venerdì 3 gennaio 2014

A step ahead of controversial transparency rules, Sanofi commits to open access Read more: A step ahead of controversial transparency rules, Sanofi commits to open access - FierceBiotech http://www.fiercebiotech.com/story/step-ahead-controversial-transparency-rules-sanofi-commits-open-access/2014-01-03#ixzz2pNQsREHB Subscribe at FierceBiotech

Sanofi is starting the New Year with a resolution to join the open-access movement. But it's keeping the vaults firmly closed on any past data--this is one resolution that applies only to future drug approvals.
Like GlaxoSmithKline ($GSK), and to a certain extent, Roche ($RHHBY) and Pfizer ($PFE) before it, Sanofi ($SNY) says it will provide data, documents and reports on studies used to back up U.S. and European applications on drugs approved after the first day of this year. And it will work with regulators to provide simple explanations of individual results to the subjects involved in their clinical studies.
Pfizer was the first big company to lay out plans to open up results to individual trial subjects. Just weeks ago the pharma giant also made a commitment to make it easier for qualified independent researchers to look over clinical data.
It's probably no coincidence that Sanofi's move to open up on new drugs coincides with the joint principles on data sharing adopted by the European Federation of Pharmaceutical Industries and Associations and the Pharmaceutical Research and Manufacturers of America, which also went into effect Jan. 1. The EFPIA--currently led by Sanofi CEO Chris Viehbacher--and PhRMA have been scrambling to come up with an industry solution to a regulatory problem. The industry groups have been waging open warfare against a proposal by the European Medicines Agency to force data disclosure in a way that is likely to shed far more light on data than the industry would like to see. From some of the companies' perspectives, the EMA is trying to open the door to confidential product information, and they are determined to stop it.
The EMA has had to delay its final policy on transparency until March while the European Union moves ahead on its own sunshine rules.
"Sanofi has a history of contributing in this collective effort of sharing clinical trial data and results with researchers and patients with initiatives such as Project Data Sphere, an independent initiative of the Life Sciences Consortium of the CEO Roundtable on Cancer, the Coalition Against Major Diseases, and Prize4Life," said Viehbacher in a statement. "Finding new therapies can be accelerated by fully sharing the successful and unsuccessful research results with other researchers. Data sharing helps to reduce duplication and allows researchers to build more effectively on the findings of other researchers. The private sector has taken a lead on this which I would hope academic researchers will follow."

