PhRMA Cheers Progress in Biotech

Report cites more than 900 biologic therapies and vaccines in development.

MICHAEL FITZHUGH

The Burrill Report

American biopharmaceutical companies are using biotechnology to develop more than 900 medicines and vaccines targeting more than 100 diseases, according to a new catalog of therapeutic candidates published by the Pharmaceutical Research and Manufacturers of America.

“These medicines reflect the cuttin
g-edge research being conducted across the country by biopharmaceutical companies in order to provide new treatment options to patients,” says John Castellani, PhRMA’s president and CEO.

http://www.burrillreport.com/article-phrma_cheers_progress_in_biotech.html

Drug Companies Sue EMA over Trials Data

MICHAEL FITZHUGH

The Burrill Report

“We consider this information to be commercially confidential.”

 

AbbVie and InterMune are suing the European Medicines Agency to block it from releasing clinical trial data requested by competitors in freedom of information requests submitted to the regulator.

The lawsuits, first reported in the Financial Times, seek injunctions to prevent the release of detailed information about AbbVie’s rheumatoid arthritis therapy, Humira’s effect on individual patients, and InterMune’s Esbriet, a therapy for idiopathic pulmonary fibrosis.

AbbVie is the U.S. pharmaceutical company recently spun off from Abbott Laboratories

In a statement provided to Bloomberg, AbbVie said that it is “seeking to protect AbbVie’s confidential and commercially-sensitive information.”

http://www.burrillreport.com/article-drug_companies_sue_ema_over_trials_data.html

New Initiative Takes on Multiple Sclerosis with Big Data

GNS Healthcare, a big data analytics company, has formed a new alliance with non-profit patient and research groups intended to uncover the genetic and biologic causes of the complex autoimmune disease multiple sclerosis and other diseases.

Backed by a $5.4 million sponsorship from Janssen Research & Development, Orion Bionetworks will use GNS Healthcare’s Reverse Engineering and Forward Simulation platform to analyze patient data pieced together from alliance members including the Accelerated Cure Project for Multiple Sclerosis, the Institute for Neurosciences at Brigham and Women’s Hospital, and PatientsLikeMe. The Marin Community Foundation’s Multiple Sclerosis Project Fund will coordinate the project and Magali Haas, currently chief science and technology officer of the non-profit One Mind for Research, will lead Orion.

“I think data-driven models like this are the future of discoveries in healthcare and the development of new drugs. It’s just too slow otherwise,” says Colin Hill, CEO of GNS. “People accepted that before, because there just wasn’t any other way. Now I think the walls of the industry are going to come crumbling down, replaced by something much more data-driven and hypothesis-free, something that’s desperately needed.”
http://www.burrillreport.com/article-new_initiative_takes_on_multiple_sclerosis_with_big_data.html

India Weighs Price Controls versus Compulsory Licensing

The new draft policy on patented drug pricing puts more emphasis on government regulation.

SHERYL P. DENKER

The Burrill Report

- See more at: http://www.burrillreport.com/article-india_weighs_price_controls_versus_compulsory_licensing.html#sthash.c3sz9UZg.dpuf

The Indian government will consider the use of price controls rather than the controversial compulsory license tool to provide affordable medicines for their citizens, according to a draft guidance issued by India’s Department of Pharmaceuticals.

Under a compulsory license, an individual or company seeking to use another’s intellectual property can do so without seeking the rights holder’s consent, and instead pays the rights holder a set fee for the license. Although rarely put into practice, governments in developing countries have issued compulsory licenses to allow local generic drug manufacturers to produce expensive patented drugs without the consent of the patent holder and then sell those drugs to patients for a price that is much cheaper than the original drug.

In the new draft policy on price negotiation for patented drugs, the Department of Pharmaceuticals says that once patented drugs are regulated by government pricing controls, the provision to issue a compulsory license on the basis of not being affordable will no longer be relevant, since the drug’s cost should be considered reasonable at that point.

- See more at: http://www.burrillreport.com/article-india_weighs_price_controls_versus_compulsory_licensing.html#sthash.c3sz9UZg.dpuf

EU Leaders Agree on Patent Package

By streamlining system, Parliament hopes to cut costs and encourage filings.

