Birkmeyer J.D.,University of Michigan |
Reames B.N.,University of Michigan |
Carr A.J.,University of Oxford |
Campbell W.B.,National Institute for Health and Care Excellence (NICE) |
Wennberg J.E.,Dartmouth College
The Lancet | Year: 2013
The use of common surgical procedures varies widely across regions. Differences in illness burden, diagnostic practices, and patient attitudes about medical intervention explain only a small degree of regional variation in surgery rates. Evidence suggests that surgical variation results mainly from differences in physician beliefs about the indications for surgery, and the extent to which patient preferences are incorporated into treatment decisions. These two components of clinical decision making help to explain the so-called surgical signatures of specific procedures, and why some consistently vary more than others. Variation in clinical decision making is, in turn, affected by broad environmental factors, including technology diffusion, supply of specialists, local training frameworks, financial incentives, and regulatory factors, which vary across countries. Better scientific evidence about the comparative effectiveness of surgical and non-surgical interventions could help to mitigate regional variation, but broader dissemination of shared decision aids will be essential to reduce variation in preference-sensitive disorders.
News Article | February 16, 2017
Pregnant women are almost twice as likely to quit smoking if they are supported from their first midwife appointment - and then are more likely to have heavier, healthier babies. Newcastle University researchers evaluated the "BabyClear" programme which follows the National Institute for Health and Care Excellence (NICE) guidance around smoking in pregnancy by screening all pregnant women for smoking using carbon monoxide monitoring. Any woman still smoking when she first saw a midwife, at around eight weeks into pregnancy, was given information about the risks to their unborn baby. She was then put in contact with agencies who could help her quit. Publishing today in Tobacco Control, the research team report that in the study of 40,000 mothers-to-be, the number of women helped to stop smoking almost doubled. Dr Ruth Bell, senior lecturer in the Institute of Health and Society at Newcastle University, who led the study, said: "This shows that if we help make it routine for midwives to ask about smoking and screen every mother-to-be for carbon monoxide, we can double the number of women who quit smoking in pregnancy and substantially increase the number of healthy babies." The team found the women who did not smoke in pregnancy, went on to have babies that were more than half-a-pound heavier (260 grams or 0.57lb) at full term, than those who continued to smoke. Even women who quit smoking during their pregnancy were shown to have heavier babies (by around 210 grams or 0.46lb) than those of mothers who smoked throughout pregnancy. This is an indicator of a healthier infant that is likely to need less medical help. The large study in NHS maternity units across the north east of England, included 10, 594 women who were smokers. Every woman was offered carbon monoxide (CO) monitoring and anyone with high carbon monoxide readings, indicating smoking, was routinely referred to Stop Smoking Services within 24 hours, where they received help to stop from a trained smoking cessation adviser. Dr Bell said: "Training midwives particularly on how to start a conversation about smoking, along with providing clear routes for women to get support to help stop smoking, ensured that national guidance was followed. This package proved to be highly effective in helping mothers to stop smoking, equivalent to nearly 100 extra quitters per year in a maternity unit delivering 3,000 babies." The study was funded by the NIHR School for Public Health Research (SPHR) and Fuse, the Centre for Translational Research in Public Health. The study examined the records of 37,726 births of single babies across the North East, including 10,594 to mothers who smoked during pregnancy. The authors report that the referral rate more than doubled (2.5 times higher) across all the trusts in the first three months after the introduction of the new approach. The introduction of the programmes was associated with a substantial increase in quitting during pregnancy, and quitting was associated with referral to stop smoking services. Further analysis of birthweight revealed that babies were significantly heavier if a mother quit smoking during pregnancy. Professor Eugene Milne, a co-author of the study and Director of Public Health at Newcastle City Council, said: "It is hard to overstate the impact that being smoke free can have on babies, mothers and families. "Reducing smoking reduces the risk of stillbirth, low birth weight, and of problems with health not only in childhood but into adult life and even old age. There is clear evidence that heavier babies have lower rates of heart disease in later life. "This new study provides important evidence that giving the right care to help pregnant mothers quit smoking is an effective and important intervention, and provides the information and help that mothers want. "We are working to ensure that all prospective mothers receive this standard of care and support in Newcastle and the North East." Economic analysis revealed that the additional cost of the intervention per delivery was £31. The work was possible as the North East has a regional approach to reducing smoking during pregnancy, embedding NICE guidance using the BabyClear approach developed by Improving Performance in Practice (iPiP). This was rolled out in the North East by Fresh, the North East's regional tobacco control programme, working with all of the North East's hospital foundation trusts and Stop Smoking Services. Smoking at the time of delivery rates have fallen in the North East since 2009/10 when 22.2% of women smoked at the time they gave birth, down to 16% in 2016. Ailsa Rutter, Director of Fresh, which supported the introduction of the approach across the region said: "These findings and the drastic decline in smoking in pregnancy in the North East by around a third since 2009 shows the vital role the NHS can play in reducing harm from tobacco. But it is vital this work continues. "There is a lack of awareness around just how smoking harms unborn babies and midwives played an important role in communicating this. Women overwhelmingly supported this approach, even if some initially found the facts shocking. "There has also been demand for this approach to be rolled out nationally by leads from the national Smoking in Pregnancy Challenge Group, a coalition working to reduce the harm caused by tobacco." Professor Linda Bauld, Co-Chair of the Smoking in Pregnancy Challenge Group and Professor of Health Policy at the University of Stirling said: "The North East of England has taken a lead in implementing recommendations from NICE and the Smoking in Pregnancy Challenge group, and progress achieved is impressive. "These results show the vital role health professionals can play when they raise the issue of smoking. These changes have undoubtedly saved lives and have implications that go beyond pregnancy for the role front line NHS professionals can play." Dr Bell adds: "Our analysis shows that by improving the links between midwives and smoking cessation services we can really help mothers give their babies the best start in life." REFERENCE: Evaluation of a complex healthcare intervention to increase smoking cessation in pregnant women: interrupted time series analysis with economic evaluation. Ruth Bell et al. Tobacco Control. Doi: 10.1136/tobaccocontrol-2016-053476 Melanie Snowdon, 40, from South Shields, welcomed her fifth child, Faith, to the world in March. When she found out she was expecting she sought support from her local stop smoking service to quit tobacco for the good of her unborn child. She's continued to be smoke free following the birth and is confident that she'll remain that way in the future. She said: "I was about 17 when I started smoking. In those days everybody smoked, we knew it was bad but you don't think about that when you're a teenager. On average I smoked about 20 - 30 a day and it wasn't until I was pregnant with my fifth child that I stopped. "I smoked through my first four pregnancies and there were no issues, I had four healthy babies. There was a big gap in between my first four and my youngest. "Not long after I married my husband, we fell pregnant again. I decided that I needed to quit smoking as my husband doesn't smoke and doesn't like it. "The thing that really hit home for me was when the midwife at hospital showed me how much carbon monoxide was in my breath and explained how smoking starves the baby of oxygen. "I was referred to my local stop smoking service and, at first, I was very sceptical. I used patches, sprays and an ecig for a little bit too and I managed to come off the cigarettes completely after seven weeks. "I couldn't quite believe the difference in weight, my daughter was 9lbs 12oz whereas all my other children were much lighter. I never believed that smokers had smaller babies. "I wasn't sure how I would do after my daughter was born but I'm still off the cigarettes and I can't see myself going back. I still carry a spray in my bag just in case but the thought and the smell of smoking really puts me off. "My husband has said he is really proud of me because he thought once the baby was born I would have started again. "I would encourage other mums to give it a try - it doesn't cost anything. I smoked for so many years and would never have thought I would be able to stop but I did."
News Article | March 2, 2017
First new medication for primary biliary cholangitis in nearly 20 years Rapid NICE approval only two months after marketing authorization in the EU; one of the fastest approvals to date for an orphan medicine NEW YORK, March 02, 2017 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept) today announced that the National Institute for Health and Care Excellence (NICE) has approved Ocaliva (obeticholic acid) for routine use by the National Health Service (NHS) in England, Wales and Northern Ireland. Ocaliva has been conditionally approved in the European Union for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA. The NHS is expected to make Ocaliva available to patients with PBC within 90 days of NICE’s final appraisal publication and Intercept will work with local reimbursement authorities to help ensure eligible patients obtain access. Although it is a rare disease, PBC is a leading cause of liver transplantation in adult women in the UK. Ocaliva is a new treatment option for patients with PBC who do not fully respond to, or are intolerant to, current treatment and remain at risk of their disease progressing toward cirrhosis, liver transplantation or death. “I