NHS could be 'overwhelmed' by people with long-term medical conditions

The soaring number of people with long-term medical conditions such as diabetes and dementia is threatening to "overwhelm" the NHS, one of the health service's most senior figures warns.
The challenges posed by patients with chronic medical conditions are so great that they represent the "healthcare equivalent to climate change" and must force the NHS to undertake a major rethink of how it cares for such patients, Dr Martin McShane says in an interview with the Guardian.
Looking after the 15.4 million people in England with at least one long-term condition already takes up 70% of the NHS's £110bn budget – £77bn – as well as £10.9bn of the £15.5bn spent on social care in England, he says. The costs are so huge that the NHS could become unsustainable unless it gives those with long-term conditions better care, with much of it provided by GPs performing enhanced roles rather than hospital doctors, says McShane, NHS England's national director for people with long-term conditions.
McShane is responsible for those ongoing illnesses or diseases that see patients become regular users of NHS services, through check-ups, tests and operations. They include arthritis, heart disease, breathing problems, obesity and mental health conditions such as depression. Their numbers have risen dramatically in recent years, largely as a result of the ageing population and lifestyle factors such as smoking, drinking and overeating.
"I would say it's the healthcare equivalent to climate change. It is putting pressure into the system, which, unless we change the way we address the problems, will overwhelm the system," says McShane.
"This is the biggest problem facing the health system and the care system and the costs are growing year on year. They are huge already and they will continue to grow."
The NHS in its current form is not well set up to look after patients who are medically complicated, especially if they have several long-term conditions, such as arthritis, heart failure and the early signs of dementia, McShane says. While the total number of people with long-term conditions is expected to stay at around 15 million, the number with three or more conditions is expected to rise from 1.9 million to 2.9 million by 2018.
"People with multiple long-term conditions often fall through the gaps as their secondary [hospital] care is highly specialised and their GP care highly generalised, with little continuum between the two, meaning those with multiple long-term conditions can fall through the gaps when confronted with confusing and fragmented secondary care," he says.
The failure so far to reorganise services for such patients means too many are not getting proper care and can end up having largely avoidable spells in hospital, which adds to the pressure on A&E units and hospital beds and also wastes vital NHS resources.
The NHS will not bridge the £30bn gap that it fears will have emerged by 2020 between its budget and the volume of care needed unless it takes radical steps to cope with rising numbers of long-term conditions, McShane warns.
Research by Professor Andrew Street, a health economist at York University, has found that while a healthy patient costs the NHS about £288 a year, those with one long-term condition cost an estimated £783, those with two cost £1,521 and those with three cost £2,559 each. Their need for frequent treatment and monitoring means that the small minority with five such conditions cost £5,512 a year and those with six about £8,083.
England is regarded as world-leading in long-term condition management, for example by helping diabetics avoid undergoing an amputation, of a finger, toe or even a limb, as a result of complications. But despite that, says McShane: "This huge need has emerged and we haven't evolved fast enough to meet it.
"General practice is doing a fantastic job, as it always has done, and specialists have become more and more specialised. But there's the needs of a new generation, the geriboomers, who are now living longer and collecting long-term conditions, so our thinking has to change."
The NHS is still set up to deal with 20th-century medical need and must evolve rapidly to better handle long-term conditions, the greatest challenge of this century, he says.
McShane wants some family doctors to do extra training and become "complex care GPs", to look after only people with long-term conditions, especially the 5% of the population who are the heaviest users of NHS services and take up most of doctors' and nurses' time. They would then lead small teams of health and social care professionals who would try to keep the patients as well as possible in their own homes. As many as 50 GP practices could come together to do that, as well as looking after elderly patients in care homes and using telehealth to monitor patients who are still living at home, he says.
Professor Chris Ham, chief executive of the King's Fund health thinktank, said: "The NHS and social care have been slow to rise to the twin challenges of an ageing population and increased prevalence of long-term conditions like diabetes. There is now an urgent need to transform how GPs treat people with these conditions, and to support people themselves to take more control over their health."
A few innovative GPs are already undertaking specialist training to expand their skills and offering extra services in their surgeries such as minor injury clinics, diagnostic facilities and access to specialist nurses and doctors, he added.
Norman Lamb, the care and support minister, agreed with McShane that services for those with long-term conditions must improve. "We want to build a fairer society, and that means providing better care to people with long-term conditions so that they are able to enjoy an independent, fulfilling life, and have the support needed to manage their health," he said.
The government's planned £3.8bn-a-year Better Care Fund, which starts in April 2015, will fund the integration of health and social care services so patients can live independently for as long as possible, while recent changes to the GP contract should help reduce avoidable attendances at A&E units, he added.

