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NIH Announces Genome Sequencing Program for Common and Rare Diseases

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Last week, the National Human Genome Research Institute (NHGRI), a division of the National Institutes of Health, launched the Centers for Common Disease Genomics (CCDG), in order to target the genomic bases of common diseases such as heart disease, diabetes, autism and stroke. NHGRI also announced the creation of the Centers for Mendelian Genomics (CMG), which will do the same genomic investigations with rare diseases, like cystic fibrosis and muscular dystrophy.

The research wing at NIH plans to give $240 million to the CCDG to identify the genetic factors in the common health issues, with another wing of NIH giving an additional $20 million.  CCDG plans to divide the total $260 million amongst Washington University, Baylor College of Medicine, the Broad Institute, and New York Genome Center, where the research teams will use another $40-80 million in additional NIH funding to map the genomes of tens of thousands of people with their population-scale sequencing techniques.

The Mission of the Centers for Common Diseases Genomics

Currently, the highly-targeting diseases are cardiovascular, metabolic and neuropsychiatric diseases. Inflammatory/autoimmune, bone/skeletal, and Alzheimer’s disease could all possibly be targeted as the program progresses. For each disease, the CCDG researchers will sequences over tens of thousands of genomes. By looking into genes that may trigger or inhibit development of diseases, CCDG may be able to use the genomic data comparing those with and those without the disease to pave way for improved treatment and prevention options.

Adama Felsenfeld, Ph.D., director of the NHGRI Genome Sequencing Program, said, “Building on existing research, they will continue to uncover new biological insights into the development of common disease. At the same time, these studies will reveal genomic variants that may increase the risk for – or in some cases, protect against – diseases, which eventually might be helpful for their clinical management.”

The 4-year NIH funding will go towards analyzing the existing sequencing, for as Richard Wilson, the principal investigator at Washington University said, “right now we can generate more data than we have the ability to analyze.” The Genome Sequencing Program Coordinating Center will use about $4 million on data analysis as well.

The Mission of the Centers for Mendelian Genomics   

Running in parallel to the common diseases genome sequencing drive, the CMG will account for analyzing the genetic makeup of rare diseases with the remaining $49 million. The National Heart, Lung, and Blood Institute (NHLBI) will contribute to both CCDG and CMG, while the National Eye Institute (NEI) will contribute to CMG alone.

The CMG program was started in 2011 with the goal of identifying the genomic causes of Mendelian diseases, rare disorders usually caused by single-gene mutations. Over the past four years, more than 20,000 human genomes have been sequenced and analyzed, and over 740 genes have been found that likely cause Mendelian diseases.

Lu Wang, Ph.D., the director of the CMG program noted, “Rare diseases provide an important window into the biology of both rare and common diseases.” By working alongside the CCDG, CMG researchers will continue to sequence the human genome to find the specific disease-causing genes.

 

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As research in the human genome mapping project grows, so does the potential to find treatments against both common and rare diseases. From that point, clinical trials are then exercised in order to test the safety and efficacy of hopefully life-saving treatments. Clinical trials are unarguably of great value in advancements in biotechnological and pharmaceutical fields. RESolutions, LLC is dedicated to patient recruitment so that you may enroll your trial on-time and on-budget. If you would like to learn more about our services or would like to speak with a representative directly, Contact Us today!

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Clinton Proposes Tax to Fight Increasing Pharmaceutical Drug Prices

Hillary Clinton speaks in Washington

Reinvesting Funds from Advertising to Research

In response to the steep increase in drug prices, Presidential hopeful Hillary Clinton is proposing new rules for pharmaceutical drug companies requiring them to set aside a budget specifically on research and development, rather than marketing and advertising. Because marketing and advertising subsidies are corporate write-offs, companies are able to count those large sums as a tax-deductible business expense. Direct-to-consumer advertising increases prescription drug costs, exactly what Clinton is trying to avoid with her proposed plan.

(Martin Shkreli’s Turing Pharmaceuticals recently raised the price of Daraprim, a 60 year old drug, from $13.50 to $750/pill, over 4000% higher than the original cost. Turing Pharmaceuticals being the only FDA-approved manufacturer of Daraprim, Shkreli was able to control the price without legal opposition.)

“One of out every four people facing higher drug costs were also unable to afford medical bills or medications; one in five said they missed a payment on a major bill,” a Consumer Reports surveyed earlier this year.

The current corporate write-offs would be used instead to invest in paying for the R&D tax credit so that research on future drugs is emphasized. Successful marketing influences the consumer to spend money on the product. By increasing marketing funds, the pharmaceutical companies increase their drug prices in order to reach higher profits. If the budget on marketing was focused elsewhere, Clinton believes that drug companies would not be influenced by the market, thus ending the cycle of increased advertising and skyrocketing drug prices.

