IQWiG: Reliable assessment of drugs is only possible on the basis of clinical study reports (CSRs)

Current study shows: Important information on effects and side effects of drugs is missing in most publications

2013-10-09

(Press-News.org) In 2012 researchers from the German Institute for Quality and Efficiency in Health Care (IQWiG) presented a study in the BMJ analysing information sources used in 16 health technology assessment (HTA) reports of drugs ("benefit assessments"). This study clearly demonstrated that publicly available sources, such as scientific journals and entries posted in trial registries ("registry reports"), contained far less information on methods and outcomes of clinical trials than non-public CSRs prepared by pharmaceutical companies.

In a second article published today in PLOS Medicine, the IQWiG researchers now show that if, instead of only assessing selected outcomes as in the first study, all patient-relevant outcomes of the clinical trials are assessed, the information deficit in the publicly available sources is even greater.

Data analysed for more than 1000 outcomes

All HTA reports of drugs completed by IQWiG between 2006 and 2011 also formed the basis for the new study. The authors included those trials from the HTA reports for which the pharmaceutical companies had provided complete CSRs to IQWiG. Publicly available information in scientific journals and trial registries was available for 86 out of 101 of these trials, so that the information provided in all 3 sources could be compared. The trials contained data on more than 1000 patient-relevant outcomes such as mortality or disease symptoms.

Huge difference in the information provided

IQWiG assessed whether the results on the patient-relevant outcomes in the trials were "completely" or "incompletely" reported. The difference in the information provided was immense: Whereas 86% of all outcomes were fully reported in unpublished CSRs, the corresponding number was only 39% for combined publicly available sources. Likewise, negative effects on patients ("harm outcomes") such as serious adverse events or treatment discontinuations were reported far less often in the publicly available sources (27 to 72% versus 84 to 92%, depending on the harm outcome investigated).

Make CSRs publicly accessible

Beate Wieseler, Head of the Drug Assessment Department, comments on this first comprehensive quantification of the information gain through full CSRs: "The publicly available journal articles and registry entries thus report less than half of outcomes of clinical trials comprehensively. At the same time, with CSRs documents are available that provide complete information on methods and outcomes. The consequence can only be: all CSRs must be made publicly accessible. One should note that IQWiG is already in a privileged position due to its legal remit to conduct benefit assessments. Other researchers and physicians wishing to be fully informed about the advantages and disadvantages of an intervention have even more difficulties in gaining access to data.

Although the proportion of clinical trials published as scientific articles or registry reports has increased in the past few years, this is unfortunately not accompanied by more complete reporting of patient-relevant outcomes. Large information gaps still remain and we cannot even guess how large these gaps are in other types of drug trials or in trials of non-drug interventions."

Need for legislation

Beate Wieseler further points out: "The plan by the European Medicines Agency (EMA) to make all clinical trial data submitted for marketing authorization publicly available from January 2014 onwards can therefore only be a first step. A central repository is required, also including data from older trials, as such trials investigate drugs widely used in current medical practice. These data would probably shed a totally new light on several drugs and their position in their therapeutic area. However, a voluntary commitment by the pharmaceutical industry, which would like to decide on a case-by-case basis which data it discloses, is insufficient. We are hoping for legislation and continue to support the All Trials Campaign (see below) so that the problem stays on the agenda."

### END

ELSE PRESS RELEASES FROM THIS DATE:

New mechanism preserving genomic integrity and is abnormal in the rare DiGeorge syndrome

2013-10-09

An international team of scientists—including researchers at GENYO, the Centre for Genomics and Oncological Research (Pfizer-University of Granada- Andalusian Regional Government)—has described a molecular mechanism that facilitates the defence of the human genome against "bombarding" by mobile DNA sequences. Abnormalities in the mechanism could be responsible for some symptoms of DiGeorge syndrome, a rare disease. The research could in the future help develop new therapies against the disease, which is caused by the microdeletion of a small part of chromosome 22. The ...

No serious adverse reactions to HPV vaccination

2013-10-09

Researchers at Karolinska Institutet in Sweden and their Danish colleagues have monitored HPV-vaccinated girls via patient data registries in order to examine the incidence of a wide range of diseases and thus determine if there are any serious adverse effects of the vaccine. Their results show no significant increase of the examined diseases in the vaccinated girls relative to their unvaccinated peers. The study included almost a million Swedish and Danish girls born between 1988 and 2000, and compared roughly 300,000 girls who had been HPV vaccinated with 700,000 who ...