 

SHERYL P. DENKER

The Burrill Report

“The unified patent package will provide uniform legal protection in 25 European countries.”

After almost 40 years of talks, E.U. leaders agreed to cut patent fees by 80 percent, making the European Union more competitive with the United States and Japan in attracting inventors.

“Intellectual property must not stop at borders,” says parliament member Bernhard Rapkay, who led group that developed the regulation setting up the unified system. “The path towards the introduction of the E.U. patent was long and troubled, but ultimately it has been worth the effort.”

National Health Service to Map DNA of Up to 100,000 Patients

Effort to focus on cancer in hopes of accelerating diagnosis, improving treatment, and sparking innovation.

 

SHERYL P. DENKER

The Burrill Report

“Understanding and harnessing genetic information offers huge potential to target effective treatments and develop new treatments and cures.”

 

The United Kingdom’s National Health Service says it will map the DNA of up to 100,000 patients with cancer and other rare diseases over the next three to five years at a cost of $162 million (£100 million).

“Britain has often led the world in scientific breakthroughs and medical innovations, from the first CT scan and test-tube baby through to decoding DNA,” says Prime Minister David Cameron, “and Britain should become the first country to introduce genetic sequencing to its mainstream health service.”

Economic pressures and an increasing scarcity of expertise give rise to the Virtual Scientific Workplace

The Burrill Report

The Burrill Report - Sponsored Podcast - The Virtual Scientific Workplace (.MP3,9.43 Mb)

Economic pressures and an increasing scarcity of expertise is leading companies to embrace new virtual networks models to draw on skilled scientific workers from around the globe to collaborate and share knowledge. In this final part of a three-part series of podcasts by The Burrill Report, produced in conjunction with Kelly Services, Harvey Yau, prodcuct manager for Kelly Services Americas Scientific Product Group, discusses the emergence of virtual networks in the life sciences, the benefits they provide, and what companies will need to do to be successful with these new workplace models.

New Combinations to Fight Cancer

Data from experiments using 5,000 different pairings of approved drugs will be made available.

Researchers at the National Cancer Institute have tested 5,000 different combinations of 100 approved cancer drugs in the hope of finding new pairings that could ultimately provide patients with more effective and safer treatments.

Scientists in the Division of Cancer Treatment and Diagnosis at the National Cancer Institute completed some 300,000 experiments aimed at testing all possible combinations of every currently approved small molecule cancer drug in the United States.

continue:
http://www.burrillreport.com/article-new_combinations_to_fight_cancer.html

Councils say better and more relevant research needed to improve clinical practice in Europe.

European Researchers Call for New Institute

DANIEL S. LEVINE

The Burrill Report

“It is imperative, morally, socially, and economically, that healthcare received by patients in Europe is based on the best scientific evidence.”

Reliable evidence needs to play a greater role in clinical decision-making in Europe and the quality of the research used to inform it needs to be improved, according to a policy briefing from the European Medical Research Councils.

The Councils, the European Science Foundation’s advocacy group for all medical research councils in Europe, offered a list of ten recommendations to address the problem and accelerate the pace at which new research findings can improve clinical practice.

 

http://www.burrillreport.com/article-european_researchers_call_for_new_institute.html

Scientists replace defective DNA with equivalent healthy DNA in battle against mitochondrial disease.

Swapping out DNA to Fight Disease

The Burrill Report

“The method has the potential to dramatically alter treatment of now-incurable mitochondrial diseases.”

Scientists at the Oregon Health & Science University have successfully replaced defective DNA in a human egg with the equivalent DNA from a healthy egg, potentially preventing women from passing on several rare mitochondrial disorders to their offspring.

In the latest study, an extension of a 2009 study performed on monkeys that led to healthy live births, many of the eggs containing the transplanted, healthy, mitochondrial DNA were properly fertilized and went on to produce healthy human embryos.

 

http://www.burrillreport.com/article-swapping_out_dna_to_fight_disease.html

Breast Cancer Study Points to Personalized Treatments

Whole genome scanning tested for clinical decision making.

DANIEL S. LEVINE

The Burrill Report

“The French consortium has opened a new door to more personalized treatment of advanced breast cancer.”

A first-of-its-kind, large scale trial of whole genome cancer testing to personalize treatment for patients with metastatic breast cancer finds in 20 percent of the cases uncovered rare and unexpected genomic activity and argues it’s time to make this approach a clinical standard.

