Molecular Insight Pharmaceuticals, Inc. (NASDAQ: MIPI) announced that the European Medicines Agency (EMEA) has approved its Phase 3 protocol for Onalta (Yttrium-90 edotreotide). Onalta is the Company’s lead radiotherapeutic product candidate under development for the treatment of metastatic carcinoid and pancreatic neuroendocrine tumors in patients whose symptoms are not controlled by conventional therapy. The compound has shown the potential to selectively deliver lethal radiation to cancer cells. The proposed Phase 3 trial will confirm that administration of Onalta results in stabilization, regression or complete remission of the carcinoid tumor, and improves carcinoid-related symptoms when compared to a high-dose regimen of the current standard therapy for this disease, Sandostatin®. With EMEA’s approval of its proposed Phase 3 protocol in hand, Molecular Insight can proceed with the final clinical trial that will position Onalta for marketing authorization in the EU.

EMEA notified Molecular Insight that its Onalta Phase 3 protocol is acceptable, including the plan to assess and manage renal tolerance, that the endpoints and measures are appropriate, and that the number of patients proposed to be presented in support of safety for the future Marketing Authorization Application (MAA) is acceptable.

John W. Babich, Ph.D., Executive Vice President, Chief Scientific Officer and President of Research and Development of Molecular Insight, said that, “This is a significant Company milestone that allows us to proceed with a pivotal trial for Onalta in Europe. A successful trial would mean an innovative and well-understood therapy could be made available to thousands of patients whose lives are being cut short and who suffer from the debilitating symptoms that accompany this disease.

“The notification by EMEA comes on the heels of our Special Protocol Assessment (SPA) agreement with FDA allowing us to proceed to a pivotal phase 2 trial for Azedra™ Ultratrace™ (Iobenguane I-131) in another neuroendocrine cancer, pheochromocytoma. Azedra is also currently being evaluated for treatment of neuroblastoma. These drug candidates represent significant opportunities to provide neuroendocrine cancer patients with innovative therapies. Molecular Insight has selected Progression Free Survival (PFS) as the primary endpoint for the proposed Phase 3 study and overall survival will be assessed as a key secondary endpoint. The regulatory progress in Europe will hopefully be followed by regulatory progress in the USA.”

According to Dr. Val Lewington, Consultant Physician, Royal Mardsen NHS Foundation Trust, “EMEA approval of this Phase 3 protocol represents a landmark in radionuclide therapy. The potential of this approach is well-recognized in Europe but this will be the first opportunity to evaluate this peptide-based radiotherapy in a major, multi-center, randomized clinical trial. It is difficult to overstate the importance of this step in the development of innovative therapies for inoperable neuroendocrine tumors which so often prove refractory to other treatment options. News of the EMEA decision will be welcomed enthusiastically both by clinicians and by patients with neuroendocrine disease.”

The proposed Phase 3 protocol will evaluate 194 patients with metastatic, progressive, somatostatin receptor-positive, carcinoid cancer, receiving either Onalta or the standard of care. Carcinoid cancer is a rare, serious and life-threatening condition that affects a group of patients with few treatment options. Once the disease has metastasized, the patients’ prognosis is poor: the best five-year survival rate is reported to be 20 - 30 percent.

Onalta is intended to complement Azedra™, Molecular Insight’s other clinical stage radiotherapeutic candidate for the treatment of neuroendocrine tumors. In 2007, Molecular Insight acquired Onalta from Novartis Pharma AG, which had conducted three Phase 1 and three Phase 2 clinical trials involving more than 300 patients.

About Onalta

Molecular Insight has been developing Onalta as a treatment for metastatic pancreatic neuroendocrine and carcinoid tumors in patients whose symptoms are not controlled by current somatostatin analogue therapy. Neuroendocrine tumors are a type of cancer that arises from neuroendocrine cells and can occur in different parts of the body. A somatostatin analogue is a synthetic compound, in this case a peptide, which functions in the body in a manner similar to the hormone somatostatin, which regulates a variety of other metabolic hormonal functions. Onalta binds selectively to tumor cells that have receptors for the peptide hormone somatostatin on their surface and serves as a carrier for targeted delivery of a lethal dose of radiation to the cancer cells through the radioactive decay of yttrium-90. The compound has been used to treat patients in Europe on an investigative basis for more than 10 years. There are no approved treatments in the U.S. for metastatic neuroendocrine tumors.

