Accelerated access to innovative medicines for patients in need.

There is broad agreement among health-care stakeholders that more must be done to ensure that patients have timely access to new and innovative medicines. Assuming that industry will continue to develop such medicines at a sustainable rate, regulators and payers become the gatekeepers. Regulators, starting in the late 1980s/early 1990s, and, more recently, payers have implemented a variety of early-access pathways or initiatives, and this practice is continuing even today. This article describes the specific approaches that have been taken in four economically developed regions, reviews their success rates, and suggests possible new directions.

The contribution of health technology assessment, health needs assessment, and health impact assessment to the assessment and translation of technologies in the field of public health genomics.

The European Union has named genomics as one of the promising research fields for the development of new health technologies. Major concerns with regard to these fields are, on the one hand, the rather slow and limited translation of new knowledge and, on the other hand, missing insights into the impact on public health and health care practice of those technologies that are actually introduced. This paper aims to give an overview of the major assessment instruments in public health [health technology assessment (HTA), health needs assessment (HNA) and health impact assessment (HIA)] which could contribute to the systematic translation and assessment of genomic health applications by focussing at population level and on public health policy making. It is shown to what extent HTA, HNA and HIA contribute to translational research by using the continuum of translational research (T1-T4) in genomic medicine as an analytic framework. The selected assessment methodologies predominantly cover 2 to 4 phases within the T1-T4 system. HTA delivers the most complete set of methodologies when assessing health applications. HNA can be used to prioritize areas where genomic health applications are needed or to identify infrastructural needs. HIA delivers information on the impact of technologies in a wider scope and promotes informed decision making. HTA, HNA and HIA provide a partly overlapping and partly unique set of methodologies and infrastructure for the translation and assessment of genomic health applications. They are broad in scope and go beyond the continuum of T1-T4 translational research regarding policy translation.

Toward a sustainable European Network for Health Technology Assessment. The EUnetHTA project.

EUnetHTA is a recently initiated EU network aiming at connecting national HTA agencies, research institutions, and health ministries to enable an effective exchange of information and to lend support to health policy decisions by the Member States. The article briefly discusses the policy background, the specific objectives, and the project structure of the network.

Does health technology assessment benefit health services and politics? The experiences of an established HTA institution: the Danish Centre for Evaluation and HTA.

Health technology assessment (HTA) was introduced in Denmark 20 years ago. However, it only came into fashion a few years ago. This happened when politicians and health service decision-makers realized that due to the increasing pressure on resources prioritization was an inescapable fact. HTA was supposed to support this effort by providing a broad spectrum of information designed for decision-making. Events speeded up from that point on: a national HTA strategy, a national HTA institution, satellite institutions, and many HTA projects were set up - at national, regional, and local levels. The diversity and decentralization of decision-making combined with a broad and interdisciplinary approach to methodology guided the development of Danish HTA. Experiences with HTA were gained from successful applications and disappointing encounters with uncontrollable political processes. Politicians seem in general to be content with the development. An evaluation of the impact of HTA has not yet been undertaken, and a good deal of work lies ahead. The implementation of HTA results will be one of the greatest challenges of the years ahead.

[Not Available]. / Hilft HTA dem Gesundheitswesen und der Gesundheitspolitik? Erfahrungen einer etablierten HTA-Institution - das dänische Zentrum für Evaluation und HTA : Erfahrungen einer etablierten HTA-Institution - das dänische Zentrum für Evaluation und HTA.

Health technology assessment (HTA) has been established in Denmark 20 years ago. However, it only came into fashion a few years ago. This happened when politicians and health service decision-makers realised that due to the increasing limitation of resources prioritization was an inescapable fact. HTA was supposed to support this effort by providing a broad spectrum of information designed for decision-making. Events speeded up from that point on: a national HTA strategy, a national HTA institution, satellite institutions and many HTA-projects were set up · at national, regional and local levels. The diversity and decentralisation of decision-making combined with a broad and interdisciplinary approach to methodology guided the development of danish HTA. Experiences with HTA were gained from successful applications and disappointing encounters with uncontrollable political processes. Politicians seem, in general, to be satisfied with the achievements. An evaluation of the impact of HTA has not yet been undertaken and a good deal of work lies ahead. The implementation of HTA-results will be one of the greatest challenges of the years ahead.

Health technology assessment in Denmark.