A French research consortium presented the findings from their study at the 2012 European Society for Medical Oncology Congress in Vienna. The results were based on biopsies performed in 402 breast cancer patients, including 26 patients for whom analyses are ongoing.

for complete article:
http://www.burrillreport.com/article-breast_cancer_study_points_to_personalized_treatments.html

Bristol-Myers Squibb Forms the International Immuno-Oncology Network - from The Burrill Report

VINAY SINGH

The Burrill Report

“The public-private partnerships formed through the collaboration will leverage the intellectual capabilities of an extensive global network.”

Bristol-Myers Squibb has created a new International Immuno-Oncology Network, a collaboration between industry, universities, and research centers from around the world aimed at furthering the scientific understanding of immuno-oncology, or the human immune system’s potential to fight cancer.

In addition to Bristol-Myers Squibb, the collaboration consists of ten academic or cancer-research institutions from around the world including The Netherlands Cancer Institute, Dana-Farber Cancer Institute, The Royal Marsden NHS Foundation Trust, the Institute of Cancer Research, and Johns Hopkins Kimmel Cancer Center.

Collaborations between academia, private research institutions, and Big Pharma has become increasingly common as pharmaceutical companies continue to look outside their own walls to acquire promising new early-stage treatments and try to lessen both the risks and costs of developing such drugs.
http://www.burrillreport.com/article-bristol_myers_squibb_forms_the_international_immuno_oncology_network.html

FDA Stretches to Go Global, from The Burrill Report

The Burrill Report

With limited resources, agency targets trouble spots and seeks cooperation.

“The $10 million is a small drop in the bucket of what our overall needs are.”

Federal regulators struggling to catch up with global supply chains are working to strengthen ties to their foreign counterparts in China and beyond to better protect public health at home, the U.S. Food and Drug Administration says.

The agency is seeking $10 million from Congress to pay for additional staffing and drug manufacturer inspections in China and is developing new risk-based approaches to ensure product safety and quality to improve its results.

“The $10 million is a small drop in the bucket of what our overall needs are,” FDA Commissioner Margaret Hamburg testified recently before the Senate Appropriations subcommittee that oversees the FDA’s budget.

But coming to terms with globalization is “a huge priority,” she says. “FDA is at the cutting edge of much of this in terms of responding to the challenges of globalization. And at the present time we don’t have the tools and authorities that we fully need to achieve that nor do we have the resources.”

What the agency is doing with the limited resources it does have is covered in a new “Global Engagement Report” [http://www.fda.gov/AboutFDA/ReportsManualsForms/Reports/ucm298576.htm] detailing its efforts and evolving responses to the growing complexities and dangers of imported food and drugs. More than 80 percent of the active pharmaceutical ingredients and 40 percent of finished drug dosages sold in the United States are manufactured abroad.
for complete article,
http://www.burrillreport.com/article-fda_stretches_to_go_global.html

Predictive Value of Whole Genome Sequencing Questioned, The Burrill Report

With the rapid drop in the cost of whole genome sequencing, there is widely held hope that we will be entering an era of medicine that will allow doctors to predict and prevent disease in part through the information they glean from an individual’s genome. But a new study raises questions about its predictive value.

In a study published in Science Translational Medicine, researchers at Johns Hopkins University used records on thousands of identical twins in registries in several Northern European countries, as well as a registry of twins held by the National Academy of Sciences. Identical twins have matching genomes. The researchers looked at how well the genome predicted an individual’s disease by seeing how often an individual with a specific disease had a twin who also suffered from the same disease. They argue that such an approach would suggest how well genome sequencing could predict risk for disease.

“We believe that genomic tests will not be substitutes for current disease prevention strategies," says Bert Vogelstein, professor of Oncology at the Johns Hopkins Kimmel Cancer Center, co-director of the Ludwig Center for Cancer Genetics. “Prudent screening, early diagnosis and prevention strategies, such as not smoking and removing early cancers, will be the keys to cutting disease death rates.”

for more:
http://www.burrillreport.com/article-predictive_value_of_whole_genome_sequencing_questioned.html

US Unveils $200 Million Big Data Initiative, The Burrill Report

The Obama Administration has launched a Big Data Research and Development Initiative in conjunction with six federal departments and agencies, making a $200 million commitment to improve the tools and techniques needed to access, organize, and glean discoveries from the huge volumes of data being generated around the world.

