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Sep
26
Sandeep Singh Dhillon
Pfizer spends billions to develop new drugs. It’s not satisfied. So it’s launching a startup – Statnews.com
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By DAMIAN GARDE @damiangarde SEPTEMBER 25, 2017

There’s a popular theory about the limitations of global pharma companies: For all their skyscrapers and strategy reviews and private jets, they’re simply too knotted up in bureaucracy to realize how many great drugs are gathering dust in their vaults.

Now, the biggest of Big Pharma is out to do something about that. Pfizer, home to nearly 100,000 employees, on Monday announced the launch of a six-person startup to develop new drugs.

This may seem odd in that Pfizer spends literally billions of dollars a year advancing treatments of its own. But the company’s executives say they simply don’t have the resources to advance all the promising compounds that catch their eye — and they believe an independent company with the scrappy ethos of a startup will be in a better position to take on that task.

“The problem is very simple: There’s too much good science and not enough resources to advance it,” said Dr. Lara Sullivan, a former Pfizer vice president who is now leading the startup.

“If you want to see grown men cry, stop a program for budget reasons, not based on science,” Sullivan said.

The new spinoff, SpringWorks Therapeutics, is getting started with $103 million from investors including Pfizer and Bain. It will focus at first on four Pfizer-invented therapies, for conditions including post-traumatic stress disorder and rare forms of cancer. All are already in clinical trials. The two most advanced therapies, targeting tumors found on connective tissue and nerves, will advance to the final stage of development in the coming year.

SpringWorks, which will be based in New York, also plans to scour the pipelines of other pharma companies for compounds that have been set aside for lack of resources, hoping to license some of them for further testing.

It’s a business idea that has been gaining steam of late.

Roivant Sciences, founded by an ex-hedge fund manager in 2014, has built a cottage industry on the same principle, licensing unwanted therapies from the likes of GlaxoSmithKline and Takeda and then launching small startups to test them.

BridgeBio Pharma, established in 2015, takes a similar approach, searching academia and pharma alike for early-stage projects in the field of inherited disease. “We have what we call a better-owner model,” CEO Neil Kumar said. “We try to advance things as far as possible until we’re clearly not the best owner for the asset.”

The concept of scavenging for waylaid gems is considered so promising that Roivant has raised more than $1 billion to widen its search. Its 32-year-old founder, Vivek Ramaswamy, landed on the cover of Forbes, and two Roivant spinoffs pulled off a pair of biotech’s largest-ever Wall Street debuts.

Neither Roivant nor BridgeBio, however, has yet brought a drug to market.

And they’re dogged by the same questions that will follow SpringWorks: If these discarded compounds are so promising, why were they discarded in the first place? And how can a startup push them along better than a multinational heavyweight?

“What I’d say is that from the ground up we’re different,” said Saqib Islam, SpringWorks’ chief financial officer and chief business officer.

The company doesn’t intend to push for quick-turnaround returns on investment, he said. And it plans to work alongside the companies that originally invented or discovered each compound — such as Pfizer — to take advantage of in-house expertise.

“We think that’s the distinction that will draw some attention from those looking to partner their assets going forward,” Islam said.

Pfizer’s decision to wade into the space follows years of navel-gazing at major pharma companies, which have long envied the agility and nothing-to-lose gusto of biotech startups.

Conscious of how the comforts of corporate largess can be counterproductive, companies including GlaxoSmithKline and AstraZeneca in the past sought to create mini startups within their own walls. But though they tried to replicate the feverish immediacy of startup culture, that proved almost impossible when the employees knew they were operating above the multibillion-dollar safety net of a huge pharma company.

Pfizer’s move to create an independent company — deliberately safety net-free — suggests the biggest wheels of the drug industry have learned an important lesson, said Bernard Munos, a former R&D executive at Eli Lilly who now consults for pharma companies.

“I think the industry has realized that they have not really been true to their words in terms of embracing innovation,” Munos said. “So this is very encouraging, frankly, especially coming from Pfizer.”

Read the original post here https://www.statnews.com/2017/09/25/pfizer-startup-new-drugs/

Sep
20
Sandeep Singh Dhillon
Malaysia moves forward to combat Hepatitis C; issuing compulsory license to produce generic treatment – MIMS Malaysia
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By Reshmin Kaur Cheema

With the rising costs of the Hepatitis C treatment, Malaysian authorities have decided to step up and take measures to ease patients’ burden. They will be following through on plans to allow for generic companies to produce a version of the expensive Sovaldi (Sofosbuvir) pill.

This sets Malaysia as the first country to initiate such move, amidst global headlines highlighting this distressing financial matter. In light of the difficulties governments are facing – especially in Malaysia – patient advocates have commended the move in widening access to treatments.

Putting things into perspective, MIMS previously reported that roughly 500,000 or 2.5% of the Malaysian population are said to be living with chronic liver conditions.

Different approach to tackling increased costs

Executive Director of South Centre, a non-governmental organisation based in Switzerland, Martin Khor wrote to The Star publication stating, “The government decision is the key to opening the door to affordable treatment. The government will have freedom to choose which drug to buy from which firm, at what prices, and with which other drugs to combine it.”

