Monday, March 30, 2015

Uncertain Health in an Insecure World – 31

“Boundary Crossing”

Mark Zuckerberg was a psychology major at Harvard. As a social media mogul at the forefront of digital culture change, Zuckerberg recently revealed that one secret to his company’s success has been converting the anonymous barrier-laden internet of history (remember early chat rooms like 1970’s Talkomatic and 1980’s CompuServe CB Simulator?) into the psychologically compelling and engaging personal data-sharing platform that is Facebook.

Sitting among one hundred North American medical school deans and hospital CEO’s, we have again heard of THE PROMISE of scientific innovation to offer personalized, precision medicine for complex chronic diseases. For decades, we have advanced fundamental studies of the genome (genomics) and its products (proteomics) as a way of mapping disease-predisposing pathways – from symptoms to molecule science. Increasingly, there have been attempts to connect these data to clinical (phenotype) information about real people that is captured in electronic medical records (EMR’s) and national healthcare databases – from singular mutation syndromes to variable disease presentations.

Thousands of hospitals and millions of healthcare providers interact with billions of patients around the world to generate zettabytes of data that bounces electronically from bedside tablets to the Cloud to Big Pharma repositories. Such personal health information (PHI) is collected under privacy rules and public policies that encrypt computer access and de-identify data under penalty of law. While well-intended, from the onset this privacy protection environment has constrained scientific contributions of PHI data to healthcare. Of note, EMR documentation demands (often tied to reimbursement) are also strongly linked to a 45% average U.S. physician burnout rate!

THE INNOVATOR Dr. Stephen Friend of Sage Bionetworks spoke to our group about “maximizing the flow of relevant insights for maximizing impact…” as the ideal framework for future crowd-sourced network modeling approaches.

Friend’s innovative approach to studying complex diseases involves three breakout concepts:
·         De-linking the generation of data by biomedical scientists from data analysis
·         Migrating PHI data generation & sharing from “guilds of experts” to patients
         ·         Converting fixed institutional EMR platforms to ubiquitous hand-held technologies

Among the novel tools under development by the non-for-profit (NFP) Sage consortium is Synapse, a portal for accessing transparent reproducible datasets that serves as the “sandbox” for studying different problems using common scientific language. This approach, heavily borrowed from the GitHub used by physicists and engineers, incents & rewards attacking challenges collaboratively through sharing scientific communities and open-access peer review.

In addition to studying complex diseases like cancers, psychoses, Dengue Fever, Alzheimer’s Disease and ALS, the Synapse consortium asks, “Why are healthy people staying healthy?” They have screened genetic sequences from nearly 600,000 highly-penetrated genetic diseases to selectively identify 50 candidate mutations that eventually yielded 15 “hero” gene sequences that appear to be protective, at least until environmental influences are factored into disease resilience.
Sage’s crowd-sourcing research movement is partnering with high technology firms (Apple) that have already created applications for health monitoring to develop windows of self-intervention for diseases such as Parkinson’s.
mPower is one such app that unpacks 100 voice dimensions of subjects saying “Ahhhh” for 5 seconds into their iPhone to predict Parkinson’s disease. This approach is also being used as a method for screening candidates for participant-centered clinical trials (using Stanford IRB e-consent). Other Parkinson’s smartphone apps can measure physical task performance (fingertip tapping) and allow users to report anecdotal “signals” (two glasses of wine improves my symptoms).

THE POINT is that just like Mark Zuckerberg at Facebook, very bright scientists in the private and NFP biomedical research sectors have recognized that the intractable bureaucracy and legal constraints to using EMR-derived PHI data as the phenotypic profile for linking to disease predisposition markers mandate an innovative connector approach. As recently revealed by Hewlett-Packard computer scientists in Palo Alto and as reported in The Economist (“Planet of the Phones”), 80% of humans will have the power of a supercomputer on their hand-held device by 2020.

THE FUTURE of complex chronic disease science and management has much more to do with “How I can (transparently) get the rest of the world to help”, instead of hiding behind the “I can’t (legally) tell you this about your chances of getting sick.

