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Dec
13
Sandeep Singh Dhillon
Chemotherapy-Free Cancer Treatments Move Closer To Reality – Forbes
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By Victoria Forster, CONTRIBUTOR

CAR T-cells first hit the headlines in 2012 after saving the life of a young girl in Philadelphia, Emily Whitehead, who was running out of options to treat her aggressive, relapsed leukemia. Doctors removed some of her T-cells, genetically modified them to attack a protein called CD19 on her leukemia cells and injected them into her bloodstream. Just three weeks later, she was in remission.

Although rare, it is not unheard-of for new treatments to achieve substantial early successes in one or two patients only to experience significant, sobering setbacks in larger-scale trials. This often at the very least dampens the media hype, but CAR T-cells have made consistent progress in garnering both considerable investments from pharmaceutical companies and strong results from clinical trials. Further promising results for the therapy were reported at the American Society of Hematology (ASH) conference this week for a host of blood cancers, including practically incurable multiple myeloma.

Blood cancers are often the experimental template for pioneering new cancer treatments, such as Gleevec for chronic myeloid leukemia, the first-ever FDA approved cancer therapy to target a specific protein found on cancer cells. Gleevec achieved spectacular clinical trial results before its approval in 2001 and went on to transform cancer medicine, changing a disease with a 30% five-year survival rate to one where over 80% of people survive for 10 years or more. This success is partly due to the ease of taking blood samples to monitor the effects of therapies compared to invasive biopsies and costly scans for solid tumor patients, but researchers are optimistic about the wider prospects of CAR T-cells.

“It’s an exciting time. Based on these results and recent FDA approvals in this field, there is reason to be confident that cell therapies, such as CAR T, may one day be the standard of care for hematologic malignancies as well as solid tumors,” said Reiner J. Brentjens, MD, Director of cellular therapeutics at Memorial Sloan Kettering Cancer Centre at the ASH meeting.

For tumors with currently dismal outcomes with conventional chemotherapy, this hopeful prediction can’t come true soon enough, and with almost 100 CAR T-cell clinical trials currently ongoing in the U.S. alone, including for pancreatic and brain cancers, options for patients with hard-to-treat cancers are increasing.

Many conventional chemotherapy agents kill cancer cells in a way which can crudely, but regrettably and truthfully, be described as carpet-bombing, indiscriminately affecting any cell which is dividing. CAR T-cells are the exact opposite, being engineered to target normally one very specific protein on whichever cells are in the crosshairs. In the case of many leukemias and lymphomas, this protein is CD19, present on B-cells, a type of white blood cell normally involved in the immune response when functioning correctly.

More specific therapies generally mean less toxicity for healthy tissues and fewer side-effects for patients. However, just as bacteria generate resistance to antibiotics, cancer cells are constantly evolving to evade the effects of chemotherapy. Many of the old chemotherapy drugs, for their many flaws, work in ways which make it difficult for cancer cells to evolve complete resistance as they affect processes key for survival such as DNA replication. However, the specificity of CAR T-cell therapies is, in this case, an Achilles heel. In one of the trials reported at ASH this week on refractory Non-Hodgkins Lymphoma, in a third of patients who relapsed after treatment, their cancer had evolved to simply not have the CD19 protein targeted by the CAR T-cells, rendering the therapy useless.

One of the current theories as to why this happens is that the CD19 negative cells may have been already present in the patient, just in tiny numbers. When the cells with CD19 were massacred by the CAR T-cell therapy, the cells without it no longer had any competition and thrived. So what is the solution? Make the therapy just a little bit less specific again. New studies are trialing CAