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The UK’s Medicines and Healthcare Regulatory Authority (MHRA) has awarded Northwest Biotherapeutics, Inc. (Bethesda, MD) the very first “Promising Innovative Medicine”, or PIM designation, for the company’s DCVax-L product.

This month’s ePatient Connections Summit in Philadelphia, PA, saw biopharmaceutical firms, medical device companies, and patient advocates discuss best practices for engaging patients online and in social media settings.

FDA’s Center for Drug Evaluation and Research is establishing an Emerging Technology Team (ETT) to assist innovative manufacturers in navigating the regulatory process and “overcoming roadblocks”.

The European Federation of Pharmaceutical Industries and Associations (EFPIA) and Canada’s Research-Based Pharmaceutical Companies (Rx&D) have welcome finalization of the text for the Comprehensive Economic and Trade Agreement (CETA).

Hemispherx Biopharma (Philadelphia, PA) has announced a set of research collaborations designed to develop “therapeutic cocktails against Ebola which may potentially overcome the emergence of drug resistance due to Ebola virus mutation”.

Value-based Pricing (VBP) was first discussed as far back as 2007 by the UK’s consumer authority, the now defunct Office of Fair Trading (OFT).

Scary tales of The-Next-Big-Social-Thing killing off the first digital marketing blockbuster have been building for years. McKinsey’s November 2013 iConsumer survey reported a 20 percent decline in email usage between 2008 and 2012, as the medium gave up ground to social networks and mobile messaging apps.

The European Medicines Agency (EMA) is reviewing available information on Ebola treatments currently under development to “facilitate evidence-based decision-making” by health authorities.

The European Federation of Pharmaceutical Industries and Associations (EFPIA) reports that it has “strengthened its commitment in China”.

This week members of the Coalition for Healthcare Communication (CHC) met to discuss issues in healthcare communications and marketing like privacy and security, off-label advertising and the Sunshine Act along with an update on the overall DC environment.

The Non-Hodgkin Lymphoma (NHL) treatment market in the Asia-Pacific (APAC) countries of India, Australia, China and Japan will expand from $1.1 billion in 2013 to $1.7 billion by 2020, with Japan remaining the largest contributor to growth, according to GBI Research.

Although biopharmaceutical companies and regulatory authorities have made considerable progress in establishing policies for bringing biosimilars to market, key differences and gaps in requirements for testing and documenting product similarity still characterize the European Union, the U.S. and other regions.

What can be gleaned about the direction that health policy will take for the next five years under the new European Commission, scheduled to take office at the beginning of November? Peter O’ Donnell address this question in his new Applied Clinical Trials column here.

It is estimated that applying big data strategies could potentially generate up to $100 billion in value annually across the US healthcare systems, specifically by optimizing innovation and improving the efficiency of research and clinical trials.

Merck KGaA has announced that it has agreed to buy Sigma-Aldrich for $140 per share in cash, or a total of $17 billion.

After almost two years of discussion and analysis, the Food and Drug Administration is finalizing a proposal for collecting data from manufacturers to help measure the performance of manufacturing operations and the quality of resulting drugs and biologics

Although it is true that there are many legitimate ways to succeed, a host of these strategies appear to be diametrically opposite of one another: Diversified versus pure play, generic versus ethical, regional versus global, R&D versus limited R&D, organic development versus acquisitions, and so on.

A second payment has now been made under the UK’s Pharmaceutical Price Regulation Scheme (PPRS).

Up until a few months ago, there was little industry interest or public support for developing vaccines and treatments to combat the Ebola virus.

Pharmacovigilance controls are becoming firmly embedded in the European Union’s post-authorization regulatory system, with the European Medicines Agency (EMA) acting as the pivot for an EU-wide network of agencies monitoring medicines throughout their market lifespan.

Biopharmaceutical manufacturing is following the general pharmaceutical market trend toward global expansion.

Merck’s Keytruda, recently approved in the US to treat patients with advanced melanoma that have stopped responding to other cancer therapies, will soon face strong competition from Bristol-Myers Squibb’s (BMS) Opdivo, an analyst from research and consulting firm GlobalData has reported.

The European Federation of Pharmaceutical Industries and Associations’ new medicines verification system will put an end to the re-sale of stolen and counterfeit drugs in the European supply chain, the organisation claims.

Early-stage and mid-size bio/pharmaceutical companies have enjoyed an embarrassment of riches over the past 18 months.

The Chinese market is attractive to foreign investors for many reasons. It is the world’s third largest market for pharmaceuticals with annual sale of US$71 billion.

The role of medical affairs is taking on greater importance, as relationships between life sciences companies and external stakeholders become more complicated.

Pharm Exec‘s 13th Industry Audit looks at how well a cross section of companies in big Pharma, mid-tier specialty and biotech are delivering against today’s hottest performance metric: shareholder value.

PEGD0814In this special Technology Issue of Pharm Exec Global Digest: Apple or Big Pharma: Who Will Lead in the Wearable Healthcare Device Space?; Google Glass - a Healthcare User’s Guide; UBISTART’s New Payout for Innovative Start-ups; and more… Click here to read the issue.