am excited to see that the substantial group of PBC patients who are not achieving treatment goals with UDCA alone or who cannot tolerate UDCA will soon be able to access the first new therapeutic option in nearly 20 years. This truly is good news for our PBC patients,” said David Jones, M.D., Ph.D., Professor of Liver Immunology at Newcastle University and Consultant Hepatologist at Newcastle upon Tyne Hospitals Trust, which hosts one of Europe's leading clinical services in the disease. “The development of new treatments for PBC is a powerful example of the medical innovation that can occur when government, industry, academia, community clinicians and, most importantly, patients come together to address an unmet need.” Ocaliva is a potent and selective agonist of the farnesoid X receptor (FXR), which is expressed at high levels in the liver and intestine and thought to be a key regulator of bile acid, inflammatory, fibrotic and metabolic pathways. In December 2016, Ocaliva received conditional marketing authorization in Europe based on efficacy and safety data derived from three randomized double-blind, placebo-controlled clinical trials evaluating the effect of Ocaliva on alkaline phosphatase (ALP) and bilirubin in patients with PBC. The marketing authorization was also supported by two clinical databases that include more than 10,000 patients from the Global PBC Study Group and UK-PBC Group, both independently confirming that achieving lower ALP and/or bilirubin levels is significantly correlated with increased transplant-free survival. The most commonly reported adverse reactions were pruritus (63%) and fatigue (22%). Adverse reactions leading to discontinuation were 1% in the Ocaliva titration arm and 11% in the Ocaliva 10 mg arm. The most common adverse reaction leading to discontinuation was pruritus. The majority of pruritus occurred within the first month of treatment and tended to resolve over time with continued dosing. “This very rapid decision by NICE, one of the fastest approvals to date for an orphan medication, is an important affirmation of the scientific innovation, clinical value and cost-effectiveness of Ocaliva by one of the most respected health technology assessment bodies,” said Lisa Bright, Intercept’s President, International. “We welcome NICE’s decision to provide broad access to Ocaliva and we owe a tremendous debt to people living with PBC and the clinical groups who helped us to achieve this milestone for the PBC community.” “It is exciting news for PBC patients that this new treatment option will now be routinely available in England, Wales and Northern Ireland,” said Collette Thain MBE, CEO of The PBC Foundation. “When I was diagnosed with PBC, UDCA was the only approved treatment option and PBC wasn’t a major priority for many researchers. Thankfully, so much has changed for people living with PBC since then. After decades of advocacy from the PBC community, we have a new treatment option, a growing awareness of the disease among the general public, greater expertise amongst clinicians and an acceleration of PBC research in the UK and around the globe.” Primary biliary cholangitis (PBC) is a rare, autoimmune cholestatic liver disease that puts patients at risk for life-threatening complications. PBC is primarily a disease of women, afflicting approximately one in 1,000 women over the age of 40. If left untreated, survival of PBC patients is significantly worse than the general population. The estimated prevalence of PBC in the UK is approximately 3.9 per 10,000 population, equating to approximately 19,175 people in England. Patients and medical leaders in the UK have played a critical role in accelerating PBC research, innovation and awareness globally. The patient community in the UK has guided Intercept’s efforts to improve PBC education and understand the unmet needs of patients and their families. The UK-PBC Study Group, a research consortium funded by the UK government through the Medical Research Council and National Institute for Health Research, played a critical role in the development of Ocaliva and was one of two clinical databases to independently confirm that achieving lower ALP and/or bilirubin levels is significantly correlated with increased transplant-free survival. Ocaliva (obeticholic acid) is a potent and highly selective agonist of the farnesoid X receptor (FXR), a nuclear receptor expressed in the liver and intestine. FXR is a key regulator of bile acid, inflammatory, fibrotic and metabolic pathways. In December 2016, Ocaliva received conditional marketing authorization in Europe for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA, conditional to the company providing further data post-approval to confirm benefit. In May 2016, the U.S. Food and Drug Administration granted accelerated approval to Ocaliva for the treatment of PBC. For full prescribing information in the U.S., visit Ocaliva.com. Hypersensitivity to the active substance or to any of the excipients and complete biliary obstruction. Elevations in alanine amino transferase (ALT) and aspartate aminotransferase (AST) have been observed in patients taking obeticholic acid. Clinical signs and symptoms of hepatic decompensation have also been observed. These events have occurred as early as within the first month of treatment. Liver-related adverse events have primarily been observed at doses higher than the maximum recommended dose of 10 mg once daily. Patients should be monitored