Source
Molecular Insight Pharmaceuticals, Inc.

BOSTON, May 18 — Safe extraction of pacemaker and implantable defibrillator leads is not a task for tyros — it requires a minimum of 40 extractions under the supervision of a fully-qualified training physician, according to a consensus statement released by the Heart Rhythm Society.

• Explain to interested patients that this report describes a consensus statement issued by a professional organization, whose members are electrophysiologists.

Moreover, once a physician has been trained in proper technique, he or she should perform a minimum of 20 extractions a year to maintain skills at a safe level, said Bruce Wilkoff, M.D., director of cardiac pacing and tachyarrhythmia devices at the Cleveland Clinic Foundation and a vice president of the Heart Rhythm Society.

The bar defining “fully-qualified training physician” was also set high: he or she must have performed at least 75 extractions as the lead operator and must have a safety and efficacy record at least as good as published norms.

Dr. Wilkoff said that “more than 80% of the panel’s time was spent on discussion of indications for lead extraction.”

The lengthy discussion indicates the difficulty in evaluating risks versus benefits of what is “an invasive procedure,” Dr. Wilkoff said.

The statement made recommendations for four specific indications — infection, chronic pain, thrombosis or venous stenosis, and “leads that are no longer clinically useful.”

According to the statement, both the device and the lead should be removed “in all patients with definite [cardiovascular implantable electronic device] system infection as evidenced by valvular endocarditis, lead endocarditis, or sepsis.”

Likewise, both the device and the leads should be removed in patients with “occult gram positive bacteremia (not contaminant) device pocket infection and in patients “with valvular endorcarditis without definite involvement of the leads and/or device.”

Device and lead removal are not, however, indicated for “a superficial or incisional infection without involvement of the device and/or leads,” nor is removal recommended to treat chronic bacteremia when the source is not the device or leads.

When chronic pain is the complaint, device removal is considered reasonable when pain at the device or lead insertion site is not manageable by medical or surgical techniques.

Device and lead removal is recommended for thrombosis or venous stenosis but the level of evidence is weaker than the evidence in support of removing devices when infection is present.

In most cases, the panel recommended removal for nonfunctioning leads, but said removal is not necessary in patients who have a life expectancy of less than a year.

The panel listed a series of scenarios in which functional leads should be removed, including “patients with life threatening arrhythmias secondary to retained leads,” or when device design or device failure “may pose an immediate threat to the patients if left in place (e.g., Teletronics ACCUFIX J wire fracture with protrusion).”

The panel also recommended removal of functional leads when the leads interfere with cancer treatment.

Dr. Wilkoff has disclosed that he has received grant/research support from Medtronic, St. Jude Medical, Boston Scientific, Spectranetics, and Biotronik. He is a consultant for Medtronic, St. Jude Medical, Boston Scientific, Spectranetics, Cook, and LifeWatch.

Primary source: Heart Rhythm Society

Source reference:
Wilkoff BL, et al “Transvenous lead extraction: heart rhythm society expert consensus on facilities, training, indications, and patient management” HRS 2009; in press (Heart Rhythm, July 2009).

St. Jude Medical, Inc. (NYSE:STJ) announced the results from the Substrate versus Trigger Ablation for Reduction of Atrial Fibrillation (STAR-AF) trial. The findings were presented during a late-breaking clinical trials session at Heart Rhythm 2009, the annual Heart Rhythm Society’s Scientific Sessions in Boston.

The STAR-AF trial, sponsored by St. Jude Medical, was an open, randomized, prospective, multicenter clinical trial involving 108 patients, administered in several Canadian and European centers. Designed as a three arm trial, it compared the generally accepted treatment approach of pulmonary vein isolation (PVI), with complex fractionated electrogram (CFE) guided therapy, and with a third arm that combined the two therapies. The primary endpoint of the trial was relief of atrial fibrillation (AF) episodes of 30 seconds duration or greater, up to 12 months after treatment.