With drug-development efforts in Alzheimer’s disease continuing to produce underwhelming results-as clinicians have long battled the inherent diagnosis and translational research challenges associated with the disorder-it’s no secret that there is increased clinical focus now on the prevention and early detection of Alzheimer’s. Those were two areas of emphasis at the recent Alzheimer’s Association International Conference (AAIC) in Denmark. In fact, data presented at the event that reported a decline in incidence of dementia in the U.S. and Europe seems to indicate that reducing the risk of Alzheimer’s and even preventing the disease may be possible. Critical to those pursuits, however, will be future learnings generated from the growing recognition that treatment approaches in Alzheimer’s should target multiple pathologies. Joyce Suhy, Ph.D., VP of neuroscience at BioClinica Inc., a provider of medical imaging solutions for clinical trials, says that people who get dementia usually have mixed pathologies, whether it’s Alzheimer’s, Lewy body dementia, for example, or other similar neurodegenerative conditions. As evidence that researchers are focusing more on multiple pathologies, Suhy cites the recent collaboration struck between the Michael J. Fox Foundation for Parkinson’s Research and the Alzheimer’s Association and the W. Garfield Weston Foundation around studying the links between the Parkinson’s and Alzheimer’s patient populations, with the hopes of reshaping future treatment targets. For Alzheimer’s, specifically, Suhy and her team at BioClinica are focused on helping drugmakers determine whether patients in clinical trials targeting the build-up of amyloid plaque in the brain-considered a crucial factor in the onset of disease-actually have that pathological feature. Most of the notable prevention trials underway are testing drugs that target amyloid. In addition, Novartis recently partnered with Banner Alzheimer’s Institute (BAI) on a clinical study that will determine whether two Novartis experimental anti-amyloid drugs can prevent or delay the emergence of Alzheimer’s symptoms. Advanced molecular imaging techniques can aid in these efforts by ensuring a patient’s pathology is matched appropriately. Further, they provide clinical investigators with sophisticated tools to monitor Alzheimer’s in their studies. Suhy chaired a session on neuroimaging at AAIC, where BioClinica team members presented recent clinical trial progress using amyloid PET screening for enrichment of patient recruitment for early-stage Alzheimer’s. She notes that advances over the past decade in amyloid phenotyping, whether through PET scans or imaging of cerebrospinal fluid (CSF) flow, have helped to better target enrollment in Alzheimer’s trials, particularly as researchers are increasingly studying subjects with more prodromal and milder cognitive impairment. In its presentation, BioClinica reported higher amyloid-negative rates due to the inclusion of earlier-stage patients, when compared to historical trials, which focused more predominately on the mild-to-moderate Alzheimer’s population. “As we’re moving towards earlier screening for enrolling patients into Alzheimer’s disease trials, we find an increasing rate of patients that are amyloid-negative,” says Suhy. “Many of these patients may never advance to mild-to-moderate Alzheimer’s.” Uncovering these earlier clues can be valuable for drug developers when recruiting patients for Alzheimer’s treatment trials targeting amyloid. In past studies, otherwise qualified individuals-who turns out didn’t have amyloid clusters in their brains-were often enrolled, adding significant noise to those trials. The challenge, Suhy says, is that these subjects typically meet the various clinical criteria for the study protocol, such as memory or cognitive decline, and, therefore, are considered good candidates for enrollment. They often progress smoothly through the screening process, and only when getting a PET scan at the end is it discovered they are amyloid-negative. “If a patient doesn’t present with amyloid in the brain, then a drug targeting amyloid will not be effective,” says Suhy. “In other words, the drug target must be present in order for a drug to work. Hence, the current and important initiatives for screening clinical trial patients using molecular imaging like PET or CSF analysis to verify the presence of pathology and enroll appropriate patients for a trial.” Suhy points out that there are several ways to enrich Alzheimer’s patient populations to increase the chances of a successful trial. Besides PET or CSF imaging, some studies are looking at genotype ApoE 2, for example. “By identifying patients earlier in the course of disease, clinicians can maximize their ability to treat and ultimately help patients,” she says. “A key aspect to this is the identification and selection of the right patients.” Key takeaways AAIC, held in Copenhagen, Denmark’s capital city, featured more than 150 data unveilings in Alzheimer’s R&D. One particular notable development, according to Suhy, is the continued promise of tau PET tracers. Tau neurofibrillary tangles, like amyloid, represent one of the hallmark pathologic signs of Alzheimer’s. Though still early in testing, it is thought that tau is more closely linked to memory decline than amyloid, and, therefore, tau imaging-and the development of ligands for early detection of Alzheimer’s risk-may eventually be a better tool for determining cognitive impairment than amyloid imaging. Another takeaway for Suhy was the release of Phase II results for Genentech drug crenezumab, a monoclonal antibody that target all forms of amyloid beta. Though crenezumab did not meet its primary endpoints for cognition and function, a secondary analysis showed promising cognition effects in the milder Alzheimer’s patient cohort when taken at a higher dose. Suhy believes this data further reinforces the importance of early screening of disease risk.

German drugmaker Merck KGaA has begun the second year of its five-year project to provide diabetes management courses for medical and pharmacy students in African Universities.