A Roadmap to Clinical Trial Efficiency

This article will discuss the process and roadmap needed for optimizing clinical study and trial and review why and how are the Trials being performed.

Clinical study is a research which involves human volunteers, also called as participants, with an intention to add to the medical knowledge.

Mainly there are two types of clinical studies, namely: Clinical Trial (aka Interventional Study) and Observational Study.

Clinical studies are the experimental studies to determine a better treatment of a particular disease. They are designed to answer specific scientific questions like:

• What side effects do the drug have?
• Does it reverses or stops the disease for which it is designed to heal?
• Are the outcomes effective than existing treatments?
• Can it be given along with the drug which is already being given for the same disease?

The answer to every question above must come in the form of scientific data and not in the form of opinions or the impressions of the scientists, doctors or patients involved with the studies.

The results can be enclosed by many factors and can be affected by bias. It is to be made sure this does not happen.

What is a Clinical Trial?

Clinical Trials are clinical studies for exploring a medical strategy, devices or a treatment, which is safe as well as effective for humans.

Also called as interventional study, clinical trial is a clinical study where participants are assigned to receive one or more inventions or  sometimes no inventions at all, for the researchers to evaluate intervention's effect on health related outcomes. For certain illness in humans or a particular group of people, these studies help to find out which medical approach will work better to their cure.

What is a Clinical Trial Intended for?

The main purpose of performing a clinical trial is nothing but a research and so strict scientific standards are followed . These standards are required to be maintained for the protection of patients and generate study results which are reliable.

How are the Clinical Trial Approved?

Before starting a Clinical Trial, the protocol or the plant of the trial is approved by an independent and unbiased group of researchers who are not going to e involved in the trials, which is called an independent scientific review.

The trial protocol is reviewed by National Research Ethics Service, which is a Research Ethics Committee. The aim is to be concerned with the rights, safety, dignity and well-being of the patients taking part in trials. This committee is made up of 7 and 18 volunteers.

The researchers are bound with the protocol they proposed at the beginning of the trial. they cannot change the protocol without approval from ethics committee. They have to report to the ethics committee in case of any happening of serious side effects in-between trial. The committee receive reports at the end of clinical trial.

How is the Clinical Trial Performed?

The process of a clinical trial begins in the laboratory. The scientists develop and test the ideas regarding the tests. Clinical trials are performed as a part of one of the final stages of a lengthy and carefully performed research procedure.

If the basic idea of clinical research is worth promising, animal testing is performed as the next step.

• Why are animals used for clinical testing?

           Using animals for clinical tests is because there is a need to find out what happens to a living              body. Replacing animals with any other alternative method of testing samples is not yet                        discovered. As the basic cell formations of animals are similar and human's body performs                  same tasks of movement, breathing, hearing, digestion, sight and reproduction, it is likely to                perform tests on them rather experimenting them on humans first. Before testing ideas on                    humans, animals such as rats are used for sampling.

• What does the animal testing indicate?

          It shows that how the biomedical approach will affect a living body and whether it is harmful               or not.

However, it may be possible that the approaches working well in labs or on animals are not necessary to show exact results in case of humans. Hence, it  is required to perform tests on humans.

As a safety measure, clinical trials are performed on a small group of people on initial stage and it is found out whether it affects or harms humans or not. The later phases of clinical trials involve larger amount of humans on which the study is focused.

      Clinical Research Coordinator : Educational Requirements and Career Options Transcript

Are you a whiz when it comes to organization and planning? Consider a career as a clinical research coordinator. These professionals manage medical and scientific research projects that investigate new medicines, therapies and treatments. A bachelor's degree in a scientific field and professional certification are required for most positions. Armed with these credentials you can also pursue positions as a clinical data management professional or a clinical research assistant.

Introduction

Clinical research studies have been responsible for groundbreaking advances like cancer-fighting chemotherapy drugs and the polio vaccine. Organizing a clinical research study requires meticulous planning and attention to detail. Duties include recruiting test subjects, scheduling appointments, collecting data and more. A bachelor's degree in Chemistry, Biology or a related field is needed along with a professional certification.

Job Duties and Skills

Testing a new medical treatment or prescription drug requires the combined efforts of many health care professionals. Clinical research coordinators are the professionals who oversee this daunting task.

Clinical research coordinators begin by finding test subjects and securing a test site, usually in a hospital, clinic or lab. During testing, the coordinator must assist with the testing process, provide medications, and answer questions from testers and subjects. After completion of the study, the coordinator is responsible for working with data management professionals to organize the data for analysis and report.

Clinical research coordinators must be organized and able to focus on many small tasks without losing sight of the big picture. Attention to detail is essential, as even the slightest error can contaminate test results. Excellent communication skills are also needed to work with doctors, nurses, research scientists, and the other professionals involved with a clinical research study.

Training Required

Employers look for candidates who have completed at least a bachelor's degree in a scientific field, such as biology, chemistry or biomedical engineering. These programs include coursework in mathematics and the sciences, as well as analytical research and laboratory skills. However, due to the administrative demands of the profession, employers also look for research and teaching experience. Many students gain these experiences in a master's degree program.