Study results indicate that after one ablation procedure, a combination of PVI and CFE guided therapy demonstrated significantly higher freedom from AF, at 74 percent, as compared to PVI alone at 47 percent and CFE guided therapy alone at 29 percent. Importantly, 94 percent of patients who received the combination of these treatments remained off any anti-arrhythmic medications at the end of the 12-month follow-up period.

Atrial fibrillation is a condition in which the upper chambers of the heart (atria) beat rapidly and erratically, affecting the heart’s ability to adequately pump blood to its lower chambers (ventricles) and subsequently to the rest of the body. The STAR-AF trial studied patients with both symptomatic high burden paroxysmal (intermittent, but frequent and prolonged AF) and those with persistent AF. These two groups represent the largest subset of patients with AF; they experience varying degrees of intermittent AF episodes. The study participants had been unresponsive to medication that is meant to alleviate symptoms or the abnormal heart rhythm itself.

“While many unanswered questions remain about the origins of and best treatment options for AF, the results of STAR-AF indicate that perhaps traditional pathways are not the optimal ones. By mapping for the areas of CFE during AF ablation, we have demonstrated an incremental benefit for patients who historically may have undergone only a PVI,” said Atul Verma, M.D., an electrophysiologist in the Heart Rhythm Program at Southlake Regional Health Centre in Newmarket, Ontario, Canada, who was the principal investigator in the study. “Moreover, I feel that using an automated method for identifying the areas of CFE removes some of the subjectivity that exists in accurately targeting those regions of interest, and may be useful to physicians as they perform AF ablation.”

CFEs are highly random and chaotic electrical signals in the atria that may be a source of origin or perpetuation of AF - they are extremely difficult to identify accurately by visual inspection alone. Therefore, an automatic algorithm from St. Jude Medical’s EnSite™ System was used to identify these regions in the patients randomized to receive this treatment course. The CFE guided therapy alone and hybrid PVI and CFE guided therapy strategies were being studied because often a PVI alone will result in either recurrence of AF or another complicated atrial rhythm that requires additional patient intervention.

“The results of the STAR-AF trial represent an important step toward understanding the best set of tools and therapies for treating cardiac arrhythmias, such as AF, and highlights St. Jude Medical’s commitment to providing pioneering research to improve treatment of this condition,” said Denis Gestin, president of the St. Jude Medical International Division. “Our sponsorship of this trial demonstrates our dedication to partnering with physicians to answer the many questions that exist about the treatment of AF.”

AF is the most common abnormal heart rhythm and affects an estimated 2.3 million people in North America and 4.5 million Europeans. AF is responsible for 15 to 20 percent of all strokes, is a contributor to heart failure and is a leading cause of hospitalizations.

Heart Rhythm 2009 takes place May 13-16 at the Boston Exhibition and Convention Center. The meeting is the most comprehensive educational event on heart rhythm disorders, offering approximately 250 educational opportunities in multiple formats. The world’s most renowned scientists and physicians will present a wide range of heart rhythm topics including cardiac resynchronization therapy, catheter ablation, cardiac pacing and heart failure and the latest technology, including state-of-the-art pacemakers and defibrillators. http://www.HRSonline.org.

Source
St. Jude Medical

Pfizer on Thursday announced a new program that would provide some of its existing customers access to more than 70 types of medications at no cost if they have recently been laid off or lost their prescription drug coverage, USA Today reports. The program — called MAINTAIN, or Medicines Assistance for Those who Are in Need — will begin July 1.

To receive the drugs, individuals must show that they have been unemployed since Jan. 1 and that they no longer have prescription drug insurance. They also must prove that they cannot pay for their medications and that they were taking a medication listed under the program for at least three months prior to losing their jobs. Those who meet the eligibility requirements would receive their medications at no cost for up to one year, or until they have insurance coverage. Pfizer will accept applications through Dec. 31 (Petrecca, USA Today, 5/15).