“Exit” Tax Strategy

This comes out weeks after Pfizer’s announcement that it would merge with Allergan, moving Pfizer’s New York headquarters to Allergan’s headquarters in Dublin, Ireland. Ireland’s lower corporate tax rate will cut Pfizer’s taxes from 25% to 18% – if applied in 2014, Pfizer would have saved nearly $1 billion of the $3.1 billion in US taxes.

 

“They’re doing it to save money on taxes. I want the Treasury Department to do everything it can to stop that kind of behavior and call it for what it is: gaming the tax system,” Clinton said about the $160 billion merger.

 

Overseas profits are not taxed until they are brought back into the US, but these taxes can be avoided by holding cash and investing abroad. Experts estimate that about 2 trillion dollars are hoarded abroad to reduce taxes. Corporate inversions allow for the overseas merger, an overall practice that lowers the US drug companies’ tax bills.

 

Currently, US companies can merge overseas if they transfer more than 20% of their shares to the foreign company they have acquired. Supporting the Obama administration’s proposal of raising the shares to more than 50% in order to discourage similar inversion deals, Clinton supports the change.

Rolling Out Proposals

Clinton’s campaign has been releasing her economic agenda, including increasing infrastructure spending by $275 billion and a higher focus on research and clean energy investments. The revenue gained by reinvesting drug funds into R&D and by employing the “exit” tax strategy is proposed to be spent on an increase in manufacturing jobs in the US.

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Any push on drug research and development would further serve both the drug industry and the general population. Clinical trials are unarguably of great value in advancements in biotechnological and pharmaceutical fields. RESolutions, LLC is dedicated to patient recruitment so that you may enroll your trial on-time and on-budget. If you would like to learn more about our services or would like to speak with a representative directly, Contact Us today!

Study Examines Major Drug Firms’ Disclosure of Clinical Trial Data 

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Study Examines Major Drug Firms’ Disclosure of Clinical Trial Data

A new study from a non-profit group focused on drugmakers’ ethical performance examines how well or poorly the largest pharmaceutical firms performed in making available data from the clinical trials they sponsored for drugs that won regulatory approval from the U.S. Food and Drug Administration (FDA) in 2012.

Published in the journal BMJ Open, the study done by several prominent bioethicists for Bioethics International examined how much information was made public on the clinical trials that supported 15 new drugs developed by major firms, looking at both legal and ethical requirements.

According to the study, 35% of clinical trials supporting new drug approvals in 2012 have not been either published in medical journals or included in the National Institutes of Health’s clinicaltrials.gov website.

Almost half of the drugs approved that year had at least one undisclosed Phase II or Phase III study. The researchers also found that for the FDA-approved drugs in 2012, only 20% of all supporting clinical trial results had been posted to a registry for trial results. Further, just 57% of trials had been registered and 56% had been described in medical literature.

The study also said its review found “wide variations” in company performance. At the upper end, the study found that GlaxoSmithKline (which in mid-2013 started a program to increase publication of company trial results), as well as Pfizer, Johnson & Johnson, made public all trial results for at least one of their drugs cleared by the FDA that year.

Coming in at the bottom were Gilead, which disclosed just 21% of trial results for its HIV cocktail drug Stribild, and Sanofi, which was similarly unforthcoming on trial results included in its application to the FDA for Aubagio, its once-a-day drug for relapsing multiple sclerosis in adults.

To encourage major drug firm to be more transparent and share trial results more broadly, Bioethics International also announced it had developed new rankings, the Good Pharma Scorecard, to assess how well the largest firms do in meeting the group’s disclosure expectations.

Jennifer Miller, Ph.D., Bioethics International president, medical ethics assistant professor at the Langone Medical Center at New York University, and the study’s lead author, says the new scorecard offers pharmaceutical firms an opportunity to gain greater public respect. She points out that the pharmaceutical industry, the most respected business sector a mere 17 years ago, is believed to be honest and ethical by only 12% of Americans today.

The new study stressed the critical importance of ensuring clinical trial results are disseminated widely, since medical care and drug therapies depend on having available full and accurate knowledge of clinical trial findings. In addition, it argued, the contributions and sacrifices of the many individuals who chose to participate in clinical trials in order to further medical knowledge may be in vain if the results of those trials are not made generally available.

Other study authors, such as Harvard Medical School pathologist Dr. David Korn, noted that nearly all of the major firms contacted during the study responded to requests for input, and many which had ranked low on the scorecard showed interest in ways to improve.

 

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Clinical trials are unarguably of great value in advancements in biotechnological and pharmaceutical fields. RESolutions, LLC is dedicated to patient recruitment so that you may enroll your trial on-time and on-budget. If you would like to learn more about our services or would like to speak with a representative directly, Contact Us today!