Professional certification is required for many positions. The Association of Clinical Research Professionals (ACRP) and the Society of Clinical Research Associates (SoCRA) both offer clinical research coordinator certifications. These certifications require periodic renewal through continuing education courses. Associate's and professional certificate programs exist to help scientific professionals prepare for the licensing examination. Such programs normally take six months to a year to complete and include coursework focusing on health care legislation, research guidelines and methodologies and medical terminology.

Career Options

Clinical research coordinators are typically employed by research hospitals, universities, pharmaceutical manufacturers and other health care organizations. Many employers look to promote clinical research coordinators to management positions because of the unique blend of scientific, technical and administrative skills that they possess. However, most opportunities for advancement will require a master's degree and some employers may require a doctoral degree.

Clinical research coordinators do not necessarily need to work in medical research. Other opportunities exist in industrial, chemical and technological research companies as well. Depending on their scientific and technical backgrounds, clinical research coordinators may find positions managing research laboratories or coordinating the efforts of teams of research scientists in a variety of industries with far reaching applications.

With their strong scientific background, research coordinators may work as research assistants or research technicians. Due to growing interests in medical research and increased need for new medicines, positions in government regulatory agencies like the Food and Drug Administration (FDA) will also be available.

Adverse Event Reporting

The activity that is most commonly associated with Pharmacovigilance (PV), and which consumes a significant amount of resources for drug regulatory authorities (or similar government agencies) and drug safety departments in pharmaceutical companies, is that of adverse event reporting. Adverse event (AE) reporting involves the receipt, triage, data entering, assessment, distribution, reporting (if appropriate), and archiving of AE data and documentation. The source of AE reports may include: spontaneous reports from healthcare professionals or patients (or other intermediaries); solicited reports from patient support programs; reports from clinical or post-marketing studies; reports from literature sources; reports from the media (including social media and websites); and reports reported to drug regulatory authorities themselves. For pharmaceutical companies, AE reporting is a regulatory requirement in most countries. AE reporting also provides data to these companies and drug regulatory authorities that play a key role in assessing the risk-benefit profile of a given drug. The following are several facets of AE reporting:

Origin of pharmacovigilance

A new breakthrough in this field only happened after an episode occurring in 1937. In that year, Sulfanilamide (Prontosil), used since 1932 for treatment of streptococcal infections, was launched as a syrup, containing diethyleneglycol as solvent. Although tested regarding aspect, taste and odor, its safety was not evaluated before launching. It was responsible for the death of 105 individuals (34 children and 71 adults) and diethyleneglycol was incriminated. This tragedy caused the American Congress to approve in 1938 the Food Drug and Cosmetic Act, under which pharmaceutical product manufacturers would have to show scientific evidences of the safety of the drugs before releasing themfor sale.

The thalidomide tragedy is a milestone in the origin and development of pharmacovigilance. Thalidomide was introduced in 1957 and widely prescribed as an allegedly harmless treatment for morning sickness and nausea. It was tested in approximately 300 patients without toxicity. It was soon linked to a congenital abnormality phocomelia, which caused severe birth defects in children of women who had been prescribed this medicine during pregnancy. In 1962, after reports of numerous cases of phocomelia, it was discontinued. In the same year, the Kefauver-Harris amendment was approved, requiring scientific evidences of efficacy and safety before drug tests in humans. ^[2] 

As a means of pooling existing data on ADRs, WHO's Programme for International Drug Monitoring was started in 1968. Initially a pilot project in 10 countries with established national reporting systems for ADRs, the network has since expanded significantly as more countries worldwide developed national pharmacovigilance centers for the recording of ADRs. Currently, 86 countries participate in the programme, which is coordinated by WHO together with its collaborating centre in Uppsala, Sweden. The collaborating center is responsible for maintaining the global ADR database, Vigibase. At present the database contains more than four million ADR reports

History of pharmacovigilance in India

The origin of pharmacovigilance in India goes back to 1986, when a formal adverse drug reaction (ADR) monitoring system consisting of 12 regional centers, each covering a population of 50 million, was proposed for India. However, nothing much happened until a decade later when in 1997, India joined the WHO Adverse Drug Reaction Monitoring Program me based in Uppsala, Sweden. This attempt was unsuccessful and hence, from 1January 2005, the WHO-sponsored and World Bank-funded National Pharmacovigilance Program for India was made operational. 
The National Pharmacovigilance Program established in January 2005, was to be overseen by the National Pharmacovigilance Advisory Committee based in the Central Drugs Standard Control Organization (CDSCO), New Delhi. Two zonal centers-the South-West zonal center (located in the Department of Clinical Pharmacology, Seth GS Medical College and KEM Hospital, Mumbai) and the North-East zonal center (located in the Department of Pharmacology, AIIMS, New Delhi), were to collate information from all over the country and send it to the Committee as well as to the Uppsala monitoring center in Sweden. Three regional centers would report to the Mumbai center and two to the New Delhi one. Each regional center in turn would have several peripheral centers reporting to it. Presently there are 26 peripheral centers. The program has three broad objectives: the short-term objective is to foster a reporting culture, the intermediate objective is to involve a large number of healthcare professionals in the system in information dissemination and the long-term objective is for the program to be a benchmark for global drug monitoring.

