eCliniqua, a publication aimed at those who conduct clinical trials, recently published an interesting look at myriad factors driving increased interest by the bio-pharmaceutical industry in researching and developing orphan drugs  http://www.ecliniqua.com/2010/08/16/orphan-drug.html.

Among the factors cited are financial incentives, unmet medical needs, advances in science, and unusual regulatory flexbility.  The piece points out that, while activity in this field is picking up, much remains to be done.  For instance, GSK's Marc Dunoyer, who heads up our rare diseases unit, told eCliniqua that there are 8,000 rare diseases and as few as 10% have approved treatments.  And although the FDA told eCliniqua that nearly 40% of all new drugs now approved by the agency are orphan drugs, reportedly only 359 orphan drugs for 150 rare diseases have been approved in total.

To address this, the National Organization for Rare Disorders, academia, industry and regulators are working to find ways to overcome obstacles that are slowing efforts to help those who suffer from rare diseases.  We're hopeful these efforts will accelerate the availability of more treatments for rare diseases.

A combination of inspiration, hope and heartache accurately describes the last 30 years of my career of treating pediatric infectious disease and working to develop vaccines that help prevent them.  Vaccines have helped us come a long way in the battle against debilitating diseases, such as polio, smallpox, rubella and diphtheria. Still, epidemics of vaccine-preventable diseases erupt, sweeping through areas, spreading illness and claiming lives.

Right now the state of California is dealing with an epidemic of pertussis, or whooping cough.  The number of cases of whooping cough reported so far this year in California is more than five times greater than during the same period last year.  Sadly, seven infants there have died from whooping cough since the beginning of this year.

People may think that this is not a serious disease, but nobody wants it.  Adolescents and adults can experience a severe cough, the complications of which can lead to vomiting and broken ribs.  Babies younger than a year are most vulnerable to complications. For them, the disease can be fatal.  

Vaccines can help prevent whooping cough, but only if people get vaccinated.  Whooping cough is one of the many diseases that children are vaccinated against as part of the routine childhood immunizations.  By ensuring that children get their recommended shots, we can help protect them from whooping cough.

Within five to ten years after completion of the childhood vaccination series, immunity to whooping cough can wear off.   That's why a booster shot is routinely recommended adolescents and adults.  The booster shot is called a Tdap vaccine, and it helps protect adults and adolescents against tetanus, diphtheria and pertussis.  In my opinion, there are two things we can do to make even great gains in the battle against many infectious diseases. First, healthcare providers can make checking vaccinations records a routine for adults, not just children.  Second, adults can ask their healthcare provider about the vaccinations that they need.

When I read about the whooping cough epidemic in California, an all too familiar sense of sadness took hold of me. Yet, I rally, knowing that there is still work to do.  I am inspired by the vision of a day when vaccines are available--and used--to help protect people of all ages from pertussis.

You can learn more about whooping cough at helppreventwhoopingcough.com.

As a major international company with a presence in every part of the globe, we are constantly looking for ways to reduce our carbon footprint while enhancing efficiencies and saving investors' money. 

Information Technology (IT) is one key area of this focus.  Over the past year, with the help of the US Environmental Protection Agency's (EPA) "Energy Star" campaign we have been working to reduce carbon output of our staff's personal computers.  This PC power management initiative was featured recently in a Forrester Research podcast covered here.  A few highlights follow below:

I recently recorded a podcast with GlaxoSmithKline (GSK), the global pharmaceutical company, and their success story of implementing a PC power management initiative that is expected to cut energy costs by ~$1 million per year.

While these savings alone should impress any IT executive - especially IT infrastructure and operations professionals who manage PCs - what I found so unique about their story came through my conversation with Matt Bartow, business analyst in GSK's research and development IT organisation, who led this initiative. In particular, GSK is a great example of how "empowering" staff to innovate can industrialise IT operations leading to significant cost savings and green IT benefits....

To ensure the project was a success, GSK enacted a two week pilot of these new settings on a diverse group of 550 volunteers with different working habits from different countries and lines of business. To refine the power settings, users were surveyed to understand if productivity, application use, or any other working behavior was impacted.

This trial run also helped GSK develop their green IT baseline to assess their financial and environmental savings potential - which turned out to be significant. When implemented across GSK's 92,000 PCs globally, these more aggressive PC power management settings are expected to save close to $1 million in energy costs each year, and a reduction in carbon emissions equivalent to taking 1,012 passenger vehicles off the road.

On August 20, we blogged about our July 28 communications with clinical trial investigators in TIDE, a prospective, cardiovascular outcome study comparing Avandia  (rosiglitazone) to Actos  (pioglitazone) as mandated by the U.S. Food and Drug Administration (FDA).  That July 28 communication summarized the two-day FDA Advisory Committee meeting (July 13-14) on the cardiovascular safety of Avandia. There is an update from FDA regarding the TIDE trial which we felt should be shared for the sake of transparency.

Since the post was published, GSK has received a request from the FDA that the company inform the TIDE Steering Committee to "re-issue a letter"  for trial participants that was sent prior to GSK's July 28, 2010 summary for investigators, Institutional Review Boards and Ethics Commitees  involved in the TIDE trial. 

The FDA wrote GSK that the July 16 letter to trial participants did "not accurately capture the results on all the voting questions posed to the advisory committee panel at this meeting. There is an overemphasis on the mortality findings that appears to dismiss the risk of ischemic cardiovascular events, particularly between rosiglitazone and pioglitazone.  Consequently, this information does not fully inform participants of the potential risks of the different treatments in the TIDE trial."

GSK is complying with this recent FDA request.

Several facts are left unclear in the New York Times article entitled "Glaxo Memo on Avandia Questioned." 