This latest update comes after the government initial plans to issue a supposed government-use license that was announced last month. Following this, Gilead Sciences went on to expand a 2014 licensing deal. This allows for seven larger generic drug makers, with operations in India, to sell copycat forms in middle-income countries like Malaysia, Ukraine, Belarus and Thailand.

The deal was originally thought up to increase access in 101 low-income nations and shut down criticism over the pricey drug status. However, Gilead still endured objections because some of those from middle-income countries cannot afford the Sovaldi drug. Thus, the Malaysian government has decided to roll out generic licensing to bypass the Gilead patent altogether.

Consumer activists say that Malaysia chose to pursue the license as a less restrictive option. They elaborated that Gilead’s ways would jeopardise an ongoing Malaysian project whereby the authorities are working hand in hand with Drugs for Neglected Diseases Initiative – a non-profit and an Egyptian company to produce a generic treatment.

Varying responses – to commend or remain sceptical

Sangeeta Shashikant, a legal advisor to Third World Network (TWN), opined that with this move, Malaysia is projected to obtain combination Hepatitis C treatments for USD300 and perhaps less. The Gilead license, on the other hand, may not reflect the most affordable prices, as pricing relies on the level of generic competition in the country.

Consumers Association of Penang stated that “It is imperative that the government sticks to its decision to issue a government-use license. This is because there are strict limits to what Malaysia can do or cannot import or produce under the Gilead license, and other factors that restrict the freedom to choose the generic companies that it can work with.”

It continued, “…Therefore, it is best for Malaysian consumers and patients that the government have both the government-use license as well as the license scheme that Gilead will extend to Malaysia. Both can co-exist, providing Malaysia with maximum policy choice.”

However, The Galen Centre for Health and Social Policy said Putrajaya should deliberate negotiating the best possible deal with Gilead – including technical support, concessions and additional assistance.

Chief executive Azrul Mohd Khalib said, “This hard-won development would see increased access to a treatment which will improve the quality of life for patients and most importantly, save lives. It represents a moral victory for the government which has worked hard to enhance access for Malaysians to innovative drugs and treatment to treat emerging health challenges.”

However, “implementing a government-use license would be outside that framework. It could represent a pyrrhic approach to a long-term problem with possible consequences and complications,” cautioned Azrul. MIMS

Full article at http://today.mims.com/my-moves-forward-to-combat-hepatitis-c-issuing-compulsory-license-to-produce-generic-treatment?elq_mid=35178&elq_cid=26983

Sep
12
Sandeep Singh Dhillon
Why The Omega-3s In Walnuts Are Not The Same As The Ones In Fish And Algae – Forbes
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By Alice G Walton,

There seem to be an awful lot of articles suggesting that people have a handful of walnuts or seeds to get their omega-3 fatty acids. While this isn’t wholly bad advice, it’s not exactly the most accurate. The problem is it assumes all omega-3s are interchangeable, when in fact they’re not. There are actually several different iterations of the fats, and unfortunately the ones that come from plants are not as healthy as the ones that come from fatty fish (and happily for vegetarians, algae).

Here’s a brief rundown: The variety of omega-3s in plants is α‐linolenic acid (ALA). The omega-3s in fish are eicosapentaenoic acid (EPA) and docosapentaenoic acid (DHA). Humans aren’t able to make EPA or DHA from scratch; we need either to eat them or to form them from shorter fatty acids (like ALA). The body is able to convert ALA into EPA and DHA through a chain of chemical reactions that generally take place in the liver. In this sequence of events, DHA is the final product, arriving a couple of steps after EPA. (For a detailed breakdown of all the reactions needed to take ALA to DHA, see this.)

The problem is that the conversion isn’t very efficient, with only a small percentage of ALA making it all the way to DHA. This is partly due to competition from omega-6 fatty acids, which people tend to eat in higher quantities than omega-3s in general.

“The human body can convert the plant omega-3s into the fish omega-3s but in many people this process does not seem to work very well,” says Philip C. Calder, a professor of Nutritional Immunology at the University of Southampton who studies omega fats. ”One reason for that might be the high amount of omega-6s that people eat – these stop the body’s conversion of plant omega-3s.”

So people who rely on the omega-3s from plants may still be low on DHA and EPA, since the conversion isn’t so efficient. In other words, eating walnuts to get your omega-3s may not be enough. “It is very important for consumers to be aware that some omega-3s come from plants and some come from fish,” says Calder. “These omega-3s are not the same thing.”

The larger issue—and the reason any of this matters at all—is that the different omega-3s have different effects on our health. The long-chain fatty acids, EPA and DHA, are well known to reduce inflammation in the body, and are particularly good for the cardiovascular system, reducing heart disease risk, and for the brain, reducing the risk of dementia. They are also important to the developing brain—pregnant women should take special care to take in enough. The bulk of ALA, which isn’t converted to EPA and DHA, still plays important roles in the body, from the structure of cell membranes to use as energy or energy storage. And omega-6s, though essential in low levels, are linked to inflammation at higher levels. Most people eat many more omega-6s, which are found in a number of foods including poultry, eggs, grains, and nuts, than omega-3s of any type.

So keep in mind the difference in the omega fats, and most importantly, the fact that while DHA, EPA, and ALA all fall under the omega-3 umbrella, they’re not interchangeable. Lumping all omega-3s together may not be wise. Lumping omega-3s and omega-6s together is probably even less wise.

“There is a role in human health for both the plant and fish omega-3s,” says Calder, “but the fish omega-3s are more active in the body. Ideally people will get their omega-3s from foods like fish.” If you don’t eat much fish, you may want to supplement with fish oil supplements. And for those who don’t eat any fish, algae oil supplements will do just fine, since algae contains DHA. In any case, remember that omega-3s from the land are ok, but the ones from the sea are much better.

Opinions expressed by Forbes Contributors are their own.

Sep
4
Sandeep Singh Dhillon
New Data Could Add $1.5 Billion To Sales Of Johnson & Johnson Blood Thinner – Forbes.com
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By Matthew Herper

New data could lead doctors to use Johnson & Johnson and Bayer’s blood thinner Xarelto to prevent heart attacks in some patients, leading to a significant sales boost. In a note to investors earlier this month, Credit Suisse analyst Divan Vamil predicted that a successful result could increase sales of the drug by $1.5 billion in the U.S. alone.

Well, the results are a success, although they come with the same trade-off as any use of a blood thinner: more bleeding. In results published in the New England Journal of Medicine and presented in Barcelona at the annual meeting of the European Society of Cardiology, the combination of a low dose of Xarelto and aspirin cut the risk of heart attacks and strokes, cut the risk of heart attacks by 14%, strokes by 42%, and death by 18%. Forty-one out of every 1,000 people who took the Xarelto-and-aspirin combo would be expected to have a heart attack, stroke, or heart-related death compared to 54 among those who got aspirin alone.

The fact that Xarelto reduced the risk of overall deaths, not just heart attacks or strokes, should convince many doctors to use it. But the study looked only at a very specific group: patients who are at high risk of a heart attack or stroke, but who shouldn’t be getting a different class of clot-preventing drugs that includes the generic clopidogrel, Effient, from Eli Lilly, or Brilinta, from AstraZeneca. Patients who have recently had a heart attack, especially those who were treated by having a heart artery propped open with a metal stent, usually get these medicines for at least several months. But the 27,395-person trial specifically recruited patients who didn’t need these other drugs, and whose disease was stable.

One big opportunity for J&J and Bayer could come from a group that represented only a quarter of the patients in the study: those who’ve come to the hospital because of blood clots in their legs, known as peripheral artery disease. These are caused by artery disease, much like heart attacks and strokes.

Bleeding occurred in three patients out of every hundred in the Xarelto plus aspirin arm of the trial, a 70% increase compared to aspirin alone, although there was no difference in the number of major or fatal bleeding episodes. Peter DiBattiste, J&J’s global head of cardiovascular R&D, says that J&J plans to file with the U.S. Food And Drug Administration for approval for the new use, including the approval of a new, 2.5-milligram dose, by the end of the year.

Jul
28
Sandeep Singh Dhillon
MSD Malaysia launches dual-mechanism drug that significantly lowers LDL-C levels – MIMS Malaysia
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MSD Malaysia has recently announced the approval of ATOZET, a combination of ezetimibe and atorvastatin in Malaysia, on 26 July – at a media launch, held at Ruyi & Lyn, Bangsar, KL. The event was graced by consultant cardiologists, Dr Jeyamalar Rajadurai and Dr David Quek, who shared further insights into the prevalence of hypercholesterolemia, disease complications and advisory on hypercholesterolemia management.

The once-daily combination tablet boasts a dual mechanism of action, treating two main sources of cholesterol in the blood. Ezetimibe inhibits the absorption of cholesterol in the digestive tract, while atorvastatin inhibits the production of cholesterol in the liver. ATOZET is indicated to reduce the risk of cardiovascular events in patients with coronary heart disease (CHD) and a history of acute coronary syndrome (ACS), either previously treated with statin or not.

It is also indicated as an adjunctive therapy to diet for use in adults with primary (heterozygous familial and non-familial) and homozygous familial hypercholesterolemia or mixed hyperlipidaemia in patients not appropriately controlled with a statin alone, or already treated with a statin and ezetimibe.

“Heart disease has been the main cause of death in both men and women. In addition, it is two and a half times more common than all cancers combined for more than a decade. It is a very important cause of premature mortality in both and women below the age of 70. And, one important cause of cardiovascular disease is high cholesterol,” shared Dr Jeyamalar.

High levels of hypercholesterolemia within the Malaysian population

According to the National Health & Morbidity Survey 2015, the overall prevalence of hypercholesterolemia – both known and undiagnosed – among young adults aged 18 years and above in Malaysia was 47.7%. There was a general increasing trend with age: from 22.0% in the 18 – 19 years age group, reaching a peak of 68.8% among the 55 – 59 years age group.