Mark Zuckerberg’s understanding of psychology was critical to Facebook encouraging large user communities to voluntarily contribute personal data for subsequent social mega-trend analytics. Bio-medical research also requires voluntary PHI contributions by hand-held device users to achieve a fundamental understanding of the biology of complex diseases.

An analytics full court press on massive big data repositories is mere chat. The social network medium is the message.

The boundary to complex chronic disease progress at scale is system-imposed health privacy constraints.

We in the Square need to cross over.

Saturday, March 28, 2015

Uncertain Health in an Insecure World – 30

“Going to Ground”

Does a twenty-seven year old man with his life ahead of him purposefully killing himself and 149 others by flying an airliner into the French Alps reflect a global health crisis? I don’t know.

But I reserve the right to ask this question, as I sit for six hours on an airplane directly facing a locked “Flight Crew Only” reinforced cabin door. The tragedy befalling the passengers and crew of Flight 9525 and other recent suicide-by-pilot crash victims remains “simply impossible”, in the words of Lufthansa’s CEO Carsten Spohr.

But clearly it is possible, Carsten.

Like others in the highly regulated airline sector, the German airline that is “extremely picky is choosing its pilots” is left grasping for an explanation as to how their industry standard pilot health & wellness vetting procedures failed.

When it comes to human health, and particularly the mental health of twenty-something males whose brains are still developing, there is no fail-safe button.
First, recall that according to the CDC and WHO, the most common cause of death in this age and gender demographic is suicide and accidental injury or poisoning (i.e. overdose). This risk is twice a great in males compared to females (26% versus 13%).

This fatal airplane crash was no accident. It was not preceded by the panicked reactions of someone losing control of a high-speed moving vehicle. According to the cockpit voice recordings, the co-pilot’s breathing remained calm and he said nothing during an eight minute programmed descent to certain death.

Second, let’s remember that the peak onset of schizophrenia and acute psychosis occurs in the 20-30 year age group, with a preponderance of young men being affected. One in 20 people with schizophrenia takes their own life; the risk is greatest at the disease's onset, often associated with a loss of employment or a relationship. Borderline personality disorder is another mental condition associated with a risk of self-harm, including suicide.

There is an as yet unexplained six month gap in Flight 9525 co-pilot’s training that will be very carefully scrutinized by after-crash investigators. Was this lapse an undetected early dissociative break or deep depression?

Third, we hear daily of the violent deaths of young combatants who have been radicalized to follow fractured ideals borne of religious fanaticism, and of the soldiers sent to fight them.

There have been no claims made by any terrorist group, and the search of the co-pilot’s personal files has not yet revealed any manifesto. But in these dangerous days, one can never be sure. 

So, in many sad ways, this victim-laden jetliner crash at the hands, quite literally, of an ill young man is ironically similar to other seemingly victim-less suicides and reckless behavior deaths of young men occurring daily around the world. 

I have posted on the lethal suburban overdose epidemic of illegally-prescribed narcotics. Your concentration has been distracted by YouTube footage of death-by-cop incidents in seemingly lawless urban ghettos.  The world cringes at continuing genocides at the hands of young thugs led by conscience-less war loads.

Just how do these global health crises and crimes against humanity differ from Flight 9525?

The obvious difference is that 149 innocents were acutely victimized by this youthful act of apparent lunacy. Every day in every corner of the globe, young men take up and run with dangerous behaviors, often beginning a slow and desperate dive into deadly chronic diseases like addiction and mental illness. Society recognizes the signs, often too late, and responds with long-term healthcare, imprisonment and/or institutionalization. In many of these instances, the only direct victims of these personal health crashes are the immediate families and friends.

But there are also collateral victims of youth-onset chronic illnesses.

The crime and strife and strain on services that accompanies youthful chronic illnesses have an indirect global health impact and economic cost that we all bear. Whether such youth-onset chronic health declines are genetically predisposed or not, their life-long impact on sufferers and society is massive.

So, while I might well ask myself the question asked by others flying 38,000 feet above the ground in a pressurized metal can, “Are these the people I want to die with?”, I am confident that the cockpit cabin door will hold.  And as I write this, there is a 100 pound flight attendant blocking access to the front cabin while one of the pilots heads into the bathroom.

What could possibly go wrong?