Given this background on pharmacovigilance in India to date, things have definitely changed for the better but at a very slow pace. The Regulatory Authority for India should be commended for introducing and implementing the Schedule Y and for reporting of all serious adverse events (SAEs) including suspected unexpected serious adverse reactions (SUSARS) from clinical trials. However, there is a need of spontaneous adverse event reporting from post-marketed medicines to the zonal centers and in turn to the National Pharmacovigilance Centers to the WHO Uppsala Monitoring Center, which at the moment is woefully lacking. Therefore, in these circumstances, the questions that arise are whether the strategy should be changed and if so, how?

Ethical issue in Clinical Research

History of Clinical Research

PLACEMENT TALK

Mr. Prashanth Krishna, Head Healthcare - IDS Infotech and Mr. Ravinder, Manager IDS- ARGUS were here to discuss career opportunities with our students............ the session was very informative & students had a chance to get the queries resolved ..... got some clicks from the event...........we are grateful to IDS for their time and knowledge they shared with us.

Indian researcher bags award for Alzheimer’s drug

London: London-based Indian researcher Mahaveer Golechha has been selected for an award for his work on a drug to cure Alzheimer’s. 
    The 27-year-old will be travelling to San Francisco to receive the Alzheimer’s Drug Discovery Foundation’s ‘Young Investigator Scholarship Award’ at the 7th Annual Drug Discovery for Neurodegeneration Conference to be held next month. 
    “I feel very proud that I have proved world class research in the area of medicine can be done in India,” Dr Golechha said on Thursday. 
    He has been recognized for his outstanding research on ‘naringin’, a bioflavonoid found in grape fruits and citrus fruits. In his study, he found that naringin possesses significant “anti-Alzheimer activity” and will form the basis of further research to develop as a cure for the degenerative disease in old age, which worsens as it progresses. 
    “Alzheimer’s disease is a neurodegenerative disorder that generally affects the elderly population. Till date the treatment was symptomatic but naringin will act at pathological level and have lesser side effects,” he said. 
    “I hope this award creates further avenues for budding scientists in my country to be recognized for their work and facilitated to follow their dreams,” said Golechha, who completed his Master’s and PhD from the All India Institute of Medical Sciences in New Delhi, where he was involved in research on Alzheimer’s and epilepsy. 
    His work on naringin has also been included for a poster presentation at the San Francisco conference. PTI

India to inspect drug firms in China: DCGI Inspection Office To Open In Beijing By Mar 1 Kounteya Sinha TNN

New Delhi: Increasing the heat on Chinese drug firms exporting medicines to India, the Drug Controller General of India (DCGI) is all set to open its first foreign drug inspection office in Beijing by March 1. 
    Around four Indian drug inspectors will be posted in China to inspect manufacturing sites and check whether good manufacturing practices (GMP) are being complied with. DCGI Dr G N Singh told TOI that the commerce ministry has cleared the proposal. 
    Health minister Ghulam Nabi Azad recently said that in the last two years, 10 Chinese bulk drug manufacturing firms were inspected and the registration certificate of one firm and 16 import licences were cancelled. 
    Ministry sources said the decision to audit Chinese drug manufacturing units was taken after several import licences of local agents were cancelled due to poor drug quality and their failure to comply with GMP. 
    “We will post Indian drug inspectors in China to send a clear message that Chinese drug firms are under watch. Gradually, China will be asked to only allow drugs exports from manufacturing sites that have been inspected by Indian drug inspectors. We want to ensure safety, efficacy and quality of Chinese drugs,” said Dr Singh. 
    “GMP of Chinese drug firms has to be certified by our regulators. This is a practice followed in most countries, including the FDA in the US. To protect the interest of Indians, we have to go international,” he added. 
    Now, more than 45% of bulk drug exporters registered in India are from China. The number of registered Chinese bulk drug manufacturers in India is around 280, and altogether 417 different drugs from the Asian giant are registered. 
    A ministry official said, “We had earlier written to Chinese Food and Drug Authority (FDA) regarding complaints that some Chinese drug firms which export bulk drugs to India might not be holding proper GMP certificates. The Chinese FDA too confirmed our apprehensions and asked us to carry out our own inspections.” On the basis of complaints and doubts on authenticity of GMP certificate, India has already cancelled 10 registration certificates and related import licences. These certificates were from Zhejinag, Jingsu, Henan province and Chongquing. 
    A DCGI note said, “Similarly, several cases of imported kits of HIV were declared to be not of standard quality by the government laboratory, which are originating from Zhejiang and Fujian provinces. These issues further support this office which stands to carry out foreign site inspections in line with other regulatory agencies of the world.”

More than 45% of bulk drug exporters registered in India are from China

Coffee Drinking May Halve Risk Of Mouth And Throat Cancer Featured Article Academic Journal Main Category: Cancer / Oncology Also Included In: Nutrition / Diet; Dentistry Article Date: 12 Dec 2012 - 3:00 PST

A new study from the US finds people who drink more than 4 cups of caffeinated coffee a day have half the risk of dying from oral/pharyngeal (mouth and throat) cancer as people who drink it either occasionally or not at all. However, the researchers say their findings need to be confirmed by more research, and for now should just be received as good news for coffee drinkers and not be used as a reason to recommend everyone should drink 4 cups of coffee a day.