According to the AP/Detroit News, medications listed for the new patient drug-assistance program include some of Pfizer’s “top money makers,” such as the anti-cholesterol drug Lipitor, the painkiller Celebrex, the fibromyalgia treatment , 5/15).

According to the AP/News, the program “could earn Pfizer some goodwill” after “long being a target of critics of drug industry prices and sales practices” (AP/Detroit News, 5/14). Scott Morgan, president of ad agency Brunner, said, “It goes beyond goodwill. There’s definitely a marketing strategy behind this about defending against generics and maintaining your consumer base. … It’s a pretty savvy move” (USA Today, 5/15).

Reprinted with kind permission from http://www.kaisernetwork.org. You can view the entire Kaiser Daily Health Policy Report, search the archives, or sign up for email delivery at http://www.kaisernetwork.org/dailyreports/healthpolicy. The Kaiser Daily Health Policy Report is published for kaisernetwork.org, a free service of The Henry J. Kaiser Family Foundation.

© 2009 Advisory Board Company and Kaiser Family Foundation. All rights reserved.

View drug information on Viagra.

Washington state lawmakers this week eliminated a budgetary proposal that would have reduced Medicaid payment rates to pharmacies to the lowest levels in the country, the AP/Spokane Spokesman-Review reports. The provision would have reduced the payment rate from 86% to 80% of the average wholesale price of branded drugs. It had been approved by the state House and Gov. Chris Gregoire (D) and was intended to address the state’s $9.3 billion shortfall for the current fiscal year.

The state’s Department of Social and Health Services moved to make the rate change on April 1, but pharmacy operators and a person living with HIV/AIDS brought a suit in federal court in Tacoma, Wash., and a judge blocked the change. According to the judge’s order, the plaintiffs likely could prove that their best interests had not been sufficiently considered and that the proposal would diminish the quality of Medicaid care in the state. Walgreen had stated that if the rate change occurred, 44 of its 111 stores in the state would no longer be able to fill Medicaid prescriptions. Other pharmacies made similar arguments. The state prepared an argument on behalf of the rate cut, but later decided against it.

Along with eliminating the payment reduction, state legislators added a provision that a DSHS analysis is required before rates can be lowered in the next budget, as well as one placing a 2% cap on any future rate cuts. DSHS spokesperson Jim Stevenson said the agency had not started to analyze what would be required to get a rate cut approved, but added, “I think we’re at a stage where we’re going to have to do it soon if we want to get ready for July 1,” the start of fiscal year 2010 (AP/Spokane Spokesman-Review, 5/13).

Reprinted with kind permission from http://www.kaisernetwork.org. You can view the entire Kaiser Daily Health Policy Report, search the archives, or sign up for email delivery at http://www.kaisernetwork.org/dailyreports/healthpolicy. The Kaiser Daily Health Policy Report is published for kaisernetwork.org, a free service of The Henry J. Kaiser Family Foundation.

© 2009 Advisory Board Company and Kaiser Family Foundation. All rights reserved.

A large, well-controlled, multi-national clinical trial program has demonstrated the effectiveness and safety of what may become the first FDA-approved medicine for idiopathic pulmonary fibrosis, or IPF.

In a Phase III clinical study program called “CAPACITY,” investigators discovered that the oral anti-fibrotic and anti-inflammatory agent, pirfenidone, could slow the deterioration of lung capacity in patients suffering from IPF.

The researchers presented their findings on Sunday, May 17, at the American Thoracic Society’s 105th International Conference in San Diego.

The CAPACITY trial consisted of two multi-national, randomized, double-blind, placebo-controlled Phase III trials (CAPACITY 1 and CAPACITY 2) designed to evaluate the safety and efficacy of pirfenidone in IPF patients with mild to moderate impairment in lung function. The primary endpoint of change in percent predicted forced vital capacity (FVC) at week 72 was met with statistical significance in CAPACITY 2 (p=0.001), along with the secondary endpoints of categorical change in FVC and progression-free survival (PFS), defined as time to either death, a 10-percent decrease in FVC or a 15-percent decrease in DLCO (diffusing capacity of the lung for carbon monoxide). The primary endpoint was not met in CAPACITY 1 (p=0.501), but evidence of a pirfenidone treatment effect on the primary endpoint was observed at several periods in that trial. Importantly, greater than 80 percent of patients in the trials completed treatment and greater than 90 percent completed the study.

An exploratory analysis of pooled data from both trials revealed that treatment with pirfenidone resulted in a 30-percent relative reduction in the percentage of patients who experienced an absolute decline in percent predicted FVC of at least 10 percent. This magnitude of decline is considered clinically meaningful, as a 10-percent decline in percent predicted FVC has been shown in multiple studies to be an independent predictor of mortality in patients with IPF. Exploratory analyses of pooled data from the two CAPACITY studies also demonstrated a statistically significant treatment effect on the primary endpoint of change in percent-predicted FVC at week 72, progression-free survival time and change in six-minute walk test distance.

“While it was disappointing that the primary endpoint was not met in CAPACITY 1, important consistencies between the two CAPACITY studies were observed in the overall treatment effect of pirfenidone,” said Paul Noble, M.D., co-chair of the CAPACITY program and professor of medicine and chief of Pulmonary, Allergy and Critical Care Medicine at Duke University Medical Center. “The treatment effect observed in the CAPACITY studies was generally consistent with that observed in the Phase III study in IPF patients conducted by Shionogi in Japan. Collectively, these three studies give us a very good sense of the impact that pirfenidone has on the progression of IPF over at least one year.”

According to the National Heart, Lung, and Blood Institute, about 200,000 Americans have idiopathic pulmonary fibrosis, a condition that scars tissue deep in the lungs. Most patients are diagnosed with the disease in their 50s and 60s, and many people live only three to five years after being diagnosed. There are no approved medications in the United States or Europe to treat the disease. Pirfenidone is approved in Japan for the treatment of IPF.

A total of 779 patients were enrolled in the CAPACITY trials at 110 sites in 11 countries. The mean age of participants was 66. To be eligible for the study, patients had to have a definitive diagnosis made by high-resolution CT scan or by biopsy, and a FVC ? 50 percent of predicted values and a DLCO ? 35 percent of predicted value.

Dr. Roland du Bois, M.D., professor of medicine at National Jewish Health, in Denver, Colo., and CAPACITY co-chair, concurred that these studies were very encouraging for IPF sufferers and added that “the safety and tolerability of pirfenidone was reassuring. The principal side effects experienced by patients in the studies were gastrointestinal discomfort and photo-sensitivity, both of which were manageable in the majority of patients.”

The CAPACITY trials follow a Phase III clinical study conducted in Japan that was reported at the American Thoracic Society’s 2008 International Conference in Toronto. That trial, which demonstrated the ability of pirfenidone to reduce the decline of lung capacity and improve progression-free survival, served as the basis for the Japanese regulatory authorities’ approval of the medicine for the treatment of IPF in Japan.

Dr. du Bois concluded, “When taken in the context of the urgent unmet medical need for new medicines to treat IPF patients, the collective efficacy and safety data from the two CAPACITY studies, corroborated by a similar study in Japan, make a case for the use of pirfenidone in this disease setting.”

InterMune, Inc., the developer of the medication, is preparing a New Drug Application (NDA) for pirfenidone for the treatment of IPF, which it expects to submit to the FDA in the summer of 2009, to be followed by a Marketing Authorization Application (MAA), which will be submitted to the European Medicines Agency (EMEA) around the end of 2009. Meanwhile, all patients in the study have been offered pirfenidone as part of an open-label, long-term safety study called RECAP.

Source:
Keely Savoie

American Thoracic Society

New research suggests that patients newly diagnosed with obstructive sleep apnea (OSA) who use a short-course of the sleep aid, eszopiclone, when beginning continuous positive airway pressure (CPAP) therapy, are more adherent with therapy in six months.

The findings were presented at the American Thoracic Society’s International Conference in San Diego on May 17.