I am not as confident that the tens of thousands of ill young men who die suddenly or who put their long-term health at risk are being clearly seen by healthcare providers and global health advocates as the dangerously vulnerable population that they truly are.

In answer to my original question – YES! – there is a global public health problem that is symbolized by the Flight 9525 co-pilot, a seemingly well twenty-seven year old who proximately caused 150 deaths as a result of an undiagnosed but serious chronic disease that became manifest high in the skies over southern Europe.

We in the Square pray for all the crash victims and their grieving families, and for all of those around the world who are gradually going to ground from deadly youth-onset chronic diseases. 

Sunday, March 22, 2015

Uncertain Health in an Insecure World – 29

“Garbage in – Garbage Out”

Ninety percent (90%) of the world’s data has been generated in the last 2 years.

A physician’s assistant just completed my life insurance physical exam and lab work. In 20 minutes or so, he recorded a medical history, measured vital statistics – height, weight, pulse, blood pressure – then collected my blood and urine for biochemistry and other assays. Just imagine how many times per second this type of medical data and personal health information (PHI) is obtained in doctor’s offices, ambulances & hospitals, and entered into databases around the world.

Global healthcare data is growing exponentially.

In fact, the average hospital generates 665 terabytes of medical and PHI data per year! California-based Kaiser Permanente healthcare system has accumulated 40-50 petabytes of insurance and treatment data from its ~9 million members!! The entire U.S. healthcare system will soon exceed a zettabyte (1021 gigabytes) of data!!!

Significant advances in desktop gene (DNA) sequencing and polymerase chain reaction (PCR) testing procedures provide doctors with rapid diagnoses of infectious microbes, drug resistance and rare diseases. These new technologies are also generating massive amounts of data. A key business strategy of some Big Pharma and global medical lab services conglomerates is to use such companion diagnostics data to bring greater treatment efficiency & effectiveness to the healthcare marketplace – so-called precision or personalized medicine.

This is the promise of “Big Data!

International Business Machines (IBM) has defined three big data characteristics: volume (i.e., hard data storage capacity), velocity (i.e., processing time after queries) and variety (i.e., structured, unstructured and semi-structured log-in files required to access internet programs). McKinsey estimates that up to 80% of U.S. healthcare system information being collected is unstructured – medical device recordings, doctor’s notes, monitor & sensor readouts, lab results, imaging studies, clinical outcomes and financial claims data.

In healthcare, there is a fourth big data ‘V’ called veracity

This blog previously noted the insecurity of PHI data (see post #21). Healthcare data quality assurance is also critical. Like financial data, healthcare data (i.e., prescription handwriting) must be error-free and credible. Poor healthcare data quality in a data warehouse can have life & death consequences, especially when using unstructured data.

Analytics is the key value proposition for healthcare big data.

The scaling up of such unstructured healthcare data requires different analytics architecture than available business intelligence tools. Industrial strength big data computing demands distributed processing capabilities across several servers (or nodes), utilizing parallel “divide & process” open architecture computing that has only recently become available.
Rapidly increasing data requires adequate alternate storage capacity. Check!

Data centers can now store data on large servers (100 terabyte capacity) for later processing using code designed for the relevant application. And while 2015 computer hard disk storage (terabytes and even petabytes), random access memory (RAM >16 megabytes) and reading capacity (>100 megabytes per second) have increased 1000-fold since the 1990’s, it was the advent of open architecture computing that made the promise of big data analytics possible. 

The vision of governments and businesses operating in the healthcare sector is to combine big data, advanced computing science and analytics to solve the complexity of chronic disease management, improve clinical trial accuracy and enable personalized treatments in daily medical practice. The only solution to big data becoming useful is prescriptive & predictive analytics (see post#18).

Before the advent of open architecture computing, data was stored on disks and computation was processor-bound. Conventional relational databases were accessed using structured query language (SQL) supported on one server. Programs written in Java script ran data analysis, wherever the processor was located, without sending the program to a data center. Processing speed declined when large data sets were presented to the server at the same time.