giovedì 2 gennaio 2014

Agreement reached in Europe on Clinical Trials Regulation

Exciting news from Brussels this morning – law that would mean researchers running a clinical trial in Europe have to register the trial before it begins and to publish summary results within a year of its end is a step closer. The committee of representatives from every EU member state government has agreed with the text of the Clinical Trials Regulation proposed by MEPs led by Glenis Willmott. This agreement now has to be formally ratified by the European Parliament and the Council of Ministers (probably in early 2014) but today’s provisional agreement is a fantastic result at a very important stage of negotiation and is down to the hard work of the MEPs and thanks to your input.
The proposals agreed today include:
  • A publicly accessible EU database, set up and run by European Medicines Agency, containing:
    • A register of all trials carried out in the EU
    • A summary of results for all trials, uploaded 1 year after the end of the trial at the latest
    • As well as a summary understandable for a layperson
    • Clinical Study Reports for all trials used in a marketing authorisation request, whether it is approved, rejected or withdrawn
  • A statement that Clinical Study Reports should, in general, not be considered commercially confidential
  • Fines to be imposed by Member States for non-compliance with the transparency requirements
  • A requirement for all trials to be registered or published in order to be used to back up a new clinical trial authorisation (will encourage the retrospective registering/publishing of old trials)
  • The Clinical Trial Master File retained for at least 25 years.
Read more on the agreement reached today on the European Parliament website.
Glenis Willmott MEP said: “For too long, unflattering studies on new medicines have gone undisclosed. Around half of all trials are never published, usually those with negative or disappointing results. It is vital we know about negative outcomes, otherwise trials can be conducted repeatedly before it becomes public knowledge they are ineffective, or even dangerous.”
The legislation will need final approval from the European Parliament.
“We are determined to finalise this before the European elections in May. This legislation will set the global gold standard for transparency in clinical trials, and I call on all EU governments to support the agreement” Ms Willmott said.
Síle Lane, Director of Campaigns, Sense About Science said: “We are very pleased to hear that EU governments have agreed the draft Clinical Trials Regulation which contains proposals that would mean all clinical trials done in Europe will have to be registered and results reported. Well done to Glenis Willmott MEP who has worked very hard to get this agreement. We know Glenis was helped enormously by the hundreds of individuals and organisations who got involved and told MEPs and Ministers their reasons why transparency is vital. Now is the time for companies and academics to listen to all these voices and commit to registering and reporting results from trials done in the past too. There’s no excuse for not publishing results but a huge public health benefit to having a complete picture of what was found in trials conducted on treatments currently available to patients.”
Dr Ben Goldacreco-founder of AllTrials said: ”This is great news, and patients around the world should be grateful for the fantastic work that has been done on this by politicians in Europe. However we must remember that it only covers new trials, starting from 2014. The vast majority of medicines prescribed today came on the market 5, 10, or 20 years ago. This new law will do nothing to improve the evidence base for those treatments. We still cannot get all the results of all the trials on even the simplest everyday treatments, the antidepressants, the statins, the blood pressure treatments, and more. Those are the drugs that doctors are using right now, and will continue to use for at least a generation. Notably, the law would also do nothing to ensure that researchers and doctors can access all the results of trials on controversial drugs such as Tamiflu.
The campaign for access to trial results began over 20 years ago. Throughout the 1990s and 2000s, industry promised that everything had changed, and that everything had become more transparent. We now know that this wasn’t true. It is unacceptable that results from the 1990s and 2000s should continue to be withheld from doctors, researchers and patients. It is unacceptable to say that these trial results were allowed to somehow continue “disappearing”, even while some of the industry was publicly promising greater transparency. With their reckless belief that they could stonewall forever, too much of the industry have stored up a big problem for themselves, and for patients. We need the results of all trials, of all uses, of all the treatments we prescribe today. Without that, we cannot practice medicine safely and effectively.”
Mark Wilson, CEO, Cochrane Collaboration said: “Cochrane is delighted with today’s agreement on clinical trials transparency;  a victory for patients, practitioners and policy-makers across Europe, and a clear and compelling message to decision-makers worldwide.  The ratification of the agreement in 2014 will be a key milestone on the AllTrials journey to ‘all results reported’ becoming a reality.”

Is it human, animal or plant? At Bayer, they're mixing all three in new R&D recipe

Can a crop science team of ag-bio investigators provide some real insights that could help advance new drugs for humans and animals? Bayer believes so, and one of its top researchers says you can expect to see the first tangible fruits of its hybrid R&D effort in the next few years.
Wolfgang Plischke, the outgoing tech/innovation chief at Bayer, told Reuters that "we expect it to be reflected in our pipeline in the next two, three, four, five years." "It" is Nimbus, a two-year-old project in which Bayer is bringing together researchers involved in crop science and human and animal drug development under one roof--even as the industry continues to segregate the fields.
Bayer decided to launch Nimbus back in 2011, committing a bit more than $40 million a year to the project. While the final products may vary, Bayer believes that basic insights into switching genes on and off or transporting active ingredients into cells spans all three disciplines and that researchers can learn from each other.
Regardless of its unique effort at integrating research, Bayer has already posted some significant gains in recent years with an R&D budget of about $4 billion a year. The company won a key approval for the prostate cancer drug Xofigo, which it now controls completely after the $2.9 billion acquisition of Algeta. The approval builds on earlier successes for Stivarga and Nexavar, now partnered with Amgen ($AMGN).

FDA lifts a partial hold on Cell Therapeutics' leukemia drug

Cell Therapeutics ($CTIC) got some good news from the FDA to start 2014. The agency has lifted a partial hold on tosedostat (IND 075503), giving the Seattle-based biotech a green light for all clinical studies of the cancer drug.
The FDA had clamped a partial hold on the mid-stage drug last summer, after a patient taking the drug in a study died of myocarditis, an inflammation of heart muscle. Cell Therapeutics shares barely budged today, though, perhaps reflecting a wary investment community which had tired long ago of the biotech's erratic course.
Cell Therapeutics bought tosedostat for a mere $10 million. The experimental therapy inhibits aminopeptidases, and the biotech is developing it in Phase II for acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS).
CTIC is perhaps best known for losing a unanimous vote by outside FDA experts after agency cancer czar Richard Pazdur laid into the program for Pixuvri (pixantrone), which failed to demonstrate a statistically significant improvement over the control arm.
"We are pleased that the FDA has responded favorably to the tosedostat clinical trial data provided and removed the partial clinical hold to allow further development of tosedostat in ongoing and future studies," said John Pagel, a researcher at the prestigious Fred Hutchinson Cancer Research Center and the principal investigator in the tosedostat first-line AML/MDS trial, in a statement.

Researchers find potential new treatment approach for pancreatic cancer

Scientists from The University of Manchester - part of Manchester Cancer Research Centre believe they have discovered a new way to make chemotherapy treatment more effective for pancreatic cancer patients. Pancreatic cancer is an aggressive cancer with poor prognosis and limited treatment options and is highly resistant to chemotherapy and radiotherapy.
But researchers believe they have found an effective strategy for selectively killing pancreatic cancer while sparing healthy cells which could make treatment more effective.
Dr Jason Bruce, from the Physiological Systems and Disease Research Group, who led the research, said: "Pancreatic cancer is one of the most aggressive and deadly cancers. Most patients develop symptoms after the tumour has spread to other organs. To make things worse, pancreatic cancer is highly resistant to chemotherapy and radiotherapy. Clearly a radical new approach to treatment is urgently required. We wanted to understand how the switch in energy supply in cancer cells might help them survive."
The research, published in The Journal of Biological Chemistry this month, found pancreatic cancer cells may have their own specialised energy supply that maintains calcium levels and keeps cancer cells alive.
Maintaining a low concentration of calcium within cells is vital to their survival and this is achieved by calcium pumps on the plasma membrane.
This calcium pump, known as PMCA, is fuelled using ATP - the key energy currency for many cellular processes.
All cells generate energy from nutrients using two major biochemical energy "factories", mitochondria and glycolysis. Mitochondria generate approximately 90% of the cells’ energy in normal healthy cells. However, in pancreatic cancer cells there is a shift towards glycolysis as the major energy source. It is thought that the calcium pump may have its own supply of glycolytic ATP, and it is this fuel supply that gives cancer cells a survival advantage over normal cells.
Scientists used cells taken from human tumours and looked at the effect of blocking each of these two energy sources in turn.
Their study, funded by the Biotechnology and Biological Sciences Research Council (BBSRC), National Institute of Health Research (NIHR) Biomedical Research Centre and AstraZeneca, shows that blocking mitochondrial metabolism had no effect. However, when they blocked glycolysis, they saw a reduced supply of ATP which inhibited the calcium pump, resulting in a toxic calcium overload and ultimately cell death.
Dr Bruce added: "It looks like glycolysis is the key process in providing ATP fuel for the calcium pump in pancreatic cancer cells. Although an important strategy for cell survival, it may also be their major weakness.
"Designing drugs to cut off this supply to the calcium pumps might be an effective strategy for selectively killing cancer cells while sparing normal cells within the pancreas."
Maggie Blanks, CEO of the national charity, the Pancreatic Cancer Research Fund said: "These findings will certainly of great interest to the pancreatic cancer research community and we'd be keen to see how this approach progresses. Finding weaknesses that can be exploited in this highly aggressive cancer is paramount, so we want to congratulate the Manchester team for their discovery."
Andrew D. James; Oihane Erice; Jason I. E. Bruce. Glycolytic ATP Fuels the Plasma Membrane Calcium Pump Critical for Pancreatic Cancer Cell Survival. The Journal of Biological Chemistry, Vol. 288, Issue 50, 36007-36019, December 13, 2013.