“From above 30 years, there is a huge jump in the prevalence of hypercholesterolemia. In fact, in a survey that was done among 13-year-old students… it was discovered that 23% of these students had high cholesterol levels. We are talking about one in four 13-year-olds with cholesterol levels of more than 5.2mmol,” she explained, depicting the dire situation of heart health in Malaysia.

A REALITY Asia study which examined treatment patterns, goal attainment and factors influencing treatment among patients in six Asian countries who were taking statins, also saw more than half of the participants (particularly in patients at highest risk of various cardiovascular disease, including those with CHD or diabetes) did not accomplish the recommended levels of LDL-C.

Reducing LDL-C levels by 27%

There is evidence suggesting that lower LDL-C levels are associated with fewer CV events: the greater the LDL-C reduction, the greater the CV risk reduction. These benefits related to LDL-C reduction are not specific to statin therapy. Therefore, it seems appropriate to reduce LDL-C as low as possible, at least in patients with very high CV risk,” elaborated Dr David Quek.

ATOZET has been shown in multiple studies to effectively lower LDL-C levels. In a 6-week, multicentre, double-blind, randomised, parallel-group study, ATOZET was shown to provide significantly greater LDL-C reduction (27%) in 196 patients with hypercholesterolemia – compared to doubling the dose of atorvastatin alone.

Additionally, the IMPROVE-IT study which evaluated the clinical benefit of ezetimibe/simvastatin combination compared to the simvastatin alone, has revealed that the combination provided incremental benefits in reducing the many cardiovascular-related risk factors in 18,144 acute coronary syndrome (ACS) patients ̶ a relative risk reduction of 6.4%.

“With the launch of ATOZET, we hope this will enable healthcare professionals to think beyond statin monotherapy when treating patients struggling to achieve their cholesterol level goals. The combination of ezetimibe and atorvastatin in ATOZET represents a new breakthrough, which MSD hopes will aid in addressing the critical unmet needs of patients in Malaysia,” expressed Chris Tan, Managing Director and Zone Leader for Malaysia, Singapore and Brunei at the official launch of ATOZET. MIMS

Jul
24
Sandeep Singh Dhillon
UN: “Scales have tipped” as more HIV-infected individuals get drugs, halving AIDS-related deaths – MIMS Malaysia
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Brenda Lau

More than half of all HIV-infected individuals are on drugs to treat the virus for the first time in the four-decade global AIDS epidemic, the United Nations said in an official report on 20 July. AIDS deaths are also almost halved of what they were in 2005, based on estimates.

Experts worldwide applauded the progress, but questioned if the billions spent in fighting the epidemic for the past two decades should have produced more impressive results.

“When you think about the money that’s been spent on AIDS, it could have been better,” expressed Sophie Harman, a senior lecturer in global health politics at Queen Mary University in London.

More resources seem to have been channelled to strengthening health systems in poor countries, she added.

“The real test will come in five to 10 years once the funding goes down,” Harman said, warning that some countries might not have the funds to sustain the U.N.-funded AIDS programmes on their own.

Worsening the problem, the Trump administration has also proposed a 31% cut in contributions to the U.N. starting this October.

UN report points towards positive outcomes

The report states that approximately 19.5 million HIV-infected individuals were taking AIDS drugs in 2016, compared to 17.1 million in 2015. According to UNAIDS, there were about 36.7 million HIV-infected individuals in 2016, up slightly from 36.1 million the year before.

Michel Sidibe, UNAIDS’ executive director said that more countries are starting treatment as early as possible, proving right the scientific findings of the approach ̶ keeping people healthy and preventing new infections.

Many studies also show that those whose infections are under control, are far less likely to pass it on to an uninfected sex partner.

“Our quest to end AIDS has only just begun,” he wrote.

The report also noted that about three-quarters of HIV-infected pregnant women now have access to medicines that prevent the virus from being transmitted to their babies. Five hard-hit African nations also now provide lifelong drugs to 95% of pregnant and breast-feeding women with the virus.

The death toll from the disease has also dropped dramatically in recent years as the drugs are now more affordable and accessible, making the illness a manageable disease.

AIDS unlikely to be eliminated forever

But Harman said that the report was unrealistic.

“I can see why they do it, because it’s bold and no one would ever disagree with the idea of ending AIDS. But I think we should be pragmatic,” she remarked. “I don’t think we will ever eliminate AIDS; so it’s possible this will give people the wrong idea,” she added.

Elsewhere, the World Health Organisation (WHO) unintentionally supported Harman’s statement, warning that there are rising levels of resistance to HIV drugs. This could undermine promising progress against the AIDS epidemic, if effective action is not taken early.

Six out of 11 countries surveyed in Africa, Asia and Latin America observed more than 10% of HIV patients on antiretroviral drugs, already have a strain resistant to most widely-used medicines.