FDA Freezes Part of Controversial IND Guidance for Non-Drugs

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FDA Freezes Part of Controversial IND Guidance for Non-Drugs

The U.S. Food and Drug Administration (FDA) has at least for the time being decided not to enforce part of the controversial guidance document it issued in 2013 stating an Investigational New Drug (IND) application will be required for research studies on humans investigating potential bioeffects from such non-drug products as food, cosmetics and dietary supplements. The agency announced the change in a notice published in the Federal Register October 30.

In explaining the partial stay of its 2013 IND Guidance, the FDA said it had gotten “comments from trade organizations, individual companies, scientific associations, public interest organizations and individuals” which “raised questions about application” of requiring an IND application for some clinical studies of ordinary food, dietary supplement, and cosmetics products.

That bland statement understates the furor set off when the FDA’s Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research in September 2013 jointly issued guidance extending its IND application requirement beyond products already defined as a “drug” by the Federal Food, Drug & Cosmetic Act. The reaction was strengthened by the fact the new Guideline sections (VI.C and VI.D) claiming that authority had not appeared in the draft version proposed by the agency and submitted for public comment in October 2010. In fact, the draft made no mention of food or cosmetics; it contained only a brief discussion of dietary supplements.

The 2013 guidance document’s new sections asserted IND applications would be required for any clinical study done on humans, even of non-drug products, if the study sought to evaluate particular types of physiologic endpoints. That new agency interpretation that any study examining such factors required an IND application essentially transformed food, cosmetic or medical supplement products into the functional equivalents of a drug.

The guidance document, for example, said an IND would not be needed for a study of a food’s nutritional value, but would be required it the study focused on any effect the food might have on body structure or function, other than nutrition. Similarly, an IND would be required, the FDA maintained, for a study intended to support health claims for a non-drug product.

The 2013 guidance document did indeed draw many comments, primarily harsh ones directed at the added provisions. Some commenters claimed the expansion was counter-productive or contrary to the agency’s statutory authority. Others argued broadening the IND requirement was unnecessary, unduly expensive or likely to lead developers to move their research offshore.

The agency’s October announcement announces the agency intention not to enforce the IND requirement for studies designed to evaluate whether a food or dietary supplement might reduce a health risk, even if intended to support a health claim, unless the study involved children less than a year old, or persons with damaged immune systems or serious or life-threatening conditions. Food products would also not need an IND to study bioeffects other than nutrition.

Due to the initial outcry over the 2013 guidelines, FDA re-opened the comment period for two months in early 2014. The latest announcement of a partial stay says the agency will again take comment, for an unspecified period, on the provisions now at least temporarily in limbo. Opponents of the guidelines are likely to comment again, but also to continue to demand the stayed provisions be removed and the guidelines republished without them.

Patient Recruitment – RESolutions, LLC

Patient Recruitment, Patient Enrollment

Patient Recruitment

Patient recruitment incorporates an assortment of services—regularly performed by a Patient Recruitment Service Provider—to expand enlistment in clinical trials. In the blink of an eye, the patient recruitment industry is guaranteed to add up to $5.9 billion every year. Understanding enlistment is the most tedious part of the clinical trial process. The main source of missed clinical trial due dates is persistent enlistment, taking up to 30 percent of the clinical course of events. Enhancing persistent enrollment rates offer pharmaceutical and medicinal gadget organizations one of the greatest chances to quicken the pace of clinical trials – making it conceivable to diminish time to advertise. As the quantity of patients required for clinical trials rises – as well-being and administrative issues drive patterns toward bigger and more trials – the interest for patient recruitment services develops. Clinical trials are directed to gather information with respect to the well-being and viability of new medication and gadget improvement. They are directed in a progression of stages, each intended to address a different reason:

 

  • Phase I  

 

Scientists test other medication or treatment in a little gathering of individuals (20-80) surprisingly to assess its security, decide a sheltered dose range, and distinguish reactions.

 

  • Phase II  

 

The study medication or treatment is given to a bigger gathering of individuals (100-300) to check whether it is compelling and to further assess its well-being.

 

  • Phase III  

 

The study medication or treatment is given to substantial gatherings of individuals (1,000-3,000) to affirm its viability, screen reactions, contrast it with regularly utilized medicines, and gather data that will permit the medication or treatment to be utilized securely.

 

  • Phase IV  

 

Post showcasing studies outline extra data, including the drug’s dangers, advantages, and ideal use.