OSA is a common disorder that leads to multiple adverse effects on health and quality of life. CPAP is recommended as the first-line therapy for most patients with OSA, and has been shown to improve sleep quality, reduce daytime sleepiness and enhance quality of life. Despite its many benefits, however, compliance to CPAP is notoriously poor.

“We know that non-benzodiazepine sedative hypnotics promote sleep onset and continuity. Additionally, they can be safely used in patients with OSA, especially those already using CPAP,” said Anita Shah, D.O., author of the study. “To date, the only consistently reliable predictor of long-term use has been compliance with CPAP at treatment initiation. Studies suggest that long-term adherence patterns may be established very early in the course of therapy.”

To test whether eszopiclone would improve early CPAP adherence, the researchers conducted a prospective, double-blind, randomized, placebo-controlled trial involving patients newly diagnosed with OSA who were beginning CPAP therapy. A total of 154 patients were recruited into the study and were randomized to receive either eszopiclone or placebo for their first 14 days of CPAP therapy. CPAP adherence was measured weekly for 24 weeks. The study period began the first day of CPAP therapy.

At the conclusion of the study, researchers found significant differences between the eszopiclone group and the placebo group. On average, patients who received eszopiclone used their CPAP devices more nights per week, and for an hour longer per night. Although the sedative hypnotics were used for only two weeks, the increased adherence with CPAP was sustained for the entire six-month study period.

“Because we know that CPAP therapy improves sleep quality, reduces daytime sleepiness, enhances quality of life and may mitigate the excessive risk for cardiovascular events associated with this disorder, this small intervention could represent a profound clinical benefit to these patients,” said Christopher Lettieri, M.D., principal investigator. “Given the poor adherence to CPAP therapy in many patients, any simple intervention that can reliably improve adherence should be strongly considered.”

This study is part of the CPAP Promotion and Prognosis - The Army Sleep Apnea Program (CPAP ASAP Trial) being conducted at Walter Reed Army Medical Center. The CPAP ASAP Trial will examine multiple outcomes related to therapeutic adherence, health care utilization, co-morbid conditions and quality of life among patients with newly diagnosed OSA.

Source:
Keely Savoie

American Thoracic Society

Pathfinders, a program designed to care for the whole person — body, mind and spirit — has been found to help women with terminal cancer cope and has improved their quality of life, according to a study led by researchers in the Duke Comprehensive Cancer Center.

“The program helped improve distress and despair during the initial three months and up to six months after diagnosis among women with metastatic breast cancer and a six month life expectancy,” said Amy Abernethy, M.D., an oncologist at Duke University Medical Center and lead investigator on the study. “Even though the women were getting sicker and experiencing more symptoms related to their cancer, they reported that they felt less distress and despair as a result of being able to better cope with the cancer.”

Pathfinders focuses on the seven pillars of personal recovery: hope, balance, inner strengths, self care, support, spirit and life review. The program provides patient navigation, counseling, coping skills training, mind and body techniques and lifestyle advice.

“The goal of the program is to teach patients coping skills for dealing with their cancer,” said Tina Staley, director of Pathfinders. “To reach this goal, we have created a common language between patients, nurses, physicians and Pathfinders for communicating coping skills.”

For this pilot study, the researchers enrolled 50 adult breast cancer patients with a prognosis of less than six months survival. The women met with a Pathfinder, a trained social worker, at least monthly, plus telephone conversations and e-mail exchanges. The social workers helped the women identify inner strength, taught them coping skills and encouraged them to engage in complementary and alternative medical services.

The researchers present their findings on a poster at the 2009 American Society of Clinical Oncology meeting in Orlando, on Sunday, May 31.

“There is a growing body of data that shows cancer patients have unmet psychosocial needs, and with programs like Pathfinders we are able to care for the whole person,” Abernethy said. “As a result, we found that this group of women reported a higher quality of life three months after being diagnosed than was expected.”

Additional authors on the study include Tina Staley, James Herndon II, April Coan, Jane Wheeler, Krista Rowe, Barbara Horne and H. Kim Lyerly of Duke.