US global share of research spending declines

The United States' global share of biomedical research spending fell from 51 percent in 2007 to 45 percent in 2012, while Japan and China saw dramatic increases in research spending. The research and development spending in the United States dropped from $131 billion to $119 billion, when adjusted for inflation, from 2007-2012, while Japan increased spending by $9 billion and China increased by $6.4 billion. Overall, Asia's share of spending grew from 18 percent to 24 percent. Europe held steady at 29 percent.
Prior analyses have suggested the United States' share of global expenditures were once as high as 80 percent.
Results of this new analysis, conducted by researchers from industry and academia, including both medical researchers and economists, appear in the Jan. 2 New England Journal of Medicine.
"The United States has long been a world leader in driving research and development in the biomedical science. It's important to maintain that leadership role because biomedical research has a number of long term downstream economic benefits, especially around job creation," says study author Reshma Jagsi, M.D., D.Phil., associate professor of radiation oncology at the University of Michigan Health System.
Despite reductions in funding from the National Institutes of Health, including a 20 percent drop in purchasing power since 2003, the researchers discovered that the United States' decline was driven almost entirely by reduced investment from industry, not the public sector. This includes support for clinical trials testing potential new therapies.
Jagsi suggests that this may boil down to fewer regulations and less expense to conduct research in Asia – labor costs less, government may be offering subsidies and the research infrastructure is not as bureaucratic as it is in the United States.
"We were surprised the impact of industry funding was that dramatic, but it's key to note that government funding is equally important to maintain or grow. Research funded through the National Institutes of Health helps scientists understand how diseases work – this will happen slower as NIH funding continues to be cut," says study author Justin Chakma, a venture capital investor with Thomas, McNerney & Partners in La Jolla, Calif.
Historically, about half of drugs approved by the U.S. Food and Drug Administration had some federal government funding during the course of the research and development.
The authors note the critical need for increased NIH funding coupled with incentives to industry for investing in biomedical research and development here. They call the lack of a coordinated national biomedical R&D strategy "disappointing."
Additional authors: Gordon H. Sun, M.D., M.S., University of Michigan Health System, VA Ann Arbor Healthcare System, Robert Wood Johnson Foundation Clinical Scholars Program; Jeffrey D. Steinberg, Ph.D., Singapore Bioimaging Consortium, Agency for Science, Technology and Research; Stephen M. Sammut, M.A., M.B.A., Wharton School of Business, University of Pennsylvania