Angie Drakulich was editorial director of Pharmaceutical Technology.

Angie Drakulich

On Sept. 12, 2012, the Pharmaceutical Research and Manufacturers of America (PhRMA) honored nine individuals for their research into and fight against Alzheimer's Disease (AD) as part of the association’s new Research and Hope Awards. 

By the year 2050, one American will develop AD every 33 seconds, and the US healthcare system will spend an estimated $1 trillion per year on caring for AD patients over age 65. These statistics were highlighted as part of two new PhRMA reports connected to the awards program and its focus on Alzheimer’s: “Researching Alzheimer’s Disease: Setbacks and Stepping Stones” and “Medicines in Development: Alzheimer’s Disease.” 

Delaying the onset of AD by just five years could “reduce the number of people with the disease by nearly half and reduce the cost for care of Alzheimer’s patients by $447 billion a year by 2050,” according to the PhRMA medicines in development report. This is just one of the reasons the association chose to focus on AD in its launch year for the Research and Hope Awards, which replace PhRMA’s Discoverer’s Award. 

It’s no secret that ongoing as well as recent setbacks have challenged research and discovery scientists working to find a treatment for AD. “Years of research has yielded five medicines that provide some symptomatic relief in some cases but has not yet resulted in an approved disease-modifying medicine,” states the PhRMA report on researching AD. “What sets AD apart from other diseases is that scientists still do not know what causes it. We may very well find a treatment first, that leads us back to the cause,” PhRMA President and CEO John J. Castellani told 

 in an interview before the award recipients were unveiled on Sept. 7, 2012.

There are two additional key challenges to developing AD medicines listed in the PhRMA research report: the limited utility of current models of the disease, which creates a barrier in preclinical testing of drug candidates; and the absence of validated noninvasive biomarkers of disease activity and progression, which delay diagnosis until patients become symptomatic. As a result, long and expensive clinical trials have become the mainstay of human studies.

This past summer, Johnson & Johnson, Pfizer, and Eli Lilly all released reports of failed clinical trials targeting the beta-amyloid protein, which is known to form brain plaques in AD patients. Some reports have alluded to lost confidence and momentum in the fight against AD as a result, not to mention potential decreases in R&D investment, but Castellani hopes that these ups and downs are just part of the biopharmaceutical discovery process. “…Researchers take the findings from the unsuccessful projects and use that new information to step forward and continue their quest,” states the PhRMA research report.

Castellani acknowledges that AD is particularly challenging and complex, with 101 unsuccessful drug trials targeting AD since 1999 and a success ratio of 1 in 34 compared with most new medicines, which have a 1 in 5 chance of moving forward. The fact that traditional animal models cannot be used in trials for AD and that human trial recruitment is difficult only add to the complexity of finding a successful AD therapeutic. Castellani has high hopes, however.

He points to the fact that there are nearly 100 new medicines in development right now for AD and dementias, boasting a full pipeline that has potential to reverse, treat, and hopefully prevent this catastrophic disease.  These drugs include a medicine that inhibits the formation and accumulation of amyloid-beta and tau protein deposits; an intranasal medicine that is able to penetrate the blood-brain barrier for mild cognitive impairment, a precursor to AD; and a gene therapy, according to the PhRMA medicines in development report.

Castellani also used the example of the NASA Mars Rover, which recently and successfully landed despite 20 out of 40 previous failed attempts by NASA to orbit or land on the planet (a 1 in 2 ratio), to explain why the industry shouldn’t give up on AD research. 

As part of its awards program and AD focus, PhRMA is highlighting the value of partnerships in disease research. The 2012 recipients, for example, feature not only industry scientists but also academic researchers, patient advocates, and volunteers working to educate the public about the disease. “The enormity of the challenges facing Alzheimer’s disease researchers means that no single company, institute, or organization can tackle the disease alone, particularly when it comes to diagnosing and evaluating the progress of the disease. There are many collaborations in the world of Alzheimer’s research…,” states the PhRMA research report. 