during treatment with Ocaliva for elevations in liver biochemical tests and for the development of liver-related adverse events. Dosage adjustments are needed for patients with moderate (Child-Pugh Class B) or severe (Child-Pugh Class C) hepatic impairment. Severe pruritus was reported in 23% of patients treated with Ocaliva 10 mg arm, 19% of patients in the Ocaliva titration arm and 7% of patients in the placebo arms. The median time to onset of severe pruritus was 11, 158 and 75 days for patients in the Ocaliva 10 mg, Ocaliva titration and placebo arms, respectively. Management strategies include the addition of bile acid binding resins or antihistamines, dose reduction, reduced dosing frequency and/or temporary dose interruption. The most commonly reported adverse reactions were pruritus (63%) and fatigue (22%). Other common adverse reactions observed in clinical trials (> 5%) were abdominal pain and discomfort, rash, oropharyngeal pain, dizziness, constipation, arthralgia, thyroid function abnormality and eczema. Bile acid binding resins such as cholestyramine, colestipol or colesevelam adsorb and reduce bile acid absorption and may reduce efficacy of obeticholic acid. When concomitant bile acid binding resins are administered, obeticholic acid should be taken at least 4-6 hours before or 4-6 hours after taking a bile acid binding resin, or at as great an interval as possible. For detailed safety information for Ocaliva (obeticholic acid) 5 mg and 10 mg tablets including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European Summary of Product Characteristics. Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, including primary biliary cholangitis (PBC), nonalcoholic steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and biliary atresia. Founded in 2002 in New York, Intercept now has operations in the United States, Europe and Canada. Intercept’s International headquarters are located in London. For more information about Intercept, please visit www.interceptpharma.com. This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the clinical relevance and utility of ALP, bilirubin and the surrogate endpoint used in the Phase 3 POISE trial to predict clinical outcomes, the acceptance of Ocaliva® (obeticholic acid) as a treatment for PBC by healthcare providers, patients and payors, the commercial availability of OCA for the treatment of PBC and timelines related thereto, the anticipated prevalence of and other epidemiological estimates and market data related to PBC, the continued development of OCA and Intercept's other product candidates, and our strategic directives under the caption "About Intercept." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of important risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: Intercept's ability to successfully commercialize Ocaliva in PBC, and Intercept's ability to maintain its regulatory approval in jurisdictions in which Ocaliva is approved for use in PBC; the initiation, cost, timing, progress and results of Intercept's development activities, preclinical studies and clinical trials, including Intercept's development program in NASH; the timing of and Intercept's ability to obtain and maintain regulatory approval of OCA in PBC in countries outside the ones in which it is approved and in indications other than PBC and any other product candidates it may develop such as INT-767; conditions that may be imposed by regulatory authorities on Intercept's marketing approvals for its products and product candidates such as the need for clinical outcomes data (and not just results based on achievement of a surrogate endpoint), and any related restrictions, limitations, and/or warnings in the label of any approved products and product candidates; Intercept's plans to research, develop and commercialize its product candidates; Intercept's ability to obtain and maintain intellectual property protection for its products and product candidates; Intercept's ability to successfully commercialize OCA in indications other than PBC and its other product candidates; the size and growth of the markets for Intercept's products and product candidates and its ability to serve those markets; the rate and degree of market acceptance of any of Intercept's products, which may be affected by the reimbursement that it may receive for its products from payors; the success of competing drugs that are or become available; the election by Intercept's collaborators to pursue research, development and commercialization activities; Intercept's ability to attract collaborators with development, regulatory and commercialization expertise; regulatory developments in the United States and other countries; the performance of third-party suppliers and manufacturers; Intercept's need for and ability to obtain additional financing; Intercept's estimates regarding expenses, future revenues and capital requirements and the accuracy thereof; Intercept's use of cash, short-term investments and the proceeds from the offering; Intercept's ability to attract and retain key scientific or management personnel; and other factors discussed under the heading "Risk Factors" contained in our annual report on Form 10-K for the year ended December 31, 2016 filed on March 1, 2017 as well as any updates to these risk factors filed from time to time in our other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intercept undertakes no duty to update this information unless required by law.