Upon reaching the 10% threshold, the WHO recommends countries to urgently review their HIV treatment programmes and switch to different drug regimens to limit the spread of resistance.

Hope still remains in cows

All hope is not lost as researchers from the International AIDS Vaccine Initiative (IAVI ) and the Scripps Research Institute have discovered that special types of antibodies produced by cows, can neutralise HIV. This could mean a vaccine in the future.

The cow’s antibodies, which would normally take humans three to five years to develop, were produced by the cow’s immune system in a matter of weeks. It also showed that it could neutralise 20% of HIV strains within 42 days. By 381 days, 96% of strains were neutralised in lab tests.

“Unlike human antibodies, cattle antibodies are more likely to bear unique features and gain an edge over HIV, said Dr Dennis Burton, part of the research team.

Creating a vaccine out of cow antibodies still remains a significant challenge – but the team says that the cattle study could help point the way. MIMS

Jul
20
Sandeep Singh Dhillon
When The Drug You Need Doesn’t Make Money – Forbes
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By Alison Bateman-House, CONTRIBUTOR

What if a drug you need doesn’t earn enough money for the company to continue making it? In thinking about this, let’s start with a hypothetical. Unfortunately, you cannot eat protein or gluten–no matter what form they are in, if you consume either, you become violently ill. So you have learned to eat a gluten- and protein-free diet (for the sake of our hypothetical, let’s not worry about whether this is in fact possible). You’ve tried everything you can find at the grocery store, online or made from scratch, and the food that best keeps you healthy and functioning is a vegan “PowerBar” made by the food company VeganDelite. You eat them at least five times a day, but apparently there aren’t many other fans, because VeganDelite announces that it will stop making its PowerBars. You call to protest this decision, only to have the customer service representative say he’s sorry that the company won’t be making your favorite product any more, but there are other vegan PowerBars on the market and surely you’ll like one of them. You protest, explaining you’ve tried everything else, but VeganDelite’s version is what works best for you. This isn’t a mere matter of preference: your health and well-being are at stake. The man apologizes but says he just isn’t able to help you: it was a corporate decision and made due to insufficient sales in the face of alternative substitutes. So you go online and start a petition, but it doesn’t get much attention or traction. You call your local news channel and your congressional representative, but nobody seems willing to join you in calling for VeganDelite to change its decision and continue manufacturing a product for which there is apparently little demand except for you.

Now let’s drop the hypothetical. The parents of one-year-old Christian Mumm are calling on pharmaceutical company GlaxoSmithKline (GSK) to make available its anti-seizure medication Potiga. However, GSK stopped selling the drug at the end of June. The company reports that “as few as 1,500 patients with epilepsy are treated with this medicine globally, with numbers continuing to decline.” 1,500 patients around the world is akin to VeganDelite making its PowerBars just for you. But while you were unable to stir much sympathy for your cause, Mumm’s hometown held a rally in support of the family’s quest to keep their son on Potiga. An online petition was started, gaining over 2,300 signatures in its first three days. Congressman John Larson has weighed in, stating, “It just is so disturbing to me that there is a solution here and all that it requires is a company to say yes, they will make available this successful drug that helps this child out.”

Potiga isn’t the only anti-seizure medicine on the market: indeed, it is apparently only used by a handful of patients. But Mumm’s doctors have tried around seven other medications and found Potiga best at preventing his seizures while preventing debilitating side effects. So Mumm and his family have good reason to desire the drug to continue being available, but GSK also has good reason to stop manufacturing and selling the product.

Is it appropriate or reasonable to demand a company to continue making a product when it does not want to? Does it matter that in one case the desired item is a drug and in the other a food? Does it matter than in one the person who needs the item is a child and in the other it isn’t?

Comparing these real and hypothetical cases highlights several issues. First, we think drugs are somehow different from other goods, even goods as vital as food. I think this is because we view drugs as inherently tied up to issues of life, death and health, even more so than our hypothetical medically necessary food bar.

Also, we tend to want to help identified victims, particularly so-called “innocent victims,” whose suffering is in no way self-inflicted due to lifestyle or behavioral choices. Our hearts ache for little Christian Mumm, who has gone through so much in his one year of life, and for his family, who must be aghast that the drug that has helped their little boy won’t be available anymore­—not even because it has been found to have additional or worse side effects, but because of a market decision. We know, as background knowledge, that both in our country and globally there are innumerable people who are unable to access the drugs they need. But it is hard to grapple with that vast problem of unnamed people, whereas a story of a Connecticut child is much easier to identify with and to feel morally engaged by.

Third, we tend to think of pharmaceutical giants as faceless, deep-pocketed entities for whom it would be no trouble at all to keep manufacturing a drug. We don’t think about the fact that the manufacturing facility being used to produce Potiga could be used to produce a more utilized drug that lots of patients could benefit from. Instead, we seem to think multinational pharmaceuticals have unlimited resources of people, manufacturing capability, raw ingredients and all the other things needs to produce, for an indefinite period of time, a drug.