Huge data sets generated by web search engines Google and Yahoo led Doug Cutting (below) to invent and the Apache Software Foundation to release the Hadoop Distributed File System (HDFS, 2003) in order to store big data. MapReduce (MAPR, 2004) software can distribute sub-tasks to 100’s or 1,000’s of servers in a Hadoop cluster (i.e., “shuffling”), map initial outputs, then reduce & track these map outputs in parallel processing jobs.

Open architecture "write once, read many" computing ecosystems like Hadoop improve processing time by using multiple servers to handle a large amount of stored data, reducing the time from query to output. Hadoop achieves high processing speed by using servers working in parallel, equalizing processing power to match the huge amount of data generated.

The healthcare sector has been strangely slow to join this big data revolution. Why?

Patient confidentiality concerns have lagged adoption compared to the retail and banking industries. Big data capture by healthcare systems, governments and Big Pharma has paralleled technical open architecture advances in computing science. Ever-increasing healthcare costs raises serious sustainability concerns that can now be confronted by aggregating insurance risks (“bundling”), managing utilization (“right care”) and tracking patient outcomes in linked databases (i.e. Kaiser Permanente’s HealthConnect).

Working with such big data at scale may eventually allow healthcare stakeholders to create value by exchanging efficacy information and incentivising greater efficiency.

Until this happens, it’s healthcare big data “garbage in – garbage out” littering the Square.  

Sunday, March 15, 2015

Uncertain Health in an Insecure World – 28

“Ebola – So Over It”

Beatrice Yardolo, Liberia’s last active Ebola case was released from hospital on March 5, 2015. Liberia’s deputy health minister, Tolbert Nyenswah, declared “victory” in their fight against Ebola.  This was no doubt a pyrrhic victory for Ms. Yardolo (in yellow) – 3 of her children died from Ebola!

The U.S. Ebola emergency began when Thomas Eric Duncan was diagnosed in Dallas on September 30, 2014 – Mr. Duncan died on October 8, 2014. The subsequent infection of a Dallas hospital healthcare work who contacted Mr. Duncan, and the return of a medical doctor to New York City from Guinea, raised U.S. public health and political concerns to a fever pitch.

In 5 short months, the Ebola crisis has gone from 800 new cases a day to zero! Previously affected countries of Nigeria, Senegal, Mali, Spain, the U.S. and U.K. are currently free of active Ebola virus disease (EVD). Sierra Leone remains an EVD ‘hot zone’, with one patient and ten exposed health workers now returning to the U.S. for treatment at specially equipped hospitals.

According to U.S. CDC and WHO counts, the current death toll from the 2014-15 West African EVD epidemic stands at 10,096, or 41% mortality among those infected. Ill-prepared small economy West African countries saw the greatest mortality, using only isolation & quarantine techniques to contain the outbreaks and “supportive care” to treat the infected.

Until recently, active treatment protocols using ZMapp and anecdotal infusions of survivors’ blood serum were relegated to small numbers of infected foreign aid workers, and were often administered once they had returned for care in their home countries. In late February 2015, San Diego’s Mapp Biopharmaceutical began ZMapp versus supportive care clinical trial in Liberia. 

The fall 2014 scramble by leading anti-viral Pharma companies and government regulatory agencies to generate clinical effectiveness data through West African clinical trials has generated some progress. A Japan Fujifilm antiviral, favipiravir, was administered to 60 Ebola patients treated at MSF centers in Guinea in December, 2014 using historical controls – results are pending. Two public-private partnerships involving the NIH & GlaxoSmithKline and the Canadian government & Merck produced vaccine versus placebo trials in 18,000 Liberian health and emergency workers in February 2015.

Were western drug approval rules promulgated by the U.S. FDA and NIH being brought to bear on Ebola research a version of “unfair trade”? Is it realistic to expect that the FDA’s typical clinical trial requirements for proving drug efficacy & safety involving hundreds to thousands of patients be applied to such remote acute infectious outbreaks?

In the fall of 2014, this academic debate raged among “experts” on the pages of high-impact medical journals such as Lancet and the New England Journal of Medicine.

Epidemiologists understand bio-statistics. As international health experts in tropical diseases, they know that the bloom of acute infectious disease cases is terribly transient. Whether treating those acutely infected or vaccinating those at greatest risk, there may be insufficient big data generated to draw scientifically robust conclusions.