Source:
Erin Pratt

Duke University Medical Center

Today, 15 new research projects aimed at bringing innovative medicines more quickly to the market have been selected to receive 246 million euros from the European Commission and the European Federation of Pharmaceutical Industries and Associations (EFPIA). The projects will foster understanding of health issues such as asthma and psychiatric disorders while increasing drug safety. They will also help improve the training of researchers and clinicians involved in medicines development. The projects were chosen following the first call for proposals launched within the Innovative Medicines Initiative (IMI), a public-private partnership - so called Joint Technology Initiative- between the European Commission and the pharmaceutical industry. With this selection, IMI has reached a key milestone. This initiative marks the first time that pharmaceutical competitors are pooling their resources, together with research organisations, patient groups and other stakeholders in large consortia, in order to develop generic, pre-competitive knowledge. The Commission’s contribution of €110 million is backed up with €136 million provided in-kind from the pharmaceutical industry. The successful projects will now enter into the final negotiation phase.

“I’m happy to see that this unique public-private partnership that was launched as a new instrument for research two years ago is bearing fruit. In times of crisis, such a model of cooperation is proving particularly well suited to answering both EU public health and economic needs,” said Janez Poto?nik, the EU Commissioner for Science and Research.

Arthur Higgins, CEO of Bayer Healthcare and president of the EFPIA, stated, “I am delighted to see that this pioneering model of collaboration between industry and the Commission has been taken up so positively all across Europe. The IMI will set new standards in data sharing and knowledge exchange.”

Better medicines reaching patients faster

The projects selected will address the main causes of delay, or “bottlenecks”, in the pharmaceutical research and development (R&D) process. The overall objective is to encourage the more rapid discovery and development of better medicines for patients while improving the competitiveness of the European pharmaceutical industry. The projects will help to increase predicted safety and efficacy of medicines, enhance data exchange between researchers and improve education and training in the sector.

The selection process: a substantial interest from stakeholders

Around 150 applications were received. The best consortia, consisting of research organisations, Small and Medium Enterprises (SMEs), academia, patient organisations, and regulatory bodies, were selected in the first peer review to form joint project teams with the corresponding EFPIA consortia. On the basis of stringent scientific criteria and their potential impact on the identified “bottlenecks”, 15 projects from these teams have been selected.

European funding to boost the R&D capabilities of the public sector and SMEs

Pharmaceutical companies within EFPIA will fully fund their own participation by providing R&D resources including staff, laboratory facilities, materials and clinical research. European Community’s funds will be allocated exclusively to other participants (public sector, SMEs, patient groups, academics).

Further steps

Contract negotiations for the 15 projects should be finished by November 2009. A second Call for Proposals is to be launched in autumn 2009. It is planned to seek proposals for projects in oncology, diagnosis of infectious diseases, chronic inflammatory diseases and knowledge management.

Background

Launched in 2007, the Innovative Medicines Initiative was one of the first Joint Technology Initiatives (JTI) to be created. The total IMI budget for the period 2008-2013 is €2 billion (1 billion from the European Community and 1 billion from the industry).

Created in 2007, the Innovative Medicines Initiative Joint Undertaking (IMI JU), representing both the European Community and the industry, implements IMI and is responsible for the launch of Calls for Proposals and the award of grants.

To find out more about IMI: http://imi.europa.eu and http://www.imi-europe.org
To find out more about Joint Technology Initiatives: http://cordis.europa.eu/fp7/jtis/
See the list of the 15 research projects as well as the name of 5 joint technology initiatives in the annex attached.

Annex: Full list of selected projects with their expected outcome

1. Non-genotoxic carcinogenesis

Expected outcome: proven reliable role of early biomarkers in the prediction of cancer development.

2. Expert systems for in silico toxicity prediction

Expected outcome: in silico prediction and expert systems for secondary pharmacology prediction and for pure chemistry-related toxicity.

3. Qualification of translational safety biomarkers

Expected outcome: new specific and sensitive safety biomarkers and their respective assays for human sample for improved predictivity between non-clinical and early clinical studies.

