Suchergebnisse

BACKGROUND: Today up to 23 nanomedicines are approved, and approximately 50 are in clinical development. In the past, first follow-on products also referred to as nanosimilars have entered the European market through the generic approval pathway. Significant differences have been observed in clinical practice raising doubt about their therapeutic equivalence. Today, leading regulatory authorities such as the FDA and EMA as well as the regulatory science community are aware of these challenges and discuss regulatory requirements. Particularly, demonstration of pharmaceutical equivalence and bioequivalence – prerequisites for generic approval according to Article 10(1) – is extremely difficult if not impossible. While nanomedicines share lots of communalities such as heterogeneity, complexity and the large molecular size with biologics, they are synthetic products and therefore, not eligible for article 10(4) biosimilar application either. PURPOSE: Here we calculated the potential cost savings that can emerge from biosimilar-like pathways for nanosimilars that would provide regulatory clarity. MATERIAL AND METHODS: The estimation of potential savings on healthcare expenditure is based on a model of the forecasted year 2020 costs of the nanomedicines and the average decrease in price for biologics observed after the introduction of biosimilars. The model was applied to markets in five different EU countries (France, Germany, Italy, Spain and the UK, combined as EU-5) and in the US. RESULTS: The predicted saving potentials for the EU-5 and the US in year 2020 are €280 million and $2 billion, respectively. In 2023, after expiry of the patents for ferric carboxymaltose and paclitaxel, additional savings of €84 million and $233 million can be expected. CONCLUSION: The biosimilar legislation that has successfully facilitated patient access to safe and cost-effective medicine could serve as a model for a yet-establish nanosimilar approval pathway. This pathway could provide a substantial saving potential to the ...

BACKGROUND: Orphan drugs (ODs) are designed to treat rare diseases (RD), which are those affecting a small number of people (prevalence <1/2000 inhabitants). PURPOSE: To assess the economic impact of drugs used to treat RD in a hospital outpatient paediatric pharmacy (HOPP) and a paediatric day hospital (PDH). MATERIAL AND METHODS: Observational, retrospective, transversal study conducted at a third-level maternal and child University Hospital during 2016. All paediatric patients(<18 years' old) were included, as well as adults with cystic fibrosis. Chemotherapy treatments administered in the Oncology Day Hospital were excluded. Pharmaceutical drug, active ingredient, number of packages, real unit cost, consumption data, medical service and treating unit were collected from pharmacy-dispensing software(Silicon(®)) The Orphanet(®) database was used to classify the drugs in ODs (recognised as orphan by the European Union or abroad) or drugs without orphan designation. RESULTS: Four hundred and ten drugs were identified to treat RD and 51 (12.4%) were ODs: 42 were dispensed in the HOPP and nine were administered in the PDH. There were 2442 patients who received at least one drug to treat RD: 2044 from the HOPP and 562 from the PDH (164 patients received treatment in both settings) Of all patients, 441 (18.1%) received at least one OD: 420 (20.5%) in the HOPP and 23 (4.1%) in the PDH (two patients in both). Drugs used to treat RDs accounted for €7.7 million: €3.8 million in the HOPP and €3.9 million in the PDH. OD cost represented 36.3% of the total pharmaceutical expenditure in drugs at the HOPP and 71% at the PDH. Bosentan, adalimumab, ivacaftor, ataluren and sildenafil were the five drugs with the greatest economic impact in the HOP budget and eculizumab, idursulfase, elosulfase, galsulfase and velaglucerase in the PDH budget. CONCLUSION: Pharmacological treatment with ODs has a great impact on direct medical costs, involving more than 50% of total pharmaceutical expenditure. Although it is more common in ...

BACKGROUND: The European Medicines Agency (EMA) is responsible for the scientific evaluation of medicines for use in the European Union (EU), providing a scientific opinion on the granting of EU-wide marketing authorisations. PURPOSE: We conducted a retrospective analysis of new active substances (NAS) that had received positive opinion by the Committee for Medicinal Products for Human Use (CHMP) of the EMA, as well as the authorisation conditions and the findings of available therapeutic positioning reports (IPT) published in the Spanish Agency for Medicines and Health Products (AEMPS). MATERIAL AND METHODS: We analysed NAS with positive opinion from January 2014 until September 2017. We collected of each NAS the therapeutic area, the type of approval, the route used,, the designation obtained and pivotal trials (according to their design features using the European Public Assessment Reports) in order to evaluate the evidence provided. On the other hand, we verified the existence of IPT and analysed its conclusions, as well as the financing conditions. When an IPT included separate analyses for different indications, we included each separately. RESULTS: In this period, the CHMP approved the use of 132 NAS (41:2014, 39:2015, 27:2016, 25 until September 2017). Of these, 11% were conditional approvals (3:2014, 3:2015, 8:2016), and four exceptional, mainly in the cancer area. Moreover, 15% of approvals were carried out by accelerated routes due mostly also to the cancer area. Regarding the orphan designation, 32% were authorised under this (14:2014, 11:2015, 10:2016, 7:2017). Concerning the pivotal trials, 27% of NAS had at least one Phase-II trial between them. Of the 70 existing IPT, only 27% were classified as an advance in therapeutics. The majority were classified as similar to alternatives, considering its use based on efficiency criteria. Twenty-four per cent were not financed or under conditions by the AEMPS. CONCLUSION: The number of marketing approvals has been a continuing downward trend. However, ...

The immense problems created by the indiscriminate laying of mines are now beginning to be recognized. ICRC surgeons have for some time reported that some of the worst injuries they see are caused by mines, and it was our Medical Division that decided it was essential to study this problem thoroughly from all relevant angles. As a result, in April 1993 the ICRC organized a multidisciplinary symposium on antipersonnel mines. The symposium gathered information on the present use of mines and the humanitarian and social consequences of such use, the trade in mines, the technical characteristics of mines, mine clearance, the professional military use of mines and the legal situation. The symposium was attended not only by specialists in these fields, but also by a representative of the International Federation of Red Cross and Red Crescent Societies, a number of non-governmental organizations that had begun to take a serious interest in the problem and some journalists. As a result, there is now more information available on the various problems caused by mines. More important, the efforts undertaken by interested persons to draw the attention of governments to the need for action have begun to bear fruit: several governments have declared a moratorium on their mines exports, the United Nations has just adopted a resolution setting up a fund for demining operations, and possible further restrictions on the use of mines will be discussed in 1994 during a United Nations conference which will review a treaty on the use of conventional weapons.