Clinical trialists and Pharma understand randomized placebo-controlled trials. Traditional clinical trials of new drugs for the chronic diseases impacting the global health of millions are safety-first by FDA design. The WHO, Wellcome Trust and MSF medical ethicists have pushed for fast-tracked drug studies without a placebo control study arm.

So who is right?

Senior WHO officials have decried the low number of Ebola cases now available for study, and the related opportunities missed to test potential therapies and vaccines. Chimerix Inc.’s clinical trial of an antiviral drug, brincidofovir, at MSF-run medical centers was recently cancelled due to the lack of new Ebola cases.

The global health panic that was “Fear-Bola” is over.

But there is still a crying need for rapid response times and alternative clinical trial designs for deadly infectious diseases like Ebola with a 41% acute mortality rate.

Absent such bold innovations, for its victims, Ebola is over in three short weeks!

Observing from afar, we in the Square think that WHO is right.

Thursday, March 12, 2015

Uncertain Health in an Insecure World – 27

“Innovation Concentration”

No one country has innovation cornered. But the concentrated creation of innovative companies that is California’s Silicon Valley is undeniable.

The Valley’s famous “fail fast” mantra isn’t an American philosophy as much as it’s a global call-to-action for tech and biotech entrepreneurs. It is peer group permission, Hell encouragement, to fail early in the pursuit of the remote possibility of making the world a faster, better, healthier or safer place.

Just what is it that makes the San Francisco Bay area so special? After spending a week in the midst of truly innovative people and companies (i.e., Hewlett Packard, Parc Xerox, Johnson & Johnson, Google, etc.), I’m in awe of the raw power and belief system underpinning Palo Alto’s commercialization engine.
The factors that make real innovation possible – the secret sauce of big new ideas and the willingness to take financial risks – exist to varying degrees all around the world.

But in the face of such opportunities, there are also stakes and payoffs so high that entire countries make pilgrimages to California to catch a mutated version of the innovation virus, only to return without the potent vaccine that makes success possible at home.

Call them (us) innovation touristas!

Not unlike start-ups that live or die on the cross of commercializing an idea, these foreigners abroad seek instant Googliness. To accelerate national innovation, they may even try market entry via “growth hackers” that paint a veneer on unfinished thoughts – intellectual properties – which remain to be thoroughly de-risked.  But a flashy website featuring a prototype does not impress Silicon Valley stalwarts, who are faced daily with moving human assets and capital away from already successful production lines to innovations, substituting proven success for business risk.

Many countries would like to have this problem.

But few countries, even ones with a strong scientific communities and select commercial successes, can afford this bold Wild West play.

So there’s the rub.

True innovation requires corporate band width and smart venture capital. It requires the capacity to quickly scale-up in the face of sudden demand. And while The Cloud and teraflop computing speeds are ubiquitous in most developed-world countries, there is still something missing.
The missing element is the highly qualified people and teams that exist in such abundance that they can power the innovation grid. INSEAD’s 2014 Innovation Index (II) calls this element the ‘Human Factor’ of innovation.  In the INSEAD 81-point measure of 140 countries, the global top-5 are Switzerland, U.K., Sweden, Finland, the Netherlands, and then the U.S.A. at #6! Regional leaders in their respective geographic zones are India, the U.S.A., Barbados, Singapore, Israel, Switzerland and, yes, Mauritius for sub-Saharan Africa.

So, the point of this isn’t about being exclusionary. It’s about realism.

Every country on Earth can enter the Olympics, but most of the gold, silver & bronze medals go to those with the best talent pool and the most proficient system for promulgating success. The 2012 London Olympics medal count for Mauritius was zero (0). The World Bank’s 2014 measure of Mauritius’ GDP per capita was a middling US$9,202. 
Countries pursuing the brass ring of fostering an innovation ecosystem are aspirational, and that’s all good! But when countries divert national attention and treasure to creating such capabilities without attending to the economic security and wellness of their people, then that’s national vanity bordering on hubris.

At the center of Michael Porter’s five forces for competition is rivalry. Economists measure rivalry by indicators of industry concentration. The concentration ratio indicates the percentage of market share held by the largest firms. When a few firms hold large market shares, the industry is concentrated, and borders on a monopoly. Less concentrated or fragmented markets are tacitly more competitive.

