.

Despite collection of patient reported outcome (PRO) data in clinical trials of antiepileptic drugs (AEDs), PRO results are not being routinely reported on European Medicines Agency (EMA) and Food and Drug Administration (FDA) product labels. This review aimed to evaluate epilepsy-specific PRO instruments against FDA regulatory standards for supporting label claims. Structured literature searches were conducted in Embase and Medline databases to identify epilepsy-specific PRO instruments. Only instruments that could potentially be impacted by pharmacological treatment, were completed by adults and had evidence of some validation work were selected for review. A total of 26 PROs were reviewed based on criteria developed from the FDA regulatory standards. The ability to meet these criteria was classified as either full, partial or no evidence, whereby partial reflected some evidence but not enough to comprehensively address the FDA regulatory standards. Most instruments provided partial evidence of content validity. Input from clinicians and literature was common although few involved patients in both item generation and cognitive debriefing. Construct validity was predominantly compromised by no evidence of a-priori hypotheses of expected relationships. Evidence for test-retest reliability and internal consistency was available for most PROs although few included complete results regarding all subscales and some failed to reach recommended thresholds. The ability to detect change and interpretation of change were not investigated in most instruments and no PROs had published evidence of a conceptual framework. The study concludes that none of the 26 have the full evidence required by the FDA to support a label claim, and all require further research to support their use as an endpoint. The Subjective Handicap of Epilepsy (SHE) and the Neurological Disorders Depression Inventory for Epilepsy (NDDI-E) have the fewest gaps that would need to be addressed through additional research prior to any FDA regulatory submission, although the NDDI-E was designed as a screening tool and is therefore unlikely to be suitable as an instrument for capturing change in a clinical trial and the SHE lacks the conceptual focus on signs and symptoms favoured by the FDA.

[Certification of colorectal cancer units -- a critical overview on the basis of unsettled aspects]. / Zertifizierung von Darmkrebszentren - eine kritische Standortbestimmung anhand ungeklärter Aspekte.

In this contribution the rational of colorectal cancer centre certification is discussed on the basis of previously published literature and several unsettled factors which are not yet considered as influencing the quality of cancer treatment. The representation of the theme cannot be comprehensive or complete. The readers should be stimulated by one or the other topic to reach their own critical assessment. Due to several limitations of the published literature and the various unsettled aspects, the purpose of colorectal cancer centre certification is called into question.

Fees on health insurance policies and self-insured plans for the patient-centered outcomes research trust fund. Final regulations.

This document contains final regulations that implement and provide guidance on the fees imposed by the Patient Protection and Affordable Care Act on issuers of certain health insurance policies and plan sponsors of certain self-insured health plans to fund the Patient-Centered Outcomes Research Trust Fund. These final regulations affect the issuers and plan sponsors that are directed to pay those fees.

[Clinical outcomes research of traditional Chinese medicine--introduce registry intensive hospital monitoring study protocol of traditiona Chinese medicine injection's safety].

Traditional Chinese medicine (TCM) injection's safety problems has been paid attention for the country and people, TCM injection's labels described the adverse reactions always shows "unclear" or blank, especially the adverse drug reactions(ADR) rates hardly reported. To save the problem, large-scale safety surveillance of TCM injection research is very important. The article introduces the research aim, research type, simple size, outcomes and ethic problem during make the plan. It is a multi-center, large sample size, registry research program about TCM injections safety monitoring. The aim is to get the ADR's rate of TCM injections. According to the "three rules" , each of the TCM injection will be observed for 30 000 cases which are inpatients. The research adopt to using the barcode system and hospital information system(HIS) & laboratory information management system(LIS) to make sure enroll all patients. Case report file (CRF) is used for record patient's information which contained 3 types(A, B, C) tables. Most of people just need to fill table A, and the patient need to fill table B when they have ADR. The outcome of the research is severe ADR. Abide by the international ethical principle to keep the patient's right.