The Danish healthcare services are mainly provided by public sector institutions. The system is highly decentralized. The state has little direct influence on the provision of healthcare services. State influence is exercised through legislation and budget allocations. The main task of the state is to initiate, coordinate, and advise. Counties, which run the hospitals, also decide on the placement of services. The hospital sector is controlled within the framework of legislation and global budgets. General practitioners occupy a central position in the Danish healthcare sector, acting as gatekeepers to the rest of the system. The system works well, and its structure has resulted in steady costs of health care for a long period. There is no regulatory mechanism in the Danish health services requiring use of health technology assessment (HTA) as a basis for policy decisions, planning, or administrative procedures. However, since the late 1970s a number of comprehensive assessments of health technology have formed the basis for national health policy decisions. In 1997, after years of public criticism of the quality of hospital care and health technologies, and on the basis of a previously developed national HTA strategy, a national institute for HTA (DIHTA) was established. There seems to be a growing awareness of evidence-based healthcare among health professionals and a general acceptance of health economic analyses as a basis for health policy decision making. This progress is coupled with growing regional HTA activity in the health services. HTA seems to have a bright future in Denmark.

The concept of health technology assessment. Views of applicants to funding of HTA projects.

OBJECTIVE: The purpose of this study is to analyze the perception of the content of health technology assessment (HTA) among health professionals applying for a state grant of DKK 10 million. METHODS: A total of 113 applications were received and analyzed. When conducting the analysis, it was assumed that the applicants' maximum five-page project description would reflect: a) the applicants' perception of what an HTA is; b) how the assessment was to be conducted; and c) what the results were going to be used for. RESULTS: More than 40% of all applications focused on treatment; in 51% only one or two professional groups were to be involved (thus interdisciplinarity was questionable); only 22% of the HTA cases were intended to form the basis for political/administrative decisions; in general, the HTAs were planned far less comprehensively than was relevant; 76% of the projects did not include a formal synthesis phase; 41% intended to use diffusion as the only method for publication of the HTA result. CONCLUSIONS: The analysis reveals several areas where DIHTA has to make an effort in order to secure that HTA in fact constitutes a comprehensive and well-documented basis for decision making. These areas concern the following topics: multidisciplinarity, the objective of HTA, comprehensiveness, the synthesis phase, and publication and utilization of the HTA result.

[Significance of idiopathic preterm birth in relation to previous and future pregnancies]. / Betydningen af idiopatisk praeterm fødsel for tidligere og efterfølgende graviditeter.

This registry-based cohort study aimed to describe the relationship between pregnancy complications in the first and second pregnancy, focussing on idiopathic and indicated preterm birth of singleton infants in either pregnancy. The cohort consisted of all women living in Denmark with a first singleton birth in 1982 and a second in the period 1982-1987 (13,967 women). The risk of a second preterm birth was not significantly different between women who had an idiopathic or an indicated first preterm birth (15.2 and 12.8% respectively). Adjustment by logistic regression analysis for other risk factors for preterm birth did not influence the relative risk (6.0 before 32 weeks and 4.8 between 32 and 36 weeks) of a second preterm birth subsequent to a first one. Women with idiopathic preterm delivery in their first or second pregnancies give birth to infants with lower birth weight in previous or subsequent pregnancies. Emergency cesarean section in a first term pregnancy was a risk factor for subsequent idiopathic preterm birth.

[Idiopathic preterm deliveries in Denmark]. / Idiopatiske praeterme fødsler i Danmark.

The objective of the study was to characterize and quantify various demographic factors in idiopathic preterm delivery. All women with a permanent address in Denmark with singleton pregnancies who gave birth to an infant in 1982 (n = 51,851) are included. The material was obtained by a linkage of the Medical Birth Register and the National Register of Hospital Discharges, using the personal identification number. The incidence of singleton preterm delivery was 4.5% (n = 2330) of which 67% (n = 1557) were idiopathic. Neonatal mortality rates were significantly lower in idiopathic compared to indicated preterm birth. Following stepwise logistic regression analysis, maternal age below 20 (adjusted odds ratio 1.63, 95% confidence interval (CI) 1.07-2.47; p < 0.03), maternal age above 30 (adjusted odds ratio 0.74, 95% CI 0.60-0.90; p < 0.004) and being married (adjusted odds ratio 0.63, 95% CI 0.43-0.94; p < 0.03) correlated with idiopathic preterm birth. It is concluded that idiopathic preterm birth is more common in single, young women and associated with lower neonatal mortality rate than indicated preterm birth.

Validation of the Danish Birth Registration.

We assessed validity and reliability of data on four serious pregnancy complications and gestational age in two national registers, the Medical Birth Register (MBR) and the National Register of Hospital Discharges (NRHD). From a cohort of all women in Denmark who gave birth to their first and second singleton infant in 1982-1987, a review was made of a selected sample of 1662 medical records. Regarding registration of pregnancy complications, there was good agreement (kappa above 0.6) between medical records and the registers, and between the registers. However, there was a tendency toward understatement evidenced by low sensitivity of three of four pregnancy complications. The level of agreement (43%) for length of gestation was disappointing. The number of systematic and nonsystematic errors indicate that there was about 52% more singleton preterm deliveries in Denmark in 1982 than previously reported (6.9% instead of 4.5%). It is concluded that the validity of the Danish birth registers should be improved by explicit definitions, increased use of raw data, and data collection by motivated professionals at birth.