PhRMA is encouraging increased public understanding and awareness of AD and its unique challenges as well. The association has issued a list of 108 ongoing clinical trials for AD patients. Many caregivers of AD patients simply don’t have the energy or time to enroll their loved ones in a trial and the patients themselves often do not have the cognitive ability to enroll themselves, Castellani told 

, but PhRMA hopes this may change as more information about the disease and about the research being done to help treat it reaches the public. 

For a list of the 2012 PhRMA Research and Hope Award recipients, including the Merck BACE team, see the PharmTech

 is a media partner to the 2012 awards, which were given at the Washington, DC, Newseum, an appropriate venue, explained Castellani, because of its focus on providing the public with information.

 interview with Castellani, including his thoughts on why Alzheimer’s Disease was selected for the launch award program over other global priority and neglected diseases and how regulatory approval systems can help AD research, can be download as a podcast at 

Articles

Delaying the onset of Alzheimer's disease by just five years could reduce the cost for care of Alzheimer's patients by $447 billion a year by 2050, according to a new PhRMA report.

Despite Setbacks, PhRMA Awards Encourage Research into and Fight against Alzheimer's

	Be sure to follow our Twitter feed from this year’s PDA-FDA Joint Regulatory Conference, taking place in Baltimore, at Twitter@PharmTechAngie or Twitter@PharmTechGroup. Today’s sessions focused on common regulatory citations (the word is that they haven’t changed much in the past 10 years), new ways to handle internal investigations from Big Pharma organizations such as Amgen and Abbott (including a bit of an audience stirup surrounding the management, or lack thereof, of Class I investigations), FDA encouragement of sending novel excipients in for review, best practices for selecting excipients for new drugs (including how to best deal with suppliers), updates on ICH Q11, and a lot of information on knowledge management, risk-based approaches (including QbD), and lifecycle approaches. More information to come tomorrow when the various FDA offices give their reports.

Be sure to follow our Twitter feed from this year’s PDA-FDA Joint Regulatory Conference, taking place in Baltimore.

LIVE from PDA FDA Joint Regulatory Conference 2012

On Sept. 12, 2012, the Pharmaceutical Research and Manufacturers of America (PhRMA) will honor nine individuals for their research into and fight against Alzheimer’s Disease (AD) as part of the association’s new Research and Hope Awards. The statistics surrounding AD have been plaguing families for decades. The disease is the sixth leading cause of death in the US today, according to the 

 , with 5.4 million people currently affected. By the year 2050, one American will develop AD every 33 seconds. 

The costs surrounding AD, from both a financial and time perspective, are also disheartening. The Alzheimer’s Association notes that the US spends an estimated $200 billion on AD per year, and families across America and around the globe are all too aware of the intense care and attention that AD patients require. 
These are just some of the reasons why PhRMA has focused on AD for its award program, which launches this year, replacing the PhRMA Discover’s award program. 

Alzheimer’s is different from other global priority diseases, Castellani told 

 in a special interview before the awards were announced, because scientists don’t yet know the cause. “The treatment may very well lead us back to the cause in this case,” he said. 

In terms of the day-to-day work being done to find a treatment and improve care, the 2012 PhRMA Research and Hope 

-The Research & Hope Award for Academic Research in Alzheimer's (presented to individuals or a team from the academic community for outstanding research in the search for a cure for Alzheimer's disease):

 Bradley T. Hyman, MD, PhD, Professor of Neurology, Massachusetts General Hospital/Harvard Medical School; and David Holtzman, Andrew B. and Gretchen P. Jones Professor and Chairman; Department of Neurology, Washington University School of Medicine. 