Lead author Janet Hildebrand and colleagues from the American Cancer Society (ACS) in Atlanta, Georgia, write about their findings in a paper published online first on 9 December in the American Journal of Epidemiology.

Oral/Pharyngeal Cancer

Oral/pharyngeal or mouth and throat cancer is rarely diagnosed in the early stages because symptoms usually do not appear until the cancer is advanced. Also, the symptoms can be mistaken for something else, such as toothache.

The cancer can sometimes be spotted early during a routine exam by a doctor, dentist, or dental hygienist, and some dentists and doctors suggest you look at your mouth in a mirror at least once a month to check for symptoms.

The most common symptoms are a mouth sore that fails to heal, or a pain in the mouth that doesn't go away.

The biggest risks for developing oral/pharyngeal cancer are tobacco and alcohol use. Most people who have it are tobacco users.

Infection with the human papillomavirus (HPV) is also a risk factor, especially in people who do not use tobacco. The number of cases of oral/pharyngeal cancer tied to HPV has risen sharply in the last thirty years.

Researchers Examine Link with Coffee

Previous epidemiological studies have suggested coffee drinking is linked to a reduced risk for mouth and throat cancer.

It has also been suggested that it may not be the caffeine in coffee, but the fact it is rich in antioxidants, polyphenols, and other compounds, that help prevent or slow the development of cancer.

For their study, Hildebrand and colleagues used data from the Cancer Prevention Study II, a prospective US cohort study that the ACS started in 1982.

That study gathered a wealth of lifestyle and health information on 968,432 men and women, including their tea and coffee consumption. When they enrolled on the study, none of the participants had cancer, but over the 26 years of follow up, 868 died from oral/pharyngeal cancer.

When they analyzed the tea and coffee consumption in relation to deaths from oral/pharyngeal cancer, the researchers found those participants who reported drinking more than 4 cups of caffeinated coffee a day had a 49% lower risk of death from oral/pharyngeal cancer compared to those who reported not drinking coffee at all or only an occasional cup.

The link was not affected by gender, tobacco and alcohol use.

The researchers found an insignificant link with decaffeinated coffee, and none at all with tea.

Conclusion and Next Step

The researchers conclude:

"In this large prospective study, caffeinated coffee intake was inversely associated with oral/pharyngeal cancer mortality. Research is needed to elucidate biologic mechanisms whereby coffee might help to protect against these often fatal cancers."

Hildebrand says in a press statement:

"We are not recommending people all drink 4 cups of coffee a day. This is just a little bit of good news for those of us who enjoy coffee."

"There may be some other effects of coffee that may prevent people with certain conditions from drinking a lot of caffeine," she cautions, noting that:

"This study is about just one cancer site among many. There needs to be much more consistent research before we can support the conclusion that coffee should be consumed for cancer prevention."

The team is now planning to analyze links between coffee consumption and cancer in a more diverse population in the ACS's new Cancer Prevention Study - 3 (CPS-3).

The Society hopes to recruit at least 300,000 adults from a range of ethnic and racial backgrounds across the US to take part in CPS-3, which aims to increase knowledge of how to prevent cancer.

There has been a lot of debate recently about the benefits and harms of coffee drinking, with more recent news suggesting the benefits probably outweigh the harms.

But researchers spreading the good news are all saying the same thing, as Hildebrand and colleagues themselves point out in this latest study: while there appear to be some health perks from drinking coffee, there are also a few cautions, and the evidence is not solid enough to actively encourage people to go out and drink coffee.

Written by Catharine Paddock PhD 
Copyright: Medical News Today 
Not to be reproduced without permission of Medical News Today 

4 medical devices that could improve trauma care and save lives December 14, 2012 9:11 pm by Stephanie Baum Read more: http://medcitynews.com/2012/12/4-medical-devices-that-could-improve-trauma-care-and-save-lives/#ixzz2FHd0mzaD

The devastating shootings at the Sandy Hook Elementary School in Newtown, Connecticut were a grim reminder that technology developed for combat injuries can be just as critical for non-military situations. Here are four medical devices, some under development for the military and others cleared by the U.S. Food and Drug Administration, that could have a significant impact on saving patients with traumatic injuries.

BleedingZ-Medica initially developed its QuikClot technology to control bleeding for military use but Ron Clark, an emergency medical room physician at the Hospital of Central Connecticut’s Emergency Department, said in an e-mail that the emergency room at the hospital began using the material this year and added that other hospitals have begun using the material.

“I have used it about 10 times for head, extremity, chest and abdominal wound bleeding with very good results,” he said. “It works by using Kaolin to promote clotting and when someone is bleeding out, it works very well.”

