[Rare diseases in professional health care]. / Seltene Erkrankungen in professionellen Versorgungssystemen.

The treatment of patients with rare diseases is a challenge for all those involved, since it frequently requires decisions on the basis of uncertainty. Uncertainty is of particular importance for the risk/benefit assessment of therapeutic interventions which "normally" imposes extensive requirements on the quality of evidence through explicit specifications. These requirements concern the assessment of potential benefit within the framework of statutory health insurance as well as the proof of efficacy and safety within the framework of pharmaceutical law. As a consequence drugs prescribed to patients with rare diseases are often applied outside approved indications, with immediate effects regarding reimbursement and drug safety. Another concern is that patients with rare diseases may be deprived of potential therapies as the requirements for clinical evidence are not fulfilled because of the rareness of the disease. The Federal Social Court takes this into consideration, but at the same time imposes strict requirements for the proof of efficacy. In contrast, the procedure of the Federal Joint Committee appears more adequate for the special requirements of rare diseases. Using some decisions of the Federal Social Court and of the Federal Court of Justice as examples, it is discussed how the uncertainty associated with the treatment of patients with rare diseases is dealt with and how this might be improved are discussed.

[HTA in the decision-making processes of health care institutions. Current state and relevant questions of regulatory health law]. / HTA in den Entscheidungsprozessen des IQWiG und G-BA. Bestandsaufnahme und aktuelle Fragen der gesundheitsrechtlichen Regulierung.

The development of the German HTA system and the corresponding HTA law began in 2000 and was concluded for the time being with the coming into force of the law on the modernisation of statutory health insurance on 1 January 2004. This law has established the Federal Joint Committee (G-BA) and the Institute for Quality and Efficiency in Health Care (IQWiG) as "new institutions" of statutory health insurance, restructured the procedures for the assessment of health services and formulated more precise assessment criteria than hitherto provided. There are other institutions in the health care system concerned with HTA which are not dealt with here.

Concentrations of faecal immunoreactive progestagen metabolites during the oestrous cycle and pregnancy in the black rhinoceros (Diceros bicornis michaeli).

The analysis of reproductive hormones in faecal samples is a possibility for non-invasive monitoring of reproductive status in free-ranging or intractable species. In the present study, faecal samples from three black rhinoceroses (Diceros bicornis michaeli) were collected at about weekly intervals during oestrous cycles and pregnancy. Daily samples were taken during the week after parturition. Total immunoreactive progestagens in faecal extracts were analysed with three different enzymeimmunoassays (EIA) that had considerable specificity for progestagens containing either a 20 alpha-hydroxyl or a 20-keto group. With each EIA it was possible to distinguish between the follicular and luteal phases of the oestrous cycle. Mating corresponded with low concentrations of faecal progestagens. Samples from five and six consecutive cycles were available from two rhinoceroses and cycle lengths of 24 and 26.5 days were calculated. All three animals became pregnant and the duration of gestation ranged from 440 to 470 days. After fertilization, the concentration of progestagens increased continuously, as in the luteal phase, reaching values 5-10 times higher between days 60 and 250. During the two weeks before parturition faecal progestagens declined and within 3-4 days post partum had reached follicular phase values. It was concluded that several immunoreactive progestagens are present in the faeces of black rhinoceroses and that their measurement with EIA enables non-invasive monitoring of the oestrous cycle and pregnancy.

Laser spectrophotometer for measuring differential cross sections of biological particles.

A tunable laser spectrophotometer is described that can accurately measure the differential scattering cross sections of small particle suspensions in the visible spectral range. The instrument was calibrated using a suspension of latex spheres of known size and index of refraction. By increasing the intensity of the illuminating laser, it should be possible to study differential scattering processes of individual particles on a microsecond time scale.