Finally, this case points out that in some cases the free market leads to results we find uncomfortable, disturbing or even morally untenable. GSK has no market incentive to produce a drug that it sells to only 1,500 people. When it comes to drug development for rare diseases, where perhaps 1,500 people globally constitutes the entire population of patients with a certain condition, companies have made it worth their while to discover and develop drugs because they are sold at astronomical prices. Because there are no other treatments, insurance companies and governments will purchase those incredible expensive drugs. But in the case of epilepsy, where other treatments beside Potiga are available, GSK would not be able to say, OK, we will continue making the drug but we must raise its price exponentially to be able to justify our ongoing investment.

That the market sometimes seems to fail us ought not be a surprise. After all, campaigners for those diseases that occur primarily in the less wealthy regions of the world have been making this argument for years. If a drug, vaccine or device can be made but those who would purchase it are not capable of paying the prices necessary for the developer to make a profit, what do we do? Do we expect philanthropic agencies to leap into the breach, as the Gates Foundation has done with the quest for a malaria vaccine? Do we say to companies that, as a price of doing business (and, in the case of GSK, quite profitably), they must also make and distribute drugs like Potiga that will likely lose money but help a small number of patients? Do we demand GSK surrender its rights to Potiga to a government or foundation that might be able to make the drug? Do we set aside money to fund such drug production?

These are weighty issues that will not be solved quickly. In the meantime, GSK ought to scour its inventory to see if there is extra Potiga that can be made available to Christian Mumm and any other patients in his situation. The energy being spent to rally and sign petitions ought be diverted to asking stakeholders in this issue–government, business and philanthropic–to resolve this ongoing issue. For the congressman who says the solution is just for GSK “to say yes,” what are you going to do to persuade the company to say yes? Is there political will to force business to manufacture and provide non-profitable but needed drugs? If not, what solution is preferable, and who will be responsible for enforcing it? Not only a one-year-old in Connecticut, but people around the world are waiting impatiently for the answer. Up until now, we’ve kicked the can down the road. When are we going to actually deal with this issue?

Read the full article here https://www.forbes.com/sites/alisonbatemanhouse/2017/07/18/when-the-drug-you-need-doesnt-make-money/#677ada4b5576

Jul
12
Sandeep Singh Dhillon
How Not To Lower Healthcare Costs – Forbes
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By Robert Pearl, M.D.,

Citing recent estimates that nearly one-third of clinical laboratory tests are unnecessary for patients, University of Pennsylvania researchers posed a question with huge cost-savings potential: What would happen if doctors were able to look at the price of these tests before ordering them?

To determine whether price transparency might change the way physicians behave, the researchers spent a year studying how frequently clinicians ordered 60 different lab tests at three Philadelphia hospitals. They randomly assigned half of the tests to a control group with no prices posted and the other half to an intervention group for which the “Medicare allowable fees” (the maximum amount Medicare will reimburse) were displayed in the electronic health record system for all clinicians to see.

The results, published in a recent issue of JAMA Internal Medicine, show that making doctors aware of lab-test pricing has almost no influence over medical decision-making. Surprised? You shouldn’t be.

Healthcare Costs In Context

With national healthcare expenditures topping $3.2 trillion annually, legislators on Capitol Hill are looking to root out the kind of wasteful spending that has flattened American wages and sent health insurance premiums soaring. At the same time, Congress is under pressure from voters not to sacrifice the quality or accessibility of medical care.

What few seem to realize, however, is that financial disincentives in healthcare rarely work and, more often, carry unintended consequences. To understand why, consider a daycare experiment carried out by economists and made famous by Freakanomics.

Several years ago, administrators at a childcare facility were asked to collect a modest late fee from parents who picked up their kids more than 10 minutes after the normal closing time.

The idea behind the experiment was to reduce the after-hours burden on caregivers. The result was quite the opposite.

Prior to implementing the late fee, parents did their best to arrive on time, not wanting to inconvenience the people caring for their kids. But when the daycare centers began assigning a fee, the parental perspective shifted. Lateness was no longer seen as an imposition on the workers but rather as a service that could be bought. Parents viewed it as simply the going rate for completing an urgent project or running an errand after work. And because the consequences had gone from personal to transactional, tardiness at the daycare centers doubled.

For physicians, medicine is much more personal than transactional. Doctors worry about whether their patients can afford healthcare coverage and, as a result, ask themselves whether a test or procedure will help. But in the context of deciding whether to order a test that adds $10 to $15 to a hospital bill, the savings hardly seem worth it.

Besides, residents who fear being grilled by an attending physician for not having the most recent laboratory information handy no doubt see the added expense as a small price to pay. And, of course, clinicians know that patients rarely pay the added cost themselves. These contextual clues are important to consider when thinking about how best to lower costs in American hospitals.

So, What Approaches Would Work?

In my experience with leading doctors, I have observed three approaches that have proven effective in modifying physician behavior.