Hyman’s current laboratory is looking to develop methods to examine clinical-pathological correlates and biomarkers in AD, as well as animal and cell models to explore the natural history of the disease.  His team is using advanced microscopy and FRET methods to detect protein-protein interactions and protein conformation

Holtzman, who established the Memory and Cognitive Disorders Clinic at the University of California San Francisco, is involved in clinical and research activities at the Washington University Alzheimer's Disease Research Center. His team’s focus has included ascertaining how apoE influences Abeta metabolism and risk for AD, the influence of synaptic activity and sleep on Abeta metabolism, examining the potential ability of anti-Abeta antibodies to act as therapeutically and diagnostically in AD, and establishing new methods to study protein metabolism in the CNS for both diagnostic and theranostic purposes. 

-The Research & Hope Award for Biopharmaceutical Industry Research in Alzheimer's (presented to individuals or team from a biopharmaceutical company for outstanding research in the search for a cure for Alzheimer's disease):

 The Merck BACE Team, including Eric M. Parker, Senior Director and Neuroscience Site Lead; Andrew W. Stamford, Director, Discovery Chemistry; Matthew E. Kennedy, Associate Director, Neuroscience; Mark S. Forman, Director, Clinical Research; and Julie A. Stone, Senior Scientific Director. 

The BACE team is evaluating the safety and efficacy of the beta-amyloid precursor protein site cleaving enzyme, or BACE, inhibitors, including MK-8931, which is Merck’s lead AD pipeline compound. Clinical trial results thus far evaluating the safety and tolerability of MK-8931 in 40 healthy adults aged 18 to 45 associated single doses of MK-8931 with marked reductions in amyloid beta peptide concentration levels. 
 http://www.merck.com/newsroom/news-release-archive/research-and-development/2012_0713.html

-The Research & Hope Award for Patient Advocacy (presented to an individual or organization that has had significant impact supporting and advocating for legislative or regulatory health policy issues

): Kate Maslow at the Keck Center Institute of Medicine (IOM). Maslow, a scholar-in-residence at the IOM National Academy of Sciences, is focusing on issues tied to the care of people with AD and other dementias. 

Among other related accomplishments, Maslow previously spent 15 years at the Alzheimer's Association, directing practice and policy initiatives to improve the quality, coordination, and healthcare outcomes of long-term services and supports for AD patients and their caregivers. 

-The Research & Hope Award for Volunteer Champion (presented to an individual who has significantly affected the lives of patients and/or home caregivers in a charitable or humanitarian way

): Neha Chauhan at the AFA Teens for Alzheimer’s Awareness. 

Chauhan, now an MBA student at Stanford, has been volunteering in the fight against AD since age 15 as the founder of AFA Teens, which is a branch of the Alzheimer's Foundation of America dedicated to youth advocacy.

Castellani refers to these four award categories as the “ecosystem” required to treat, prevent, and defeat AD once and for all. “Each one of these recipients brings a perspective that is necessary for success when you are trying to battle a disease like Alzheimer’s,” he told 

The science award winners were selected by a special committee of the PhRMA Foundation. The advocacy awards were selected by a committee made up of PhRMA and cohost representatives. 

 is a media partner to the awards program, whose recipients are being honored at the Newseum in Washington, DC, on Sept. 12, 2012.

 interview with Castellani, including his thoughts on why Alzheimer’s Disease was selected for the launch award program over other global priority and neglected diseases, how regulatory approval systems can help AD research, and what the award recipients are working on now, you can download a podcast online at 

. Look for interviews with the winners in the December issue of 

John Castellani, President of PhRMA, talks to PharmTech about its 2012 Research and Hope Award recipients.

Just Announced: PhRMA's Research and Hope Award Recipients

n, with 5.4 million people currently affected. By the year 2050, one American will develop AD every 33 seconds.

		The costs surrounding AD, from both a financial and time perspective, are also disheartening. The Alzheimer’s Association notes that the US spends an estimated $200 billion on AD per year, and families across America and around the globe are all too aware of the intense care and attention that AD patients require. These are just some of the reasons why PhRMA has focused on AD for its award program, which launches this year, replacing the PhRMA Discover’s award program.

		Alzheimer’s is different from other global priority diseases, Castellani told 

 in a special interview before the awards were announced, because scientists don’t yet know the cause. “The treatment may very well lead us back to the cause in this case,” he said.

