Search results for "Orphan drug"
showing 9 items of 9 documents
Trends in individual reimbursement of orphan drugs in Latvia in 2008–2011
2014
Orphan drugs (ODs) are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases affecting less than 5 in 10 000 people in the European Union (EU). These drugs are called “orphans” because the pharmaceutical industry has little interest, under normal market conditions, in developing and marketing products intended for only a small number of patients suffering from very rare conditions. Because of the small market, ODs are often very expensive. Whereas decisions surrounding orphan designation and marketing authorization of ODs are taken at the EU level, decisions governing pricing and reimbursement of ODs are a member state responsibility…
Impact of orphan drugs on Latvian budget
2016
Background Number of orphan medicinal products on the market and number of rare disease patients, taking these usually expensive products, are increasing. As a result, budget impact of orphan drugs is growing. This factor, along with the cost-effectiveness of orphan drugs, is often considered in the reimbursement decisions, directly affecting accessibility of rare disease therapies. The current study aims to assess the budget impact of orphan drugs in Latvia. Methods Our study covered a 5-year period, from 2010 to 2014. Impact of orphan drugs on Latvian budget was estimated from the National Health Service’s perspective. It was calculated in absolute values and relative to total pharmaceuti…
Adjuncts for ovarian stimulation: when do we adopt “orphan indications” for approved drugs?
2009
Several drugs, shown to be safe for other uses, have proven to be highly effective adjuncts for ovarian stimulation. The authors evaluate these "orphan" indications and make recommendations so that more patients will benefit from their use.
Consensus report from the 9th International Forum for Liver Magnetic Resonance Imaging: applications of gadoxetic acid-enhanced imaging
2021
Abstract Objectives The 9th International Forum for Liver Magnetic Resonance Imaging (MRI) was held in Singapore in September 2019, bringing together radiologists and allied specialists to discuss the latest developments in and formulate consensus statements for liver MRI, including the applications of gadoxetic acid–enhanced imaging. Methods As at previous Liver Forums, the meeting was held over 2 days. Presentations by the faculty on days 1 and 2 and breakout group discussions on day 1 were followed by delegate voting on consensus statements presented on day 2. Presentations and discussions centered on two main meeting themes relating to the use of gadoxetic acid–enhanced MRI in primary l…
The genomic landscape of the Ewing Sarcoma family of tumors reveals recurrent STAG2 mutation.
2014
The Ewing sarcoma family of tumors (EFT) is a group of highly malignant small round blue cell tumors occurring in children and young adults. We report here the largest genomic survey to date of 101 EFT (65 tumors and 36 cell lines). Using a combination of whole genome sequencing and targeted sequencing approaches, we discover that EFT has a very low mutational burden (0.15 mutations/Mb) but frequent deleterious mutations in the cohesin complex subunit STAG2 (21.5% tumors, 44.4% cell lines), homozygous deletion of CDKN2A (13.8% and 50%) and mutations of TP53 (6.2% and 71.9%). We additionally note an increased prevalence of the BRCA2 K3326X polymorphism in EFT patient samples (7.3%) compared …
Rufinamide in children and adults with Lennox-Gastaut syndrome: first Italian multicenter experience
2010
This is the first multicenter Italian experience with rufinamide as an adjunctive drug in children, adolescents and adults with Lennox-Gastaut syndrome. The patients were enrolled in a prospective, add-on, open-label treatment study from 11 Italian centers for children and adolescent epilepsy care. Forty-three patients (26 males, 17 females), aged between 4 and 34 years (mean 15.9 ± 7.3, median 15.0), were treated with rufinamide for a mean period of 12.3 months (range 3-21 months). Twenty patients were diagnosed as cryptogenic and 23 as symptomatic. Rufinamide was added to the baseline therapy at the starting dose of 10mg/kg body weight, evenly divided in two daily doses and then increased…
PHP7 Orphan Drugs for Rare Diseases in the European Union: Bridging the Gap between Drug Development and Unmet Medical Needs?
2011
Rare diseases and orphan drugs: Latvian story
2014
Background Ten years have passed since Latvia became a Member State of the EU in 2004. As a result European regulations, including those related to rare diseases and orphan drugs, have been applied to Latvian legislative system. Orphan diseases have been recognized as a priority area for action in the public health system, though there are significant differences in the national healthcare services for rare diseases among the EU States. This study aims to determine situation in the field of rare diseases in Latvia and compare it with other European countries. Methods We used the national plan for rare diseases, EUCERD reports, Orphanet data, Latvian and European regulations, publicly availa…
Clinical research focused in European adult women with minority diseases: Do we try hard enough?
2021
We analyzed the European countries participation in clinical trials addressing to new drug development focused on rare diseases in women comparing to more prevalent diseases as breast cancer. Participation was not associated with type of healthcare system neither socio-economic features, but it was associated with population size. Protocol ratios focused on breast cancer vs. orphan drugs and rare diseases was 15:1 and 9:1, respectively, mainly focused on ovarian cancer. Protocol number was insufficient to evaluate the success of Regulation (EC) 141/2000, it is necessary to increase the scientific quality and the number of really new molecules.