1. Inspire healthy competition. When it comes to their performance, doctors are highly competitive. They like being perceived as “the best” among their peers–a point of pride ingrained in physicians throughout their training. Therefore, presenting doctors with unblinded performance data has the power to modify their behavior. For example, showing which doctors are “best” at achieving high-quality and cost-efficient outcomes would inspire all physicians to learn from, and compete with, the top performers.

I have seen this competitive spirit in action when comparing lengths of stay among hospitalized patients at different Kaiser Permanente medical centers. By measuring outcomes, we know that some physicians are better at helping their patients improve faster and get home sooner. When confronted with this data, the physicians who lagged their peers most often made the necessary adjustments to improve.

2. Appeal to the heart. Physicians usually know when expenditures add little value and they care about the cost impact on their patients. Therefore, the second opportunity to shift behavior is to reframe the problem in human terms, with a focus on the patient, not the dollars.

Devi Shetty, a successful cardiovascular surgeon in India, communicates the hospital’s P&L to all physicians and hospital staff each day. But he doesn’t quantify the bottom-line impact in terms of rupees; instead, he translates it into the number of free surgeries the facility can perform the next day. By showing everyone from housekeepers to department chiefs how a dollar saved can improve a patient’s life, Dr. Shetty’s entire staff strives to lower costs.

3. Make the right thing the easiest thing to do. According to the Penn study, “91% of resident physicians reported that unnecessary lab testing was due to the habit of entering repeat daily lab test orders on the patient’s first day of admission.” Doctors who place these “routine standing orders” at the outset of a patient’s stay rarely discontinue the lab studies even after a patient is stable.

That’s why, except for certain life-and-death situations, it would make more sense for doctors to order these tests one day at a time. Requiring them to do so would make it easier for doctors to consider whether the test is necessary.

The Power Of Perception

Perception in healthcare–what doctors see, hear and feel–is often as powerful as logic. Usually, changing perception in healthcare requires a change in context, not a change in logic.

For example, emailing doctors a warning about unnecessary laboratory testing or requiring them to attend a lecture on the rising cost of medical care won’t change their actions. Do as the researchers did–simply post lab-test prices–and doctors will see it as little more than administrative interference.

These approaches fail to produce the predicted or desired outcomes in healthcare because they ignore what truly motivates physician behavior.

By revealing how the best clinicians are achieving high-quality and cost-efficient results, more doctors will make the effort to improve. By helping doctors connect the dollars they save with the mission-driven outcomes they want, more physicians will do the right thing. And by making it easier for physicians to reconsider the patient’s clinical status each day, the number of wasteful laboratory tests will decline.

Money can have a major impact on physician perception and behavior. But we should remember that using money as motivational tactic alone will, as often as not, produce unintended and undesirable results.

Dr. Robert Pearl is the author of “Mistreated: Why We Think We’re Getting Good Health Care- And Why We’re Usually Wrong” and a Stanford University professor. Follow him: @RobertPearlMD.

Jul
4
Sandeep Singh Dhillon
Did You Grow Up Thinking You’re Allergic To Penicillin? Guess Again – Forbes
Pharma Extra, Pharma Notables
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By Rita Rubin

For years, whenever a health-care provider asked whether you were allergic to any medications, you might have dutifully noted yes, penicillin, which happens to be the most common drug allergy of all. You might not remember ever having an allergic reaction to penicillin, but that’s what your parents always told you, and they wouldn’t steer you wrong.

Well, a couple of new studies suggest that unless your symptoms were pretty dramatic, you might not be allergic to penicillin after all. Researchers tested children and adults who’d been labelled as allergic to penicillin but whose symptoms were relatively mild and found that most tolerated the drug just fine.

Why should you care whether you or your children are allergic to penicillin?

While penicillin is the oldest antibiotic, it and other members of the penicillin family, such as amoxicillin, are still effective, first-choice treatments for a wide range of bacterial infections. But once people are labelled as allergic to penicillin–previous studies suggest that 5% to 20% of the population has been deemed allergic to the drug– doctors instead prescribe second-choice, broader-spectrum antibiotics, which, in the case of garden-variety bacterial ear infections, is kind of like swatting a fly with a sledgehammer.

Broad-spectrum antibiotics kill beneficial bacteria as well as the bad actors, increasing the risk of side effects such as diarrhoea and the possibility that some microbes will develop antibiotic resistance. On top of that, those broad-spectrum antibiotics cost a lot more than generic penicillin or amoxicillin.

In a study published online Monday by the journal Pediatrics, researchers enlisted the help of parents of children ages 4 to 18 who were seen in the emergency department at Children’s Hospital of Wisconsin. The scientists asked 605 parents who reported that their children were allergic to penicillin if they would complete an allergy questionnaire.

Questions included the age of the child when diagnosed with a penicillin allergy, why the drug was prescribed, symptoms of an allergic reaction, how long after the first dose it occurred and whether a parent, doctor or both diagnosed the allergy. The questionnaire also listed 17 symptoms, ranging from a rash to throat swelling and asked the parents to check the ones that occurred in their child after taking penicillin.

