(Press-News.org) Ruxolitinib (trade name: Jakavi) has been approved since March 2015 for the treatment of adults with polycythaemia vera, a rare disease of the bone marrow. It can be used when the drug hydroxyurea is ineffective or not tolerated. The German Institute for Quality and Efficiency in Health Care (IQWiG) examined in a dossier assessment whether this drug offers an added benefit over the appropriate comparator therapy.
According to the findings, ruxolitinib offers better relief of individual symptoms and improves quality of life. Dyspnoea and muscle cramps are more frequent, however. The study design generally limits the informative value of the data, which is why IQWiG can derive a hint of a non-quantifiable added benefit from them.
One quarter of the patients in the control arm not treated according to approval
The drug manufacturer used a multicentre, open-label, randomized controlled parallel group trial in its dossier (RESPONSE). It compared ruxolitinib with the best available therapy (BAT). The study design had two features that are important for the interpretation of the results. On the one hand, the BAT was individually specified only after the patients had been randomly assigned to the control group. On the other, only 75% of the participants in the control group were treated in compliance with the recommendations in the approval.
Since the BAT was specified only after randomization, no analyses can be conducted on patients with approval-compliant treatment in whom randomization was maintained. However, the results of the total population of the RESPONSE study were largely in line with the ones of the population with approval-compliant treatment, which is why also IQWiG used the data of the RESPONSE study for the benefit assessment. Overall, the data only have limited informative value, however.
Patients complain of fatigue less frequently
The results of the RESPONSE study showed that patients in the ruxolitinib arm complained of fatigue less frequently and considered their health status to be better. The participants who received ruxolitinib assessed their quality of life to be better at least with regard to physical function. However, there were also results to the disadvantage of the new drug: Dyspnoea and muscle cramps were more frequent.
Group differences were sufficiently large
The informative value of the results is limited because of the study design. However, the effects were so large that they cannot be caused by the study design alone. Overall, IQWiG derived a hint of a non-quantifiable added benefit from the dossier.
IQWiG had conducted the first assessment of ruxolitinib according to the Act on the Reform of the Market for Medicinal Products (AMNOG) in August 2014, but in a different therapeutic indication (myelofibrosis).
G-BA decides on the extent of added benefit
This dossier assessment is part of the early benefit assessment according to AMNOG supervised by the Federal Joint Committee (G-BA). After publication of the dossier assessment, the G-BA conducts a commenting procedure and makes a final decision on the extent of the added benefit.
INFORMATION:
An overview of the results of IQWiG's benefit assessment is given by a German-language executive summary. In addition, the website gesundheitsinformation.de, published by IQWiG, provides easily understandable German-language information.
More English-language information will be available soon (Sections 2.1 to 2.6 of the dossier assessment as well as subsequently published health information on informedhealthonline.org). If you would like to be informed when these documents are available, please send an e-mail to info@iqwig.de.
Brussels, [7 August 2015] - An unresolved inflammatory response is likely to be involved from the early stages of disease development. Controlling inflammation is crucial to human health and a key future preventative and therapeutic target. In a recent ILSI Europe's article published in the British Journal of Nutrition, a coalition of experts explain how nutrition influences inflammatory processes and help reduce chronic diseases risk.
Inflammation is a normal component of host defence, but elevated unresolved chronic inflammation is a core perturbation in a range of ...
Simon Fraser University scientist Jonathan Moore has authored new research suggesting that a proposed controversial terminal to load fossil fuels in the Skeena River estuary has more far-reaching risks than previously recognized.
In a letter newly published in the journal Science Moore and First Nations leaders and fisheries biologists from throughout the Skeena watershed refer to the new data, which is on the Moore Lab site.
Moore is a Faculty of Science and a Faculty of Environment professor of ecology and conservation of freshwaters and the Liber Ero Chair of Coastal ...
HOUSTON, August 6, 2015 - This week marks the 50th anniversary of the Voting Rights Act. This week also marks a watershed ruling by a federal appeals court striking down the controversial Texas voter ID law as violating that landmark civil rights act.
A new study conducted by the University of Houston Hobby Center for Public Policy and Rice University's Baker Institute for Public Policy examines the impact of the contested Texas law in U.S. Congressional District 23 (CD-23).
The study suggests that the most significant impact of the Texas voter photo ID law on voter ...
Psychologists from Cardiff University have developed the first self-assessment test designed to help clinicians diagnose autism in adults.
Published in the Journal of Autism and Developmental Disorders the test measures the extent to which adults are affected by repetitive behaviours - one of the criteria used to diagnose autism.
These behaviours include common habits and routines, such as lining up objects or arranging them in patterns, fiddling obsessively with objects, or insisting that aspects of a daily routine remain exactly the same.
Researchers say that the ...
A web-based programme to encourage more frequent handwashing reduces the risk of catching and passing on respiratory tract infections to other household members, a randomised trial of more than 16000 UK households published in The Lancet has found. Users of the programme, called PRIMIT [1], also reported fewer gastrointestinal infections, a lower demand for consultations with their doctors, and fewer antibiotic prescriptions.
"Our findings suggest that a simple, cheap internet programme to encourage handwashing can reduce the risk of infection by around 14%. Because most ...
Adding the price tag to prescription medicines worth more than £20 in England is just a "headline grabbing gimmick," which, among other things, could potentially mislead patients into believing that cheaper drugs are somehow less important, says an editorial in Drug and Therapeutics Bulletin (dtb).
On 1 July this year, health secretary for England Jeremy Hunt announced plans to print the indicative cost of medicines on all packs of those worth more than £20 alongside the phrase "funded by the UK taxpayer."
The initiative aims to encourage more people to ...
There is very little evidence to recommend avoiding certain types of dietary carbohydrate, known as the FODMAP diet, to ease the symptoms of irritable bowel syndrome, or IBS for short, concludes a review of the available data in Drug and Therapeutics Bulletin (dtb).
IBS is characterised by abdominal pain/discomfort and altered bowel frequency in the absence of any obvious gut abnormalities. Symptoms can include abdominal bloating, which eating can worsen.
Up to one in five of the population is thought to be affected, with women twice as likely to develop IBS symptoms ...
Highlights
In a population-based study, poor kidney function was strongly related to decreased blood flow to the brain.
Poor kidney function was linked to stroke and dementia most strongly in participants with decreased blood flow to the brain.
A growing body of research suggests a link between kidney impairment and brain disorders.
Washington, DC (August 6, 2015) -- Impaired kidney function may lead to decreased blood flow to the brain, and ultimately to the occurrence of stroke or dementia. The findings, which come from a study appearing in an upcoming issue ...
Simon Fraser University scientist Jonathan Moore has authored new research suggesting that a proposed controversial terminal to load fossil fuels in the Skeena River estuary has more far-reaching risks than previously recognized.
In a letter newly published in the journal Science Moore and First Nations leaders and fisheries biologists from throughout the Skeena watershed refer to the new data, which is on the Moore Lab site.
Moore is a Faculty of Science and a Faculty of Environment professor of ecology and conservation of freshwaters and the Liber Ero Chair of Coastal ...
BOSTON - Researchers at Beth Israel Deaconess Medical Center (BIDMC) have identified a new vitamin B3 pathway that regulates liver metabolism. The discovery provides an opportunity to pursue the development of novel drug therapies to address obesity, type 2 diabetes and related metabolic diseases.
Published in the August 2015 issue of Nature Medicine, the new findings show that a small molecule called N1-methylnicotinamide prevents metabolic complications caused by a high-fat diet.
"Our laboratory investigates the metabolic effects of nicotinamide adenine dinucleotide ...