News Article | March 2, 2017
Not intended for UK- or US-based media Merck, a leading science and technology company, announced today that the UK National Institute for Health and Care Excellence (NICE) has issued a positive Final Appraisal Determination (FAD) recommending the routine National Health Service (NHS) use of Erbitux® (cetuximab) in combination with either FOLFIRI or FOLFOX as a first-line treatment for patients with RAS wild-type metastatic colorectal cancer (mCRC). Erbitux has long been established and reimbursed as a first-line standard-of-care treatment option in most European countries and many other countries worldwide. "The NICE decision is great news for patients and their families in England, as it now means they will have access to this effective first-line treatment," said Maya Martinez-Davis, Global Head of Oncology Franchise for Merck's biopharma business. "Our efforts in working with NICE to reach this important milestone are part of our relentless commitment to bringing tailored, effective therapies to patients worldwide." This decision expands the previous NICE recommendation, which endorsed the use of Erbitux in combination with either FOLFOX or FOLFIRI solely for patients whose cancer had spread only to the liver (liver-limited disease). It is based on robust data from Phase III clinical studies having demonstrated that Erbitux in combination with either FOLFIRI,, or FOLFOX,,, as a first-line treatment for patients with RAS wild-type mCRC, confers significant benefit in patient outcomes. "Patients with metastatic colorectal cancer in the UK have very limited access to effective first-line treatments," said Jola Gore-Booth, Founder/CEO of the colorectal cancer patient advocacy group, EuropaColon. "We are therefore delighted with NICE's decision, as it means there is now more choice for patients in England regarding treatment options that they can benefit from." Both the European Society for Medical Oncology and the US National Comprehensive Cancer Network clinical guidelines also recommend first-line treatment with Erbitux, in combination with either FOLFOX or FOLFIRI, for patients with RAS wild-type mCRC., Erbitux has obtained marketing authorization in over 90 countries worldwide. To date, more than 480,000 patients with mCRC have been treated with Erbitux. For further information and press materials please visit http://www.merckgroup.com/media-center-oncology. All Merck Press Releases are distributed by e-mail at the same time they become available on the Merck Website. Please go to http://www.merckgroup.com/subscribe to register online, change your selection or discontinue this service. By publishing a Final Appraisal Determination (FAD), the National Institute for Health and Care Excellence (NICE) has made final recommendations on how Erbitux with FOLFOX/FOLFIRI should be used in the NHS. If there are no successful appeals, the final recommendations will be issued as NICE guidance. Approximately half of patients with mCRC have RAS wild-type tumors and half have RAS mutant tumors. Results from studies assessing RAS mutation status in patients with mCRC have shown that anti-epidermal growth factor receptor (EGFR) monoclonal antibody therapies, such as Erbitux® (cetuximab), can improve outcomes in patients with RAS wild-type mCRC.- Colorectal cancer (CRC) is the third most common cancer worldwide, with an estimated incidence of more than 1.36 million new cases annually. An estimated 694,000 deaths from CRC occur worldwide every year, accounting for 8.5% of all cancer deaths and making it the fourth most common cause of death from cancer. Almost 55% of CRC cases are diagnosed in developed regions of the world, and incidence and mortality rates are substantially higher in men than in women. Erbitux® is a highly active IgG1 monoclonal antibody targeting the epidermal growth factor receptor (EGFR). As a monoclonal antibody, the mode of action of Erbitux is distinct from standard non-selective chemotherapy treatments in that it specifically targets and binds to the EGFR. This binding inhibits the activation of the receptor and the subsequent signal-transduction pathway, which results in reducing both the invasion of normal tissues by tumor cells and the spread of tumors to new sites. It is also believed to inhibit the ability of tumor cells to repair the damage caused by chemotherapy and radiotherapy and to inhibit the formation of new blood vessels inside tumors, which appears to lead to an overall suppression of tumor growth. The most commonly reported side effect with Erbitux is an acne-like skin rash that seems to be correlated with a good response to therapy. In approximately 5% of patients, hypersensitivity reactions may occur during treatment with Erbitux; about half of these reactions are severe. Erbitux has already obtained market authorization in over 90 countries world-wide for the treatment of colorectal cancer and for the treatment of squamous cell carcinoma of the head and neck (SCCHN). Merck licensed the right to market Erbitux outside the US and Canada from ImClone LLC, a wholly-owned subsidiary of Eli Lilly and Company, in 1998. Merck has an ongoing commitment to the advancement of oncology treatment and is currently investigating novel therapies in highly targeted areas. All Merck Press Releases are distributed by e-mail at the same time they become available on the Merck Website. Please go to http://www.merckgroup.com/subscribe to register online, change your selection or discontinue this service. Merck is a leading science and technology company in healthcare, life science and performance materials. Around 50,000 employees work to further develop technologies that improve and enhance life - from biopharmaceutical therapies to treat cancer or multiple sclerosis, cutting-edge systems for scientific research and production, to liquid crystals for smartphones and LCD televisions. In 2015, Merck generated sales of Euro12.85 billion in 66 countries. Founded in 1668, Merck is the world's oldest pharmaceutical and chemical company. The founding family remains the majority owner of the publicly listed corporate group. Merck, Darmstadt, Germany holds the global rights to the Merck name and brand. The only exceptions are the United States and Canada, where the company operates as EMD Serono, MilliporeSigma and EMD Performance Materials.