Internal bleeding Arsenal Medical is developing a foam that can be injected to control intra-abdominal hemorrhaging. It’s injected at the site of the injury before the injured person is transported to a hospital unit. It compresses the wound internally and conforms to the contours of the internal organs around the injury site. It’s designed to expand the “golden hour” during which trauma patients can be effectively treated by up to three hours and reduce the number of deaths from these wounds. It received a $15.5 million Phase 2 contract from theDefense Advanced Research Projects Agency to develop the medical technology for the mild Brain injury Medical device company InfraScan’s handheld hematoma detector secured 510(k) clearance from the FDA last year. Originally used for combat troops in Afghanistan, the device uses near infrared technology to screen patients for intercranial bleeding in up to two minutes. It acquires EEG data wirelessly and can be used with an Android phone.Hemorrhage control Canadian medical device startup Innovative Trauma Care developed a clamp to control severe bleeding in as little as five seconds. It seals the skin to create a temporary clot to provide stability until the wound can be surgically repaired, according to the company’s website.

Read more: http://medcitynews.com/2012/12/4-medical-devices-that-could-improve-trauma-care-and-save-lives/#ixzz2FHdUi9lA

BOSTON, MA, December 04, 2012 – Perceptive Informatics, a leading eClinical solutions provider and a subsidiary of PAREXEL International Corporation (NASDAQ: PRXL), today announced that its ClinPhone® Randomization and Trial...

BOSTON, MA, December 04, 2012 – Perceptive Informatics, a leadingeClinical solutions provider and a subsidiary of PAREXEL International Corporation (NASDAQ: PRXL), today announced that its ClinPhone® Randomization and Trial Supply Management (RTSM) technologies have been used in over 3,000 studies involving more than 289,000 sites and 1.7 million patients, helping pharmaceutical sponsors to achieve more efficient data collection, faster patient enrollment and more streamlined trial supply management.

“The pressures on the pharmaceutical industry to improve the productivity of the drug development process are greater than ever before, and our goal is to enable clients to successfully meet those challenges by maximizing the value of their clinical trial technologies,” said Nick Richards, Vice President, Product Management, Perceptive Informatics. “Our deep experience is creating significant competitive advantage for us and, going forward, we remain committed to the continuous improvement of our offering based upon this expertise.”

Built on the leading-edge PAREXEL MyTrials™ platform  and combined with PAREXEL’s extensive medical and clinical expertise, ClinPhone RTSM technologies offer validated randomization methods, automated inventory control, dispensing and titration management, emergency code break capabilities and real-time supply reporting. The solutions provide sophisticated reporting to monitor trial management performance, trending and forecasting information, and in-depth data analysis. Clinical trials worldwide can be supported in multiple languages through the solutions, which provide secure access, detailed audit trails, transaction logging and compliance with FDA 21 CFR Part 11.

In addition to the ClinPhone RTSM solution, the comprehensive eClinical portfolio from Perceptive Informatics includes Electonic Data Capture (EDC), Clinical Trial Management Systems (CTMS) and Medical Imaging as well as Electronic Patient Reported Outcomes (ePRO). The PerceptiveeClinical Suite provides convergence among systems for real-time data interchange and enables diverse applications to work synergistically.

For more information about the ClinPhone RTSM technologies from Perceptive Informatics, visit www.perceptive.com/ClinPhone-RTSM.

Ground-breaking Liver Cancer Treatment NOVEMBER 26, 2012 BY CLAIRE AL-AUFI

Surgeons in the UK have pioneered a new treatment for cancer that is both unique and promising. The treatment involved isolating a cancerous liver by cutting off its blood supply and at the same time, dousing the organ with a “chemo bath.”

The procedure enables saturation of the tumours with chemotherapy while restricting the effects solely within the infected organ. Brian Stedman, is the consultant radiologist at Southampton General Hospital, who led the team that used the procedure, known as Chemosaturation with Percutaneous Hepatic Perfusion (CS-PHP). There were two patients who underwent the operation because their livers had become cancerous.

A cancerous liver is one where mutated cells grow and take over healthy cells. It is called primary liver cancer if it begins there, but metastatic if it spreads from outside, as with eye melanoma. Around 21, thousand Americans contract primary liver cancer every year. It is unusual in being a cancer on the increase. There are no obvious symptoms, which makes early diagnosis a matter of luck rather than screening. Men are two times more likely to suffer from it than women.

Symptoms to be aware of with advanced stage liver cancer might include tiredness, abdominal bloating, pain high up on the right of abdomen, in your back or shoulder area. Also nausea, loss of appetite, feeling full while losing weight, weakness, fever, and yellowing of the eyes and the skin.

This new CS-PHP treatment was developed in Europe and used successfully in America, Germany, Ireland, Italy and France. A study published in the spring, out of the Moffitt Cancer Center in Tampa, reports that the cancer sufferers that underwent the procedure lived five times longer than patients who underwent traditional treatment.

There are several cancers that invade the liver from original tumors in other organs. Melanoma of the eye is one such. Cancer of the liver is virtually untreatable and four months is the average longevity with only 10% of sufferers living past a year.

CS-PHP significantly adds to the life of melanoma patients while improving the quality of life since the disease progresses no further. The chemotherapy cocktails are infused straight into the liver using a catheter. Blood in the veins, that normally would enter the liver, is diverted and filtered through a purpose made, ‘double-balloon’ catheter to remove all traces of the drug before returning to the organ.

The new methodology enables higher concentrate drugs to be targeted on the liver. It gives maximum impact with minimum ‘collateral damage’. Dr. Stedman comments on the technique,

”To cut off an organ from the body for 60 minutes, soak it in a high dose of drug and then filter the blood almost completely clean before returning is truly groundbreaking.”