		In terms of the day-to-day work being done to find a treatment and improve care, the 2012 PhRMA Research and Hope 

The Research & Hope Award for Academic Research in Alzheimer’s (

presented to individuals or a team from the academic community for outstanding research in the search for a cure for Alzheimer’s disease): Bradley T. Hyman, MD, PhD, Professor of Neurology, Massachusetts General Hospital/Harvard Medical School; and David Holtzman, Andrew B. and Gretchen P. Jones Professor and Chairman; Department of Neurology, Washington University School of Medicine.

		Hyman’s current laboratory is looking to develop methods to examine clinical-pathological correlates and biomarkers in AD, as well as animal and cell models to explore the natural history of the disease. His team is using advanced microscopy and FRET methods to detect protein-protein interactions and protein conformation.

		Holtzman, who established the Memory and Cognitive Disorders Clinic at the University of California San Francisco, is involved in clinical and research activities at the Washington University Alzheimer’s Disease Research Center. His team’s focus has included ascertaining how apoE influences Abeta metabolism and risk for AD, the influence of synaptic activity and sleep on Abeta metabolism, examining the potential ability of anti-Abeta antibodies to act as therapeutically and diagnostically in AD, and establishing new methods to study protein metabolism in the CNS for both diagnostic and theranostic purposes.

The Research & Hope Award for Biopharmaceutical Industry Research in Alzheimer’s 

(presented to individuals or team from a biopharmaceutical company for outstanding research in the search for a cure for Alzheimer’s disease): The Merck BACE Team, including Eric M. Parker, Senior Director and Neuroscience Site Lead; Andrew W. Stamford, Director, Discovery Chemistry; Matthew E. Kennedy, Associate Director, Neuroscience; Mark S. Forman, Director, Clinical Research; and Julie A. Stone, Senior Scientific Director.

		The BACE team is evaluating the safety and efficacy of the beta-amyloid precursor protein site cleaving enzyme, or BACE, inhibitors, including MK-8931, which is Merck’s lead AD pipeline compound. Clinical trial results thus far evaluating the safety and tolerability of MK-8931 in 40 healthy adults aged 18 to 45 associated single doses of MK-8931 with marked reductions in amyloid beta peptide concentration levels.
		http://www.merck.com/newsroom/news-release-archive/research-and-development/2012_0713.html

The Research & Hope Award for Patient Advocacy 

(presented to an individual or organization that has had significant impact supporting and advocating for legislative or regulatory health policy issues): Kate Maslow at the Keck Center Institute of Medicine (IOM). Maslow, a scholar-in-residence at the IOM National Academy of Sciences, is focusing on issues tied to the care of people with AD and other dementias.

		Among other related accomplishments, Maslow previously spent 15 years at the Alzheimer’s Association, directing practice and policy initiatives to improve the quality, coordination, and healthcare outcomes of long-term services and supports for AD patients and their caregivers.

The Research & Hope Award for Volunteer Champion

 (presented to an individual who has significantly affected the lives of patients and/or home caregivers in a charitable or humanitarian way): Neha Chauhan at the AFA Teens for Alzheimer’s Awareness.

		Chauhan, now an MBA student at Stanford, has been volunteering in the fight against AD since age 15 as the founder of AFA Teens, which is a branch of the Alzheimer’s Foundation of America dedicated to youth advocacy.

		Castellani refers to these four award categories as the “ecosystem” required to treat, prevent, and defeat AD once and for all. “Each one of these recipients brings a perspective that is necessary for success when you are trying to battle a disease like Alzheimer’s,” he told Pharmaceutical Technology.