4. Strengthening the monitoring of the benefit/risk of medicines

Expected outcome: new methodologies in pharmacovigilance and pharmacoepidemiology

5. Islet cell research

Expected outcome: better understanding of ?-cell proliferation, differentiation and apoptosis permitting the identification of approaches to preserve ß cell function aiding the development of preventive and curative treatments for diabetes types 1 and 2.

6. Surrogate markers for vascular endpoints

Expected outcome: biomarkers/surrogate endpoints for micro- and macrovascular hard endpoints in diabetes clinical research and new in vitro or in silico tools to test novel therapies.

7. Pain research

Expected outcome: improved understanding of the pathways and mechanisms mediating different kinds of pain, and markers for patient stratification and quantitative pain assessment for efficient testing of new analgesics.

8. New tools for the development of novel therapies in psychiatric disorders

Expected outcome: blood/CSF markers, imaging and/or electrophysiological measures suitable for clinical assessments to be used for preclinical models with sensitive pharmacodynamic markers that are closely linked with psychiatric disorders

9. Neurodegenerative disorders

Expected outcome: translatable animal and human volunteer models for better prediction of clinical efficacy of new therapies in patients with Alzheimer’s disease, Parkinson’s disease and multiple sclerosis.

10. Understanding severe asthma

Expected outcome: a large longitudinal patient cohort enabling validation of novel biomarkers and development of diagnostic criteria for mechanistic and therapeutic trials.

11. COPD patient recorded outcomes

Expected outcome: a framework for better understanding of patients’ experience of chronic obstructive pulmonary disease (COPD) leading to better strategies for measuring clinical trials outcomes

12. European Medicines Research Training Network

Expected outcome: a European biopharmaceutical research training platform providing a sustainable academia-industry cross-disciplinary approach to efficient organisation of training courses on emerging science and technologies across Europe.

13. Safety sciences for medicines training programme

Expected outcome: training programme integrating all safety-relevant disciplines linking animal and human/patient safety data thereby facilitating a more holistic evaluation of new medicines

14. Pharmaceutical medicine training programme

Expected outcome: establish a network of academic centres that delivers postgraduate training programmes in pharmaceutical medicine including quality management of the processes and outcomes.

15. Pharmacovigilance training programme

Expected outcome: customised training programmes for professionals in pharmacovigilance from industry and regulatory agencies to support proactive pharmacovigilance and risk management of medicines.

On Joint Technology Initiatives

JTI’s are a major new element of the EU’s 7th Research Framework Programme. They provide a way of creating new partnerships between publicly and privately-funded organisations involved in research, focussing on areas where research and technological development can contribute to European competitiveness and quality of life.

5 JTI’s have been developed so far: Innovative Medicines Initiative (IMI), Embedded Computing Systems (ARTEMIS), Aeronautics and Air Transport (Clean Sky), Nanoelectronics Technologies 2020 (ENIAC), Hydrogen and Fuel Cells Initiative (FCH).

Source
EFPIA

Some patient advocates are lobbying to legalize “compassionate use” of experimental drugs for people with terminal illnesses who have exhausted other treatments, prompting a debate about how and when to provide such patients with treatments that could prolong their lives, the New York Times reports.

Most insurers do not cover unproven treatments, and physicians have concerns that using experimental treatment could give patients false hope and cause unnecessary pain. Drugmakers are concerned that if there are unfavorable outcomes from providing patients with an unproven treatment, it could hurt the drug’s chances of receiving FDA approval. At the same time, FDA does not want to grant drugs and their makers the opportunity to skip clinical trials. Currently, patients must individually apply to receive treatments through compassionate use (Harmon, New York Times, 5/17).

Reprinted with kind permission from http://www.kaisernetwork.org. You can view the entire Kaiser Daily Health Policy Report, search the archives, or sign up for email delivery at http://www.kaisernetwork.org/dailyreports/healthpolicy. The Kaiser Daily Health Policy Report is published for kaisernetwork.org, a free service of The Henry J. Kaiser Family Foundation.

© 2009 Advisory Board Company and Kaiser Family Foundation. All rights reserved.

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