He contrasted the new and more traditional treatments where the prognosis for sufferers is dire because of the awful side effects of even low concentrate chemotherapy that wreaks havoc on the whole body. There are possibilities for CS-PHP to be used as a treatment method for other tumors such as in the colon or breast.

Novartis drug helps patients with rare inflammatory diseases

FMF, TRAPS are rare diseases

* Drug reduced attack frequency by 50 pct in FMF patients

* Relieved symptoms in TRAPS patients for average 3 months

ZURICH, Nov 11 (Reuters) - Novartis' Ilaris helps reduce patients' symptoms and the frequency of attacks in two rare inflammatory diseases, mid-stage studies showed, as the Swiss drugmaker looks to expand the use of the medicine.

Results of two separate studies on Sunday in patients with Familial Mediterranean Fever (FMF) and TRAPS - rare genetic diseases which can cause fever, rash and joint pain - both met their primary endpoints, Novartis said in a statement.

Both studies are being presented at the American College of Rheumatology (ACR) meeting in Washington D.C.

Ilaris or ACZ885, which blocks a protein called interleukin-1 beta that is thought to increase inflammation, is already sold for treating cryopyrin-associated periodic syndromes, a rare inflammatory disorder.

Novartis is also hoping to file the drug this year for regulatory approval in systemic juvenile idiopathic arthritis (SJIA), a debilitating disease that can affect a child's growth.

Results of the phase II study showed the drug helped 100 percent of FMF patients reduce the frequency of attacks by at least 50 percent during three months of treatment.

Eight of the nine patients in the trial did not have an attack during the three months and blood markers of inflammation also normalised.

There are currently no approved treatments for FMF or TRAPS, rare genetically-inherited anti-inflammatory diseases, which can affect both children and adults.

Novartis is hoping to show the drug can be beneficial in treating rare inflammatory diseases after receiving a setback last year when U.S. health regulators rejected Ilaris for use in gout over concerns about side effects.

New data from a mid-stage study on the use of Ilaris in TRAPS showed that patients who came off therapy after being treated with the drug did not have a relapse for three months on average.

Earlier data from the study showed that 90 percent of patients experienced a significant improvement in symptoms after just one week of treatment with Ilaris. This rose to 95 percent after two weeks. (Reporting by Caroline Copley; Editing by Elaine Hardcastle)

HEALTHCARE

New Study Uses Animal Model Similar To Humans And Shows BPA Can Affect Thyroid Function Main Category: Endocrinology Also Included In: Pregnancy / Obstetrics; Public Health Article Date: 16 Nov 2012

In utero exposure to bisphenol A (BPA) can be associated with decreased thyroid function in newborn sheep, according to a recent study accepted for publication inEndocrinology, a journal of The Endocrine Society. Hypothyroidism is characterized by poor mental and physical performance in human adults and in children can result in cognitive impairment and failure to grow normally. 

BPA, a major molecule used in the plastic industry, has been shown to be an endocrine disruptor that could exert deleterious effects on human health. Most investigations have focused on reproductive functions, but there is evidence that BPA might have negative effects on other endocrine systems including thyroid function. The current study used sheep, a relevant model for human pregnancy and thyroid regulation and ontogeny, and analyzed the internal exposures of the fetuses and their mothers to BPA and determined to what extent those exposures may be associated with thyroid disruption. 

"Our study is the first to show that BPA can alter thyroid function of pregnant animals and their offspring in a long-gestation species with similar regulation of thyroid function as humans," said Catherine Viguié, PhD, of Toxalim, Research Centre in Food Toxicology in Toulouse, France and lead author of the study. "Because of the potential consequences of maternal/fetal thyroid disruption on neural and cognitive development, we think that our study warrants the need for further investigations on the effect of BPA on thyroid function." 

This study was conducted on adult ewes that had multiple pregnancies before being included in the experiment. Some of the pregnant ewes received daily subcutaneous injections of BPA while the remainder were allocated to the control group. Blood samples were taken from jugular blood, amniotic fluid, placenta samples and cord blood to determine levels of BPA, thyroid-stimulating hormone (TSH) and thyroxine. Results showed that maternal and fetal exposure to BPA was associated with disruption of thyroid function of both the pregnant ewes throughout pregnancy and the newborns as characterized by a decrease in circulating thyroxine levels. 

"BPA concentrations in the mother blood in this experiment were fluctuating between injections from 15 to 1 time the highest blood levels reported in pregnant women in the literature," notes Viguié. "As a consequence, although this study clearly indicates that BPA has the potential to alter thyroid function in living pregnant animals and their offspring, it cannot be considered as fully conclusive in terms of risk for human health in the actual conditions of exposure of human populations." 

"In other words, although our study clearly indicates that BPA-induced thyroid disruption is possible, it does not indicate how probable such a disruption is to occur in real conditions," added Viguié. "Thus, the main merit of our work is to encourage others, including epidemiologists, to scrutinize and qualify carefully such a probability." 