In the innovation industry, competition advantage is concentrated in the Silicon Valley. The global innovation space is imperfect, and the results aren’t always fair.

National dreams of building an innovation economy are not manifest destiny.

In the Square, dreaming big ideas is encouraged. It’s innovation’s reality that bites…  


Saturday, March 7, 2015

Uncertain Health in an Insecure World – 26

“The Global Attention Deficit Economy”

A key life question for most humans is, "What do I attend to?" The answer to this question determines what is acted upon, individually and across society.

Human beings' capacity to pay attention is key to the flow of ideas. But attention cannot be manufactured like an economic commodity.

Two factors contribute to “the global attention deficit economy”… One is medical… One is digital.


Attention deficit hyperactivity disorder (ADHD) is a rapidly growing developed world diagnosis.

According to the U.S. Centers for Disease Control & Prevention (CDC), the number of U.S. children carrying the diagnosis of ADHD in 2011 had increased 50% since 2000, to 11% of those aged 4 to 17 years, or 6.4 million children. In 2013, the U.S. Food & Drug Administration (FDA) accused all major ADHD drug makers of “false and misleading advertising” to parents, teachers and doctors.

Adults with trouble concentration at work or difficulties maintaining relationships are vulnerable, and are being diagnosed with ADHD at unprecedented rates. While childhood ADHD rates have recently plateaued, rapid continued adult ADHD growth is projected through 2020.

Recent revelations about pharmaceutical company marketing of expensive stimulant drugs to a growing population of ADHD adults raise troubling concerns. Direct-to-consumer prescription drug advertising on TV and social media creates confusing messages.

Express Scripts has calculated that adult ADHD diagnoses are up 50% from 2008 to 2012. In 2015, it’s estimated that 44% of all ADHD prescriptions will be filled by adults >18 years old.

The diagnosis of ADHD requires multiple medical assessments and clear-cut testing. But there is ample evidence that doctors in pill mills are pushing powerful drug prescriptions with minimal assessment and limited follow up.

--- // ---

More video content is digitally uploaded to YouTube in one month than all that created by U.S. television networks in the past 65 years.

Each minute, 100 hours of video are uploaded with 1 billion views each month! What the New York Times editor puts on page 1 is de facto more important than what is found on page 7. But what gets the most YouTube views is much less obvious. Search engines like Google do not totally define or determine attention, but can draw attention to new solutions

Dr. Bernardo Huberman, Senior Fellow at Hewlett-Packard (HP) Labs in Palo Alto, California relates big data platforms to what he calls "the attention economy". He articulates an emerging problem for humankind - "tuning in" to what is relevant in the digital world, while ignoring what is not. Dr. Huberman's team is working to understand how to draw attention to what is truly important on your computer monitor or smart phone screen... Clever screen solutions are still years off.

Since 2000, HP has converted the super-computing speed of four large mainframe cabinets to small "compute" components the size of a suitcase. HP projects that in less than ten years, hand-held smart phones will offer super-computing power. Given the rapid increase in global smart phone penetration, that is both amazing and frightening!

The Kaiser Foundation (2010) and Active Healthy Kids Canada (2012) have linked excessive hand-held device use in children & adolescents to ADHD and other developmental brain disorders. There are no such expert guidelines for device use in adults.

It is increasingly clear that ADHD is a conflation of global health and digital world challenges.

So, the next time you cross the Square, turn off your smart phone, and arrive attentive.   

Sunday, March 1, 2015

Uncertain Health in an Insecure World – 25

“Global Unfair Exchange of Drugs”

Sir Nigel Crisp, the former U.K. National Health Service chief executive and Secretary of Health, recently described an “unfair exchange” – the importation of health care workers and exportation of U.S. and Euro-centric ideologies of what is “good” health.
This blog has previously discussed the nearly $1 trillion global pharmaceuticals market (see post #23), and that ~50% of world-wide sales are in the U.S. QUESTION: Does developed world new drug development do people living in less developed countries any “good”?