Implications of idiopathic preterm delivery for previous and subsequent pregnancies.

OBJECTIVE: To describe the relationship between pregnancy complications and fetal outcome in first and second pregnancies, focusing on idiopathic and indicated preterm birth of singleton infants in either pregnancy. METHODS: Included in the study were 13,967 women in Denmark who gave birth to their first singleton infant in 1982 and a second infant in 1982-1987. Information on pregnancy and birth was obtained by linking the National Medical Birth Register and the National Register of Hospital Discharges, based on personal identification numbers. RESULTS: The risk of a preterm second birth in women with idiopathic and indicated preterm first birth did not differ significantly (15.2 and 12.8%, respectively). However, women with idiopathic preterm birth in the first pregnancy tended to repeat idiopathic preterm birth twice as often as women with indicated preterm birth repeated indicated preterm birth (11.3 versus 6.4%). Adjustment by logistic regression analysis for other risk factors for preterm birth did not influence the relative risk (6.0 before 32 weeks and 4.8 for 32-36 weeks) of a second preterm birth after a first preterm birth. Women with idiopathic preterm delivery in their first and second pregnancies gave birth to infants with lower birth weight than in previous or subsequent pregnancies. Emergency cesarean delivery in a first term pregnancy was a risk factor for subsequent idiopathic preterm birth. CONCLUSION: Idiopathic preterm birth is associated with emergency cesarean delivery at term in previous pregnancies and infants with lower birth weight in previous and subsequent pregnancies.

Idiopathic preterm deliveries in Denmark.

OBJECTIVE: To characterize and quantify various demographic factors in idiopathic preterm delivery. METHODS: All women with a permanent address in Denmark and a singleton pregnancy who gave birth to a preterm infant in 1982 (N = 51,851) were included. The material was obtained by a linkage of the Medical Birth Register and the National Register of Hospital Discharges, using personal identification numbers. RESULTS: The incidence of singleton preterm delivery was 4.5% (N = 2330), of which 67% (N = 1557) were idiopathic. The neonatal mortality rate was significantly lower with idiopathic than with indicated preterm birth. Following stepwise logistic regression analysis, age under 20 (adjusted odds ratio [OR] 1.63, 95% confidence interval [CI] 1.07-2.47; P < .03), age above 30 (adjusted OR 0.74, 95% CI 0.60-0.90; P < .004) and being married (adjusted OR 0.63, 95% CI 0.43-0.94; P < .03) correlated with idiopathic preterm birth. CONCLUSION: Idiopathic preterm birth is more common in single, young women and is associated with a lower neonatal mortality rate than indicated preterm birth.

Physical examinations and laboratory tests in antenatal care visits in Denmark. Do reported practice and current official guidelines concord with results of literature reviews? A nationwide study of the public scheme of shared antenatal care in general practice, centres of midwifery and hospital outpatients' clinics.

OBJECTIVE: To analyse physical examinations and laboratory tests reported in antenatal care visits in relation to official guidelines and reviews of appropriateness. DESIGN: A nationwide cross sectional study based on questionnaires completed by general practitioners (GPs), midwives, and hospital doctors. Physical examinations and laboratory tests in connection with one specific visit were reported. SUBJECTS: The questionnaires were completed by 722 GPs (61% of eligible from a random sample), 584 midwives (86% of eligible), 250 hospital doctors who made health examinations in pregnancy, week 16-18 (63% of eligible), and 181 hospital doctors who saw women with at-risk pregnancies (55% of eligible). RESULTS: General practice: weight, blood pressure (BP) measurement, and test for proteinuria were reported in more than 90% of visits. Urine culture was reported in 46%, and cervical smear in 41% of first visits. Rubella antibody test at the first visit was only reported in 23% of nulliparae. Vaginal examination was reported in 95% of first visits. Fewer were reported in second (27%) and third (48%) routine visits during pregnancy. Midwives: checks of BP, oedema, and proteinuria were reported in more than 95% of visits irrespective of week of gestation. Vaginal examination was reported in about a third of checkups. Hospitals: vaginal examination was reported in 66% of checkups in at-risk pregnancies. CONCLUSIONS: A surplus of resources were spent on (repeat) examinations and tests with little or no documented benefit. Cervical cytology was grossly overused. Urine culture and rubella serology were not sufficiently applied.