India revokes Roche patent in new blow for Big Pharma By Kaustubh Kulkarni and Ben Hirschler | Reuters – Fri 2 Nov, 2012

MUMBAI/LONDON (Reuters) - India dealt a fresh blow to the international pharmaceutical industry on Friday as its patents appeal board revoked a patent granted six years ago on Roche's hepatitis C drug Pegasys.

The Intellectual Property Appellate Board (IPAB) cited a lack of evidence that the drug was any better than existing treatments and its high price as reasons for the decision.

Pegasys was the first medicine to win protection in 2006 under India's new patent regime and the revocation will rekindle tensions between New Delhi and global drugmakers worried by the country's tough stance.

The decision follows another high-profile setback for the industry in March, when India granted the first ever compulsory licence to domestic drugmaker Natco to sell cheap copies of Bayer's cancer drug Nexavar.

Multinational drug manufacturers see India's $12 billion drug market as a huge opportunity, but are wary of what they see as lax protection for intellectual property in a country where generic medicines account for more than 90 percent of sales.

Indian generic companies, which do not need to plough money back into future research, can produce drugs at a fraction of the cost of originator firms like Roche or Bayer.

Sankalp Rehabilitation Trust, an advocacy group for cheaper medicines, had challenged the Pegasys patent with the IPAB, saying the drug was costly and gave the Swiss company a monopoly in the market for the drug.

The market price of Pegasys is 436,000 rupees for 48 weeks of treatment, although it is also available at a discounted price of 314,496 rupees, Sankalp said in a statement.

Pegasys is given in combination with another drug, ribavirin, which costs 47,160 rupees for the same period, Sankalp added.

The appeals board on Friday termed Sankalp's plea "valid."

PAYING FOR INNOVATION

Roche, which can appeal the decision to the Supreme Court, said it was reviewing the IPAB decision and could not comment on it in detail. But it said a solid system of patent protection was essential to ensure research into new treatments.

"Many of the generic drugs today used in India were once patent-protected and are only available to society because companies such as Roche were willing to take a risk by investing in new innovative drugs," a company spokesman said.

He added that Roche was assessing a number of different ways to make drugs accessible to patients in poorer countries, including volume discounts, rebates and price capping.

Campaigners for greater healthcare access contend that the best way to ensure low drug prices is to maximise generic competition by challenging unjustified patents.

Leena Menghaney, a manager in New Delhi for Medecins Sans Frontieres (MSF), said this was particularly important in a disease area like hepatitis C, which is a growing problem in many Asian countries and often hits the most vulnerable in society.

"This case shows that if people choose to use different public health safeguards in Indian law to check abuse of the patent system, then indeed they do work," she said.

Another case involving drug patents is currently in front of India's the Supreme Court, with Novartisbattling against an earlier decision refusing it a patent on cancer drug Glivec.

India is also taking a tougher line on drug pricing more generally, with plans to increase dramatically the number of essential drugs subject to price regulation.

(Reporting by Kaustubh Kulkarni; Editing by Tony Munroe and David Cowell)

Cognitive behavioural therapy (CBT) for anxiety in people with dementia: study protocol for a randomised controlled trial

Background

Many people with dementia experience anxiety, which can lead to decreased independence, relationship difficulties and increased admittance to care homes. Anxiety is often treated with antipsychotic medication, which has limited efficacy and serious side effects. Cognitive behavioural therapy (CBT) is widely used to treat anxiety in a range of populations, yet no RCTs on CBT for anxiety in dementia exist. This study aims to develop a CBT for anxiety in dementia manual and to determine its feasibility in a pilot RCT.

Methods

Phase I involves the development of a CBT for anxiety in dementia manual, through a process of (1) focus groups, (2) comprehensive literature reviews, (3) expert consultation, (4) a consensus conference and (5) field testing. Phase II involves the evaluation of the manual with 50 participants with mild to moderate dementia and anxiety (and their carers) in a pilot, two-armed RCT. Participants will receive either ten sessions of CBT or treatment as usual. Primary outcome measures are anxiety and costs. Secondary outcome measures are participant quality of life, behavioural disturbance, cognition, depression, mood and perceived relationship with the carer, and carer mood and perceived relationship with the person with dementia. Measures will be administered at baseline, 15 weeks and 6 months. Approximately 12 qualitative interviews will be used to gather service-users' perspectives on the intervention.

Discussion

This study aims to determine the feasibility of CBT for people with anxiety and dementia and provide data on the effect size of the intervention in order to conduct a power analysis for a definitive RCT. The manual will be revised according to qualitative and quantitative findings. Its publication will enable its availability throughout the NHS and beyond.

Off-label Drug Use

What is off-label drug use?

In the United States new drugs are tested in clinical trials (research studies) before they are approved for use in the general public. The clinical trials are done to prove that the drug:

• Works to treat a certain medical condition
• Works the way it is expected to
• Is safe when used as directed

When the US Food and Drug Administration (FDA) is satisfied that the drug works and is safe, it and the maker of the drug create the drug label. This is not an actual label that sticks onto a bottle, but a report of very specific information about the drug. The FDA must approve this report, which is made available to all health professionals who prescribe or sell the drug.