Before administering the questionnaire, the researchers consulted with a pediatric allergist about which symptoms were “high risk,” or likely the result of an allergic reaction, or “low risk,” or likely not due to an allergic reaction. High risk symptoms included facial, lip or throat swelling, while low risk symptoms included a rash, itching or hives.

Of the 597 children whose parents completed the questionnaire, nearly three-fourths had low-risk symptoms. The researchers had enough funding to offer testing for penicillin allergy to the parents of 100 low-risk children whose symptoms had been confirmed by their primary physician. The testing consisted of the standard three-tier process: administering penicillin first by a skin prick (also called scratch) test, then by injection and then orally.

“We are prepared to handle serious reactions if one were to occur,” lead author Dr. David Vyles explained to me.

But it turned out that none of the 100 children tested were found to be allergic. In other words, the questionnaire did a good job of identifying the children who really weren’t allergic to penicillin. But, Vyles said, much bigger studies are needed before the questionnaire alone could be used to identify patients labeled as allergic who could safely take penicillin.

In a related study published in April in the journal Emergency Medicine Australasia, researchers in Sydney performed the three-tier allergy test on 100 adult emergency department patients who reported a penicillin allergy. The Australian scientists did not first screen the patients to determine whether their allergy symptoms were high risk or low risk, but they did exclude those who had previously experienced anaphylaxis, a severe, life-threatening allergic reaction, after taking penicillin. Of the 100 patients tested, the researchers found that 81 weren’t actually allergic to penicillin.

“It would be great to examine whether a combination of risk assessment and oral challenge alone could ‘de-label’ the majority of reported penicillin allergies in children.” emergency department physician Dr. Joseph Marwood, lead author of the Australian study, told me in an email. “If this could be demonstrated as safe, without labor-intensive and uncomfortable skin prick testing, it could quickly become practice in the pediatric ER and help children receive the most appropriate antibiotic for their initial illness and beyond.”

Why would people think they’re allergic to penicillin when they’re not?

Vyles thinks parents and even physicians are sometimes too eager to blame penicillin for symptoms that actually are related to the underlying illness. In his study, the children’s primary care doctors had witnessed the supposed allergic reaction in only 14 of the 100 who were tested. In the majority of cases, the primary care doctors diagnosed a penicillin allergy based on what the parents told them.

Personal experience spurred Vyles, an assistant professor of pediatrics at the Medical College of Wisconsin in Milwaukee, to investigate the question.

When his now 9-year-old son was 2, he developed a rash after taking amoxicillin. Because of the rash, the boy’s pediatrician concluded he was allergic to penicillin.

The next time Vyles’ son had an ear infection, his doctor prescribed a broad-spectrum antibiotic instead of amoxicillin. Vyles says he would have had to pay more than $100 more out-of-pocket for the brand-name broad-spectrum antibiotic than for generic amoxicillin.

So he did what only a physician parent could do: He got amoxicillin instead and “challenged” his son with a dose to see if he had any kind of a reaction. Nada. Same thing happened with his daughter, now three. She developed a rash four days after she began taking amoxicillin, so her pediatrician labeled her as allergic to penicillin drugs. Vyles, by then highly skeptical, gave her a dose of amoxicillin, and she did fine.

Again, do not try this at home, but you might want to see an allergist. Says Vyles, “Anybody who has penicillin allergy and questions it should go through the testing.”

Jun
30
Sandeep Singh Dhillon
FDA list of Drugs without Generic Competition to Prevent Price Gouging – 2017 Business Insider
Pharma News, Pharma Notables
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By Lydia Ramsey

The FDA just took a step that could increase competition and prevent price gouging on drugs that seemingly jump in price overnight.

In May, Food and Drug Administration commissioner Dr. Scott Gottlieb said he wanted to make it more difficult for drugs that are off patent to jack up the price of the medication because they don’t face any competition. The biggest example of this was Daraprim, a decades-old drug that then-Turing CEO Martin Shkreli increased in price from $13.50 to $750 a pill.

To keep that from happening in the future, the FDA on Tuesday published a list of more than 200 drugs that have fallen off patent where the FDA hasn’t received any applications for a generic drug version of that drug.

Before now, it wasn’t too clear which drugs had generic alternatives and which didn’t. That left room for drug companies to buy up those drugs without people noticing and quietly raise their prices. The list could help keep a closer eye on the pool of drugs without generic competition. Ideally, other companies could make generic alternatives to increase competion, lower prices, and keep the drugs from becoming the target of price hikes.

“No patient should be priced out of the medicines they need, we must do our part to help patients get access to the treatments they require,” Gottlieb said in a tweet. “Competition and access are foundational elements to continued pharmaceutical innovation, public health improvement,” he said in a follow-up tweet.

The list includes everything from simple chemical compounds to antibiotics, and glucagon hydrochloride, an emergency diabetes drug that raises the level of blood sugar when it falls too low.

Here is the list

https://www.fda.gov/downloads/Drugs/ResourcesForYou/Consumers/BuyingUsingMedicineSafely/UnderstandingGenericDrugs/UCM564441.pdf