Pharmaceutical organizations submit trial information to the U.S. Nourishment and Drug Administration (FDA) as a major aspect of a New Drug Application, the application for FDA endorsement to advertise a medication in the U.S. Tolerant enlistment service suppliers instruct people in general about the estimation of clinical trial cooperation and the measures set up to ensure study members. The 2001 “Will and Why Survey” of more than 5,000 individuals in the U.S. demonstrated that 81 percent of the populace didn’t know about protections, for example, the Declaration of Helsinki, The Belmont Report, Institutional Review Boards, and the educated assent process. On the other hand, in the wake of finding out about these defensive measures, almost 40 percent of respondents reported they would will probably take an interest in a clinical trial.

Notwithstanding the improvement of a noteworthy cluster of enlistment strategies throughout the most recent decade, the test of enrolling patients for clinical trials has kept on developing at a disturbing rate. The disappointment of these new strategies to enough address the test has as much to do with their intrinsic confinements as it does with the absence of a far reaching procedure for applying them. Clinical Patient Recruitment is not a solitary movement, yet rather a large group of obligations that range from conventional advancement and study possibility to site choice and different exercises from First Patient In (FPI) to LPI.

Accomplishing LPI points of reference requires broad arranging and hard information, so we bolster these exercises with an arrangement of exclusive information resources and instruments, including an achievability database, overall site determination database, our Scenario Planning as well as Recruitment Calculator (SPARC), site following and preparation device, and site-particular enlistment arranges. These assets are intended to build the rate and viability of all clinical patient enlistment exercises.

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Clinical trials are unarguably of great value in advancements in biotechnological and pharmaceutical fields. RESolutions, LLC is dedicated to patient recruitment so that you may enroll your trial on-time and on-budget. If you would like to learn more about our services or would like to speak with a representative directly, Contact Us today!

Drug Making Use of Re-engineered Virus Okay-ed for Melanoma 

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Drug Making Use of Re-engineered Virus Okay-ed for Melanoma

The U.S. Food and Drug Administration (FDA) on October 27 approved an oncolytic cancer treatment, using a genetically modified infectious virus both to shrink tumors and to stimulate the patient’s immune system to fight the cancer.

The Amgen product, approved under the trade name Imlygic, and also known as talimogene laherparepvec, or T-VEC, uses a re-engineered form of a live herpes simplex virus, which in its original form causes cold sores, to target and destroy the melanoma tumor cells, and to boost the patient’s immune response. The drug also includes a cytokine protein, GM-CSF (for granulocyte-macrophage colony-stimulating factor, to draw immune system defenders to the tumor.

The FDA based its approval of the drug on a multicenter, randomized open-label Phase III clinical study known as OPTim, which enrolled 436 patients with advanced or surgically untreatable melanoma. Injecting Imlygic into participants’ visible tumors, the study compared the rate of partial or complete response lasting continuously over at least six months for patients who received Imlygic and those given just GM-CSF.

The study showed 16.3% of patients receiving Imlygic had such a response, compared with 2.1% of patients receiving just GM-CSF. Among patients who experienced a durable response of six months or more, about 30% showed a complete response and 70% a partial one.

The most frequent adverse reactions to the new drug were flu-like symptoms, chills, fever, fatigue, or nausea, generally of mild or moderate severity and lasting up to three days. The median time until response for patients getting Imlygic was about four months.

Despite the tumor eradication or reduction and relief patients experienced from the most lethal type of skin cancer, the new drug has not been shown to cure melanoma. The FDA, in approving Imlygic, also noted it has not yet been shown to extend the overall longevity of patients or to have any effect on melanoma patients in whom the disease has metastasized into their brain, bones, lungs, liver, or other internal organs.

Researchers are also still trying to gauge how broadly Imlygic promotes immune responses: whether it only targets cancer cells which its virus has invaded, or whether it stimulates immune responses to all cancer cells it encounters.

While similar drugs are in use elsewhere in the world, Imlygic is the first oncolytic virus cancer treatment to win FDA approval. A number of other oncolytic virus treatments are currently in development, using several others viruses, and researchers hope this may be the start of a whole new class of treatments to add to the cancer-fighting arsenal.

Amgen is also exploring whether Imlygic would be even more effective if combined with Bristol-Myers Squibb’s checkpoint inhibitor Yervoy, which like other drugs of its class, aims at removing cancer cells’ chemical shields that prevent the immune system from attacking them. In early results from a clinical trial of the combination of Imlygic and Yervoy, about half of a very small test group showed positive responses to the combined treatment. A trial of Imlygic with another checkpoint inhibitor, Merck’s Keytruda, is also planned.

 

Enroll Your Clinical Trial On-Time and Efficiently By Contacting Us Today!

Clinical trials are unarguably of great value in advancements in biotechnological and pharmaceutical fields. RESolutions, LLC is dedicated to patient recruitment so that you may enroll your trial on-time and on-budget. If you would like to learn more about our services or would like to speak with a representative directly, Contact Us today!

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