The drug label gives information about the drug, including the approved doses and how it's to be given to treat the medical condition for which it was approved. When a drug is used in a way that is different from that described in the FDA-approved drug label, it is said to be an "off-label" use. This can mean that the drug is:

• Used for a different disease or medical condition
• Given in a different way (such as by a different route)
• Given in a different dose than in the approved label

For example, when a chemotherapy drug is approved for treating one type of cancer, but is used to treat a different cancer, it is off-label use.

Off-label is also called "non-approved" or "unapproved" use of a drug.

Is off-label drug use legal?

The off-label use of FDA-approved drugs is not regulated, but it is legal in the United States and many other countries. An exception to this is the use of some controlled substances, such as opioids (pain medicines like morphine and fentanyl). These drugs cannot legally be prescribed in the United States except for approved purposes.

While it is legal for doctors to use drugs off label, it is not legal for drug companies to market their drugs for off-label uses. This has been a hot news topic recently, as drug companies have been fined because they promoted drugs for uses not approved by the FDA. Off-label marketing is very different from off-label use.

Why are drugs used off-label?

Older, generic medicines are the ones most often used off label. New uses for these drugs may have been found and there is often medical evidence to support the new use. But the makers of the drugs have not put them through the formal, lengthy, and often costly studies required by the FDA to officially approve the drug for new uses.

Off-label drug use is common in cancer treatment. There are many reasons for this:

• Some cancer drugs are found to work against many different kinds of tumors.
• Chemotherapy treatments often use combinations of drugs. These combinations might include one or more drugs not approved for that disease. Also, drug combinations change over time as doctors study different ones to find out which work best.
• Cancer treatment is always changing and improving.
• Oncologists and their patients are often faced with problems that have few approved treatment options.
• Oncologists and their patients may be more willing to try off-label drugs than other medical specialties.

What problems are caused by off-label drug use?

Reimbursement

The biggest problem is getting insurance plans to pay for off-label drug use (reimbursement). Many insurance companies will not pay for an expensive drug that is used in a way that is not listed in the approved drug label. They do this on the grounds that its use is "experimental" or "investigational."

In cancer treatment, these issues have been largely addressed through 1993 federal legislation that requires insurance to cover medically appropriate cancer therapies. This law includes off-label uses if the treatment has been tested in careful research studies and written up in well-respected drug reference books or medical journals. In 2008, Medicare rules were changed to cover more off-label uses of cancer treatment drugs.

Still, the health insurance coverage laws and regulations are complex. If your doctor is thinking about off-label drug use, you and your doctor should carefully check your health plan's coverage. If coverage is denied the first time, it may help for the doctor to send the insurer copies of peer-reviewed journal articles or other respected sources that support the off-label use.

Legal risk

Another problem is that off-label drug use often does not reflect "standard of care" treatment. This leads to possible concern about the legal risk of prescribing off-label should a patient have an unwanted or bad outcome from the treatment.

Lack of regulation and information

The FDA does not regulate the practice of medicine. In general, once the FDA approves a drug, licensed doctors can use it for any purpose they consider medically appropriate. Off-label use can vary greatly from one doctor to another. It depends on the doctor's preferences, knowledge, and past patient experiences.

One of the biggest problems related to widespread off-label use is the lack of information about how to best use the drug beyond what was approved. One of the most reliable and easy-to-find sources of information available to health professionals, caregivers, and patients is the drug label. But the label can only contain the information that has been approved by the FDA, and it does not mention off-label uses.

The medical literature reports clinical trials, including those that are not part of the FDA approval process. This is the main source of off-label use information, although treatment guidelines may also offer options that include off-label use. Treatment guidelines are based on information from medical literature, including clinical trials, and recommend standard ways to treat certain diseases.

Lack of information on off-label drug use and outcomes may also put patients at a higher risk for medication errors, side effects, and unwanted drug reactions. It is important that the patient and doctor talk about the possible risks of using the drug and weigh them against the possible benefits.

How common is off-label drug use?

Little information is available on off-label prescribing in oncology in the US. Yet, a study done in 2008 found that 8 out of 10 cancer doctors surveyed had used drugs off-label. Off-label drug use is well-documented and very common in certain settings, such as pediatrics and HIV/AIDS care.

Studies have reported that about half of the chemotherapy used is given for conditions not listed on the FDA-approved drug label. In fact, the National Cancer Institute (NCI) has stated, "Frequently the standard of care for a particular type or stage of cancer involves the off-label use of one or more drugs." Actual off-label use is likely much higher because chemotherapy is only one aspect of cancer treatment. Studies have yet to look at all the drugs used in cancer treatment, such as anti-nausea drugs and pain medicines.

What other ways are drugs used off label in cancer treatment?

One example of off-label drug use is tricyclic antidepressants to treat certain types of pain. This old class of antidepressant drugs is approved and labeled to be used for clinical depression. Today these drugs are seldom used for depression because safer drugs are now available to treat it. But doctors have found that the tricyclics often work very well in treating certain types of pain.

Another example is lorazepam (Ativan®), an anti-anxiety drug often used as an anti-nausea drug in cancer treatment. In oncology, Ativan is most commonly given under the tongue (the sublingual route), which is also not listed on the drug label. In this case, it is being given for an off-label use and by an off-label route.