According to EvaluatePharma’s 2015 report, the top-10 global medical indications for drug sales are Type-2 diabetes, hypertension, rheumatoid arthritis, HIV, Hepatitis C (HCV), multiple sclerosis, asthma, hyperlipidemia, breast cancer and chronic obstructive lung disease (COPD).  Several of these are so-called ‘lifestyle diseases’, which could be prevented by healthy diet habits and smoking cessation. Star Trek’s Mr. Spock, Leonard Nimoy, warned of smoking dangers long before his recent death from COPD at age 83.
Their investor-focused 2015 projection of bio-technologies and small molecules expected to enjoy >$1 billion blockbuster worldwide sales by 2020 includes long-acting insulin, and drugs for LDL cholesterol lowering, asthma, psoriasis, cystic fibrosis, heart failure, pulmonary hypertension, schizophrenia and (of course) many solid and liquid cancers.

Big Pharma’s current blockbusters include drugs for diabetes, lipid-lowering and COPD, as well as expensive new immune-modulators and monoclonal antibodies (Mab’s) for cancers and rheumatoid & sero-negative arthritis. Serious diseases related to altered immunity (autoimmunity) or programmed cell death (apoptosis) may soon be improved by immune-modulators. Small molecule blockers of Protein Cell Death Ligand (PD-L1) binding to T-cell expressed Protein Cell Death-1 (PD-1) receptors are an exploding new drug class. 
Not much else is expected to change regarding the top disease-targeting drugs by 2020, with the exception of a new Biogen Idec drug for multiple sclerosis and a new Sanofi interleukin-antagonist Mab for eczema.

Of note, while the top-10 world-wide chronic diseases are expanding in the un- and under-developed world (see post #10), causing significant morbidity and mortality, the only U.S. FDA-approved and late phase III research new drug that is likely to impact sub-tropical and tropical populations is a Dengue Fever vaccine from Sanofi. 

The WHO estimates that >170 million people world-wide have HCV infection. Treatment with Gilead’s new antiviral, Sovaldi, currently costs US$84,000 in the U.S., US$66,000 in the U.K. and US$57,000 in Germany. In 2014, Gilead tried to deeply discount Sovaldi treatment to US$900 through deals with Egypt's government-run health system (per capita GDP = US$3,243), and with India’s generic drug maker Sciences (per capita GDP = US$1,499).  In 2015, an Indian patent for Sovaldi production was denied, threatening Gilead’s low price plan.  
The India Patent Office had previously rejected Novartis’ 2013 attempt to obtain patent protection for its anti-cancer drug Glivec. Médecins Sans Frontières Executive Director Dr. Manica Balasegaram criticized Gilead for requiring “providers in developing countries to comply with a web of onerous and potentially harmful procedures that aim to preserve Gilead’s ability to charge exorbitant prices in developed countries.

Giving Gilead the benefit of the doubt, we could consider the Sovaldi price break a form of corporate social responsibility (CSR). Cynically speaking, it could also be selective cost-shifting to achieve large population market entry (i.e., health care market ‘cherry picking’). Jansen’s Global Public Health CSR initiative appropriately fosters important research on neglected tropical diseases (NTD) and on multi-drug resistant tuberculosis (MDR-TB) resulting from TB under-treatment in China, Russia, India and sub-Saharan Africa.

In reality, only rich countries can afford these amazing breakthrough drugs, which are sold there at high prices designed to recoup their R&D and M&A costs before patent protection is lost. Poor countries of the world must still get by on older “bread and butter” treatments that they can afford. Differential drug pricing in wealthy versus poor countries creates major public trust issues for Pharma.

Offsetting rising public health care expenses with private sector Pharma profits may be a developed world market-based ‘value proposition’.‘Ethical’ Pharma’s use of in-country generic drug producers may not be in the best interests of local patients, but it does assure long-term commercial success for U.S. and European pharmaceutical interests.

What is clear is that Pharma’s profits are again steadily rising, and are projected to improve through 2020. What is unclear is whether the global “unfair exchange” of drugs is helping or harming the health of those becoming ill in poor countries.

While U.S. and Euro-centric ideologies often permeate the Square, we must reject the "good" not done when these views patently ignore universal rights to good health.