News Article | February 23, 2017
EBSCO Information Services (EBSCO) has been chosen as an approved content provider for the National Institute for Health and Care Excellence (NICE) framework. The agreement allows purchasers from libraries, health care and social care-related organizations in the National Health Service (NHS) to search, order and purchase medical and healthcare-related content through GOBI®, the leading web-based acquisitions tool for finding, ordering and managing e-books and print books for libraries. GOBI is provided by GOBI® Library Solutions from EBSCO, (GOBI Library Solutions) which partners with libraries to streamline their workflows for monographic acquisitions. As a part of EBSCO, GOBI Library Solutions has an office in the United Kingdom as well as a knowledgeable customer service staff to provide support to customers. With access to more than 13 million titles, including more than one million e-books, GOBI supplies print books and e-books to thousands of medical, academic, and government libraries in more than 60 countries. In addition to offering a variety of health sciences e-books and print books in the medical, nursing and allied health fields, GOBI Library Solutions helps librarians streamline their collection development workflows by providing: GOBI Library Solutions Vice President of Sales, UK, Ireland and Nordic Countries Gareth Smith says the recognition of EBSCO as an approved NICE Framework content provider gives organizations access to high-quality health-care content from GOBI Library Solutions. “The appointment of EBSCO to the NICE Electronic and Print Content Framework Agreement helps NHS libraries and organizations that use the GOBI acquisitions tool to quickly find and acquire books they need to better manage the medical needs of patients.” For more information, please visit: gobi.ebsco.com About GOBI Library Solutions from EBSCO GOBI® Library Solutions from EBSCO, formerly YBP Library Services, offers over 13 million print and e-books to academic, research and special libraries worldwide. From streamlining workflows to partnering with library staff, GOBI Library Solutions is committed to providing the best solution for libraries’ acquisition, collection development and technical service needs. For more than 40 years, the mission has remained the same—to partner with libraries in providing access to the broadest selection of scholarly content available. For more information, visit the GOBI Library Solutions website at: gobi.ebsco.com. About EBSCO Information Services EBSCO Information Services (EBSCO) is the leading discovery service provider for libraries worldwide with more than 11,000 discovery customers in over 100 countries. EBSCO Discovery Service™ (EDS) provides each institution with a comprehensive, single search box for its entire collection, offering unparalleled relevance ranking quality and extensive customization. EBSCO is also the preeminent provider of online research content for libraries, including hundreds of research databases, historical archives, point-of-care medical reference, and corporate learning tools serving millions of end users at tens of thousands of institutions. EBSCO is the leading provider of electronic journals & books for libraries, with subscription management for more than 360,000 serials, including more than 57,000 e-journals, as well as online access to more than 900,000 e-books. For more information, visit the EBSCO website at: http://www.ebsco.com. EBSCO Information Services is a division of EBSCO Industries Inc., a family owned company since 1944. For more information, please contact: Kathleen McEvoy Vice President of Communications (800) 653-2726 ext. 2594 kmcevoy(at)ebsco(dot)com
News Article | February 27, 2017
National Institute for Health and Care Excellence (NICE) Publishes Final Guidance Covering Reimbursement of Galafold for Fabry Disease CRANBURY, N.J., Feb. 27, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, has commenced the commercial launch of the precision medicine Galafold in the United Kingdom (UK) following the final publication of reimbursement guidelines by the National Institute for Health and Care Excellence (NICE) Highly Specialised Technologies Evaluation Committee (EC). Galafold is reimbursed within the National Health Service (NHS) in England as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation. "Galafold is the first oral treatment as well as the first precision medicine now available in the UK for Fabry patients 16 years and older who have an amenable mutation,” stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “Following the final NICE publication, which we believe reflects the significant value of our oral precision medicine approach, we are pleased that all appropriate patients will now have access to Galafold. The finalization of pricing and reimbursement in the UK is a significant milestone for Amicus as we navigate the country-by-country processes to launch Galafold throughout the EU as rapidly as possible.” Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. As a precision medicine, Galafold is designed to restore alpha-Gal A activity in patients who have amenable mutations (an estimated 35% to 50% of the Fabry population). “We are delighted that Fabry patients with amenable mutations will now have the option to be prescribed an oral treatment for the management of their disease,” commented Christine Lavery, Group Chief Executive of The MPS Society, UK and President of the Fabry International Network (FIN). “Our mission is to inform and support patients and families in all aspects related to their disease, therefore having access to new therapeutic opportunities is an important consideration and opportunity in a patient’s journey.” The European Commission granted full approval for Galafold on May 30, 2016, as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease and who have an amenable mutation. About Galafold™ and Amenable Mutations Galafold™ (migalastat) is a first-in-class chaperone therapy approved in the EU as a monotherapy for Fabry disease in patients with amenable mutations. Galafold works by stabilizing the body’s own dysfunctional enzyme, so it can clear the accumulation of disease substrate in patients who have amenable mutations. A proprietary in vitro assay (Galafold Amenability Assay) was used to classify more than 800 known GLA mutations as “amenable” or “not amenable” to treatment with Galafold. The current EU label includes 313 GLA mutations that have been identified and determined to be amenable based on the Galafold Amenability Assay, which represent between 35% and 50% of the currently diagnosed Fabry population. Healthcare providers in the EU may access the website www.galafoldamenabilitytable.com to quickly and accurately identify which mutations are categorized as “amenable” or “not amenable” to Galafold. Amicus expects to submit updates to the label as additional GLA mutations are identified and tested in the Galafold Amenability Assay. Important Safety Information Treatment with GALAFOLD should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. GALAFOLD is not recommended for use in patients with a nonamenable mutation. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu. About Fabry Disease Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb ). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including the central nervous system, heart, kidneys, and skin. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease, and stroke. The symptoms can be severe, differ from patient to patient, and begin at an early age. All Fabry disease is progressive and may lead to organ damage regardless of the time of symptom onset. About Amicus Therapeutics Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, financing plans, and the projected cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe or our other product candidates if and when approved; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; and the potential that we will need additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. With respect to statements regarding projections of the Company's cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2015 and Quarterly Report on Form 10-Q for the quarter ended September 30, 2016. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.
News Article | December 1, 2016
The Telegraph and even the Guardian have big headlines claiming that the National Institute for Health and Care Excellence (NICE) is recommending that speed bumps. The Telegraph goes so far as to say “Axe speed bumps to save lives”, because evidently all that decelerating and accelerating at speed bumps causes pollution. This question is of interest not just in the UK, as people complain about speed bumps everywhere. There are websites devoted to getting rid of them, claiming that they cause pollution, damage to vehicles (especially sports cars), back injuries to passengers and of course, they slow down emergency vehicles. The Telegraph quotes a totally fair and balanced source, the Royal Automobile Club, whose spokesperson agrees: The suggestion that local authorities should think again about speed humps which cause motorists to brake and then accelerate again is an eminently sensible suggestion and has the potential to improve the quality of air locally. Nice also acknowledges the importance of improving the flow of traffic – since air quality is often at its worst where congestion appears, a point that the RAC has made on a number of occasions. Yup, get rid of bike lanes, speed bumps and everything else that they claim causes congestion if you want to get rid of air pollution. But as Carlton Reid of Bike Biz notes, the report says nothing of the kind about speed bumps. Instead the report recommends that they be designed properly and that speed limits are enforced. Because in fact, if people are driving the speed over a well-designed speed bump they don’t have to decelerate; you can drive over them smoothly. That is point of them: to control speeding. And they are far more effective and less polluting than the stop signs used to control speed in North America. But as Peter Walker, also of the Guardian notes, the Guardian article doesn’t mention their first and best advice: get people out of polluting cars. Except the report actually does; The ways to reduce air pollution and improve health are well known to urbanists and planners and are right there in the guidelines in black and white: The report makes some silly suggestions, such as planting foliage between bike lanes and cars, (as if there is room for that) and siting cycle routes on highly polluted roads (which are usually the most direct.) Their architectural suggestions are ludicrous too: Because it is pretty hard to minimize the need for motorized travel unless you build to the density that creates what they call canyons, and even harder to have living spaces all face away from the road. But in fact, the report is pretty sensible and realistic. It is also right about speed bumps: design them properly and they work as promised.