The FDA mandated the TIDE trial, and the Agency asked GSK to write a summary of a two-day Advisory Committee hearing (July 13-14) and the FDA's subsequent notification to GSK that the TIDE trial was being placed on partial clinical hold. The summary was directed to a very specific audience:  investigators (the doctors doing the trials), Institutional Review Boards and Ethics Committees that oversee the ongoing TIDE clinical trial with Avandia.

Specifically, the FDA asked: "Within one week, you must inform all investigators, institutional review boards (IRBs) and ethic committees involved in the TIDE trial of the new safety data/information presented at the FDA Advisory Committee meeting held on July 13 and 14, 2010.  Please also include information regarding the deliberations, recommendations and voting from the panel members regarding the specific questions posed at the meeting.  This information may be utilized by investigators and IRBs to update the informed consent document for patients currently enrolled in the TIDE trial.  In addition, a written summary of this information/data should be submitted to your IND file."

GSK agreed the letter was important for investigators so that they could inform patients in the TIDE trial about the Advisory Committee meeting.  GSK then prepared the letter, which the chair of the TIDE Steering Committee approved. The letter was not a general outreach to physicians.

The summary was to be brief (9.5 pages here) and sent quickly (sent within one week). To provide some context, the transcripts of the meeting total 934 pages. Written briefing materials for the committee had been posted by FDA online and the GSK letter alerted investigators to the documents and provided a link to available materials. Contrary to the NY Times report, the summary mailed by GSK provided full detail on the questions posed to the Advisory Committee and the vote on each.

The summary outlined presentations by FDA agency personnel, GSK and others as well as major discussion points. A brief summary--which is what was required--certainly cannot cover the entirety of the briefings or hearing presentations but both GSK and the chair of the TIDE steering committee agreed that the summary reflected the science and data discussed at the advisory committee meeting. A copy of the letter is available for public review.

As we have in past natural disasters, GSK is working closely with partner humanitarian organizations to assist in the response to the terrible floods in Pakistan.  And that includes our staff who live and work in that country.  The note below was posted on our internal company website:

GSK steps up response for Pakistan flood relief

The situation in Pakistan is changing rapidly as floods have swept down the country and even more rains are expected in the coming weeks.  As the needs are emerging, GSK Pakistan is stepping up its response, working with a number of organizations in all flood-affected areas to provide shelter, clean water, healthcare and food.

On August 18, GSK made a contribution of £100,000 (roughly $150,000) to support agencies which are organizing food and medicines distribution, including funds specifically earmarked for the World Food Programme (WFP), the International Red Crescent (IRC) and medicines to the provincial government agencies reaching some of the remotest areas.  

Despite huge logistical challenges, GSK Pakistan's medicines have been among the first to reach many marooned villages. Our donated medicines have been distributed by a variety of relief aid agencies such as the Abaseen Foundation, the first to reach 20,000 people in the remote Swat valley, and International Medical Corps (IMC) which rapidly mobilized six medical teams to the Khyber Pakhtunkhwa Province in the immediate aftermath of the floods.  With a temporary halt in the rains, people have been able to make their way to the mobile clinics set up by IMC, which have conducted over 6,000 health consultations, treating some of the most pressing cases such as acute diarrhea, skin disease and acute respiratory infections.     

GSK Pakistan has also provided medicines to several other relief agencies reaching areas further south.  For example, the Pakistan Medical Association (PMA), working in Sindh, where widespread destruction has displaced an estimated 3.5 million people from their homes, and also the Pakistan's National Health Emergency Preparedness and Response Centre (HEPR) and The National Commission for Human Development (NCHD), which has already reached over 80,000 patients in mobile medical camps in all the flood affected districts.

In line with GSK's principles for humanitarian support, we are continuing to evaluate the ongoing healthcare needs and will provide further support at the request of the Ministry of Health and local/international NGOs who are involved in the recovery efforts. 

GSK Pakistan employees have been making contributions in cash and in kind. A number of international appeals have been launched.  Employees can make a personal donation via the following website links.

International Federation of Red Cross and Red Crescent Societies Appeal

International Medical Corps has been operating in Pakistan since 1984, providing primary health care services and water/sanitation facilities to displaced Pakistanis as well as to Afghan refugees in the frontier areas.

US-based employees can send contributions to the American Red Cross, Box 37295, Washington, DC 20013, and earmark gifts for the Pakistan Floods Disaster.

Earlier this week we were delighted to announce the launch of a major initiative in Washington, DC with Donovan McNabb and the Washington Redskins.  See below for more, or visit the redskins website.

 August 17, 2010, Washington, D.C. - The Washington Redskins and GlaxoSmithKline (GSK) announced today they are teaming up with Redskins quarterback and health advocate Donovan McNabb to help tackle the nation's childhood obesity epidemic.  As the newly appointed 2010 Redskins Health Ambassador, McNabb will work side by side with Washington, D.C. youth to demonstrate fun and easy ways to make exercise part of their daily lives.  Beating childhood obesity is a top priority on the local and national agenda, gaining momentum through the Redskins' commitment to the Play 60 movement for an active generation and first lady Michelle Obama's "Let's Move!" campaign.  D.C. has one of the highest childhood obesity rates in the nation, which has long-term implications since obesity is linked to chronic diseases like diabetes, heart disease and some forms of cancer.

"We can beat this!  I'm so excited to teach kids and their parents how exercising for 60 minutes a day will keep them healthy and help prevent chronic diseases.  Football has always motivated me to stay active, fit and healthy, and I want to motivate kids to find an activity that will help them do the same," said McNabb.  "Seeing the effect chronic disease has had on my father's life has pushed me to help kids take up healthy habits and provide them with the right tools for a healthier future.  Like I tell my father - together, we can beat this."