Penn study: Visits to multiple HIV clinics linked to poorer outcomes

2013-10-09

PHILADELPHIA—Patients who received care at multiple HIV clinics—as opposed to only one— were less likely to take their medication and had higher HIV viral loads, a new study published in the journal AIDS and Behavior of almost 13,000 HIV patients in Philadelphia from Penn Medicine found. The findings reinforce the notion that continuous care with one provider/clinic is optimal for outcomes and even reducing transmissions, and can help cut down on duplicative HIV services that contribute to higher health care costs. "It's about retention in care, but also continuity, ...

Scientists use blur to sharpen DNA mapping

2013-10-09

HOUSTON – (Oct. 9, 2013) – With high-tech optical tools and sophisticated mathematics, Rice University researchers have found a way to pinpoint the location of specific sequences along single strands of DNA, a technique that could someday help diagnose genetic diseases. Proof-of-concept experiments in the Rice lab of chemist Christy Landes identified DNA sequences as short as 50 nucleotides at room temperature, a feat she said is impossible with standard microscopes that cannot see targets that small, or electron microscopes that require targets to be in a vacuum or cryogenically ...

Peer pressure's influence calculated by mathematician

2013-10-09

A mathematician has calculated how peer pressure influences society. Professor Ernesto Estrada, of the University of Strathclyde's Department of Mathematics and Statistics, examined the effect of direct and indirect social influences – otherwise known as peer pressure – on how decisions are reached on important issues. Using mathematical models, he analysed data taken from 15 networks – including US school superintendents and Brazilian farmers – to outline peer pressure's crucial role in society. Professor Estrada said: "Our modern society is a highly-interconnected ...

Discovery of a 2,700-year-old portico in Greece

2013-10-09

A 2,700-year-old portico was discovered this summer on the site of the ancient city of Argilos in northern Greece, following an archaeological excavation led by Jacques Perreault, Professor at the University of Montreal's Centre of Classical Studies and Zisis Bonias, an archaeologist with the Hellenic Ministry of Culture and Sports. In ancient Greece, the portico—stoa in Greek—was a long, open structure that often housed shops and delineated public squares from the city—the agora. "Porticos are well known from the Hellenistic period, from the 3rd to 1st century BC, but ...

Novel gene therapy enables persistent anti-tumor immune response

2013-10-09

New Rochelle, NY, October 9, 2013—Cancer immunotherapy can successfully use the body's own immune system to kill tumor cells. But some current approaches to stimulate an antitumor immune response are short-lived, with limited clinical effectiveness. A new gene transfer strategy that introduces modified, immune-stimulating human stem cells is both feasible and effective for achieving persistent immunotherapy to treat leukemias and lymophomas, according to a study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article ...

Multivitamins with minerals may protect older women with invasive breast cancer

2013-10-09

October 9, 2013 — (BRONX, NY) — Findings from a study involving thousands of postmenopausal women suggest that women who develop invasive breast cancer may benefit from taking supplements containing both multivitamins and minerals. The new research, published today in Breast Cancer Research and Treatment, found that the risk of dying from invasive breast cancer was 30 percent lower among multivitamin/mineral users compared with nonusers. "Our study offers tentative but intriguing evidence that multivitamin/mineral supplements may help older women who develop invasive ...

When it comes to the good cholesterol, fitness trumps weight

2013-10-09

Bethesda, Md. (Oct. 9, 2013)—There's no question that high levels of good cholesterol—also known as high-density lipoprotein (HDL)—seem to be protective against heart disease. Rather than depositing fat into the blood vessels the way the "bad" cholesterol (low-density lipoprotein (LDL)) does, HDL appears to carry cholesterol away from blood vessels to the liver. From there, the liver processes it for removal from the body. However, adequate levels of HDL might not be enough. Several recent studies have suggested that many cases of heart disease occur in people with ...

Football players suffer more injuries when their team is ahead

2013-10-09

Male football players are at a greater risk of injury five minutes after a card has been given or after a goal has been scored. The frequency of player injuries also increases when their own team is in the lead. These are the findings of researchers at the Sahlgrenska Academy, who, in collaboration with Fifa, have analysed injuries over the last three World Cup tournaments. Football players do not just injure themselves whenever and however. On the contrary, injury frequency follows a clear pattern that is dependent on how various events in the course of a match affect ...