News Article | March 1, 2017
Breast cancer patients who receive the drug Herceptin for nine weeks as part of their post-surgery chemotherapy regime may enjoy better health outcomes, according to new research led by UCL, compared to those who receive it for 12 months, the period currently recommended in the English NHS. The researchers calculated that switching to the nine-week course of Herceptin could also save the NHS over £100 million a year. The study, a cost-effectiveness analysis of Herceptin, was published in the journal PLOS ONE. Breast cancer is the most common cancer in women in several countries, including the UK and US. Around a quarter of women diagnosed with early-stage breast cancer have HER2-positive tumours, meaning that their tumours have an overactive human epidermal growth factor receptor 2 (HER2) gene and are more aggressive. In these patients, adding Herceptin to the post-surgery chemotherapy regime has led to significant increases in survival rates and the amount of time before patients suffer a recurrence of the disease. However, Herceptin is expensive and can cause side effects, the most serious of which is potentially life-threatening heart problems. The drug's manufacturer Roche recommends that women take the drug for a year after surgery, based on their trial information, and this is the current recommendation in the UK. When the National Institute for Health and Care Excellence (NICE) approved the drug in 2006, the Evidence Review Group's report stated that it wished to see further evidence included on the effect of using the drug for shorter lengths of time. In 2014, there were 46,085 new breast cancer cases in women and the researchers estimated that 5,678 of these patients were eligible for Herceptin. Switching these patients to the nine-week treatment would have saved the NHS £132 million at 2014 prices. In addition, there was a gain of 4,773 quality-adjusted life-years, which are often measured in terms of the person's ability to carry out the activities of daily life, and freedom from pain and mental disturbance. This study, which was carried out with City, University of London, analysed three options -- giving Herceptin after surgery for 12 months, 9 weeks, or not at all. The researchers considered eight studies carried out mainly in Europe and the US, and were able to perform an analysis that combined the results from two of these studies, which each followed patients for five years after they received Herceptin. These two studies covered 2,149 patients who received the drug for a year, 54 who received it for nine weeks and 1,131 who weren't given Herceptin. The scientists looked at three outcomes in the five-year period - overall survival rates, the rates of secondary cancer and whether any patients died of a heart attack. The analysis involved extrapolating patients' disease status and quality of life for 50 years into the future, to see what the long-term effects were. "This work provides additional evidence for Herceptin as a drug for breast cancer treatment. Where it differs from other work is that it shows the potential benefits of nine weeks of treatment instead of 12 months, in terms of both reduced costs and improved patient outcomes," said Dr Caroline Clarke (UCL Institute of Epidemiology and Health Care), who led the research. "Overall, our results suggest that the 9-week option could be the best option for both patients and the NHS, as it is apparently just as effective as the 12-month duration, safer in terms of leading to fewer cardiac side effects, and cheaper." The analysis had limitations including the fact that it was based on a model that incorporated two similar trials, rather than on a single trial directly comparing the three different options, so the results should be viewed with caution. Also, the trial used for the 9-week data had a relatively small number of patients in it and hence there is some uncertainty in the results.
News Article | February 21, 2017
Healthcare organisations should consider abandoning this costly and ineffective treatment New evidence suggests that receiving low intensity pulsed ultrasound (LIPUS) to speed up bone healing after fracture has little or no impact on pain or recovery time, say a panel of international experts in The BMJ today. They say LIPUS does not represent an efficient use of health resources and recommend that it should be stopped. Their advice is part of The BMJ's 'Rapid Recommendations' initiative - to produce rapid and trustworthy guidance based on new evidence to help doctors make better decisions with their patients. Both the new evidence and the guidance are published by The BMJ today. Every year around 4 in 100 people of all ages have a fracture - and up to 10% of these experience slow or complicated healing. As such, fractures have been a target for numerous interventions to aid recovery. LIPUS was approved for fracture healing by the US Food and Drug Administration (FDA) in 1994 and is also supported by the UK National Institute for Health and Care Excellence (NICE). Each device costs between US$1300 and $5000 and data suggest it is commonly used in clinical practice. But some studies have shown that the potential benefits of LIPUS on bone healing are highly uncertain. So The BMJ's guideline panel - made up of bone surgeons, physiotherapists, clinicians and patients with experience of fractures - carried out a detailed analysis of the latest evidence. They judged, with moderate to high certainty, that LIPUS has little or no impact on time to return to work, time to full weight bearing, pain, the number of subsequent operations, or time to healing assessed with radiographs (known as radiographic healing). As such, they unanimously recommend against LIPUS for patients with any bone fractures or osteotomy (the surgical cutting of a bone to allow realignment). "We have moderate to high certainty of a lack of benefit for outcomes important to patients, and, combined with the high costs of treatment, LIPUS represents an inefficient use of limited healthcare resources," they write. It is unlikely that new trials will alter the evidence, they add. And they suggest that future research "should focus on other interventions that have a greater probability to speed up healing." The BMJ is working with MAGIC, a non-profit research and innovation programme, to develop Rapid Recommendations. These accelerate the translation of evidence into practice by answering the questions that matter quickly and transparently through trustworthy recommendations. Every Rapid Recommendations panel is free from financial conflicts and includes experts and people with lived experience. For more information about MAGIC, visit: http://magicproject. For more information about Rapid Recommendations: http://www. BMJ is a healthcare knowledge provider that aims to advance healthcare worldwide by sharing knowledge and expertise to improve experiences, outcomes and value. For a full list of BMJ products and services, please visit bmj.com/company