“In the same way that past federal investments in information technology R&D led to dramatic advances in supercomputing and the creation of the Internet, the initiative we are launching today promises to transform our ability to use Big Data for scientific discovery, environmental, and biomedical research, education, and national security,” says John Holdren, assistant to the President and director of the White House Office of Science and Technology Policy.

The National Institutes of Health and the National Science Foundation will be among the top recipients of funding, along with the Departments of Defense and Energy, and the U.S. Geological Survey. Besides advancing the development of technologies needed to collect, store, preserve, manage, and analyze the growing volume of digital data being generated on a daily basis, the initiative aims to harness the knowledge gleaned to advance scientific discovery, strengthen national security, and transform teaching and learning. Money will also be allocated to expand the skilled workforce needed to make sense of it all.

for more:
http://www.burrillreport.com/article-us_unveils_200_million_big_data_initiative_.html

GAO Sees Gloomy Fiscal Outlook without Health Reform

As the U.S. Supreme Court decides the fate of the Patient Protection and Affordable Care Act, a new report from the Government Accountability Office says that healthcare costs could soar if the law is overturned, further adding to the long-term challenge posed by the federal deficits and debt.

In its latest update of the federal government’s long-term fiscal outlook, the GAO makes projections based on two models, one baseline model that generally reflects current law, and another alternative model that assumes cost-containment mechanisms are not sustained over the long term. Even with the Affordable Care Act implemented as intended, the “structural gap between revenues and spending driven by rising healthcare costs and demographics” would not be closed, according to the GAO.
But spending on healthcare would grow “much more rapidly” in the alternative scenario, causing spending on Medicare and Medicaid to grow to more than 8 percent of GDP by 2030 from 5 percent of GDP in 2010. The baseline model finds Medicare and Medicaid growing to 7 percent under the baseline simulation, it says.

http://www.burrillreport.com/article-gao_sees_gloomy_fiscal_outlook_without_health_reform.html

The Burrill Report see page 5 of 35 page April report

Downturn Raises Profile of Government and Foundations as Sources of Early-Stage Funding

Grants and contracts can provide financing for ventures too risky for traditional investors

By Linda Molnar

At a time when early-stage life sciences companies face challenges raising money, they are increasingly finding the federal government and foundations can be good sources of non-dilutive funding. This was not always the case. As recent as five years ago, there was a large disconnect between these sources of funding and traditional venture capital, let alone entrepreneurs themselves.

The deep recession that began in 2008 and the significant fall-off in early-stage venture deals and investments have helped to change this. The venture capital world has still not returned to the levels of funding of early-stage life sciences companies of the past years and may never return to those levels given the changes the venture capital industry is undergoing today.

Groups Seek Congressional Action on Drug Shortages - 03/23/2012 - The Burrill Report

Groups Seek Congressional Action on Drug Shortages - 03/23/2012 - The Burrill Report

PUBLIC HEALTH

Groups Seek Congressional Action on Drug Shortages

Discounted generic drug application fees proposed as incentive to curb shortfalls.

MICHAEL FITZHUGH

The Burrill Report

“We believe appropriate mechanisms, such as a generic user fee program, could provide a crucial economic tool for FDA to provide incentives to manufacturers, and should be enacted as quickly as possible”

A group of more than 30 organizations seeking to address drug shortages in the United States has written a letter urging Congressional action to solve the problem.

The letter, sent to Senator Tom Harkin, D-Iowa, by groups representing clinicians, healthcare facilities, drug manufacturers, and patient advocates, urges Congress to consider implementing a generic user fee program in the upcoming Prescription Drug User Fee Act reauthorization, or PDUFA.

 The fees could be used to allow the U.S. Food and Drug Administration to “offer reduced application fees for products in short supply, or discounted fees if a company demonstrates that its contingency plans are sufficient to reduce the risk of a shortage if production is halted,” they suggest.

for complete article, please visit the Burrill Report link:
Groups Seek Congressional Action on Drug Shortages - 03/23/2012 - The Burrill Report