		The science award winners were selected by a special committee of the PhRMA Foundation. The advocacy awards were selected by a committee made up of PhRMA and cohost representatives. Pharmaceutical Technology is a media partner to the awards program, whose recipients are being honored at the Newseum in Washington, DC, on Sept. 12, 2012.

 interview with Castellani, including his thoughts on why Alzheimer’s Disease was selected for the launch award program over other global priority and neglected diseases, how regulatory approval systems can help AD research, and what the award recipients are working on now, you can download a podcast online at

On Sept. 12, 2012, the Pharmaceutical Research and Manufacturers of America (PhRMA) will honor nine individuals for their research into and fight against.

PhRMA’s Research and Hope Awards Unveiled

IMS Health released its latest global spending report over the summer, confirming that times for the pharma market are indeed changing. The dreaded “patent cliff” that has likely been haunting the dreams of pharma business leaders for the past few years may still exist, but there is more to look forward to. Estimates from IMS Health seem to demonstrate that the industry has not only been planning ahead to ensure that the plague of patent expiries doesn’t kill business altogether, but has done quite a good job at having a backup plan. Take a look:

		A pipeline rebound is on the way. An expected 160 to 185 new molecular entities (NMEs) are expected to be launched between 2012 and 2016. To compare, 140 NMEs were launched between 2006 and 2010.

		Global spending on medicines is expected to grow 3 to 6%, reaching a total $1.2 trillion in 2016. Spending on generic drugs may reach as much as $430 billion, and on brand drugs as much as $645 billion. The increase is being driven by volume growth in pharmerging markets and higher spending by developed nations, report IMS.

		With any good news of course, comes the chance of not-so-good news.

		In the United States, spending on pharmaceutical drugs is expected to grow 1 to 4% between 2012 and 2016. This growth is considered a recovery but still historically low, says IMS. Part of the reason for the predicted slow growth is the new low-cost generic drugs that will be hitting the market and reducing overall spending. The US Generic Drug User Fee Act (GDUFA) has passed and is being implemented this fall (Oct. 1, 2012 to be exact). GDUFA means good news for consumers and for FDA in terms of obtaining additional user fees to meet its review needs, but it’s no secret that the industry makes less money on generics than on branded products. As a result, the US share of global spending is expected to decline from 41% in 2006 to 31% by 2016.

		That said, IMS predicts “an accelerated shift in spending to generics” as well as to biologics and biosimilar products over the next four years globally. With US Healthcare Reform reaffirmed by the Supreme Court, the pathway for biosimilar products remains open and will be a major focus of biopharmaceutical development efforts in coming years

		Other changes, reports IMS, include increased spending by the pharmerging markets (including the BRIC nations). Such spending is expected to grow 12 to 15% by 2016 and will surpass spending by the EU5 countries (France, Germany, Italy, UK and Spain) for the first time.

		The top therapeutic areas will account for 42% of all global spending on drugs. While the top targets are not expected to change (e.g., drugs targeting cancer, cardiovascular diseases, diabetes, Alzheimer’s and autoimmune diseases, and asthma and other respiratory conditions), IMS does expect that greater availability of existing and new medicines will transform care worldwide. Specifically, while gaps for some treatments will remain (largely in developing nations meaning that the industry still needs to focus on this area) global priority diseases and novel mechanisms of action are likely to provide more options for patients across the board, reports IMS.

		I look forward to watching the pharma landscape grow in new and different ways over the next four years as these regulatory and spending developments shape the industry’s direction.

IMS Health released its latest global spending report over the summer, confirming that times for the pharma market are indeed changing.

IMS Predictions Foretell Change, Positive Change

	Cargo theft, for many years, was a problem that largely plagued the electronics industry, but criminals have recently shifted more attention to the far more lucrative pharmaceutical trade. In March 2010, thieves masterminded a heist of $75 million worth of cancer, psychiatric and blood-thinning drugs from a Connecticut warehouse by cutting a hole in the ceiling and dismantling alarms during a severe storm that masked their activities from the local authorities. To curb future cargo theft, it is imperative that pharmaceutical companies develop solid, globally applicable conveyance security programs.

Cargo theft, for many years, was a problem that largely plagued the electronics industry, but criminals have recently shifted more attention to the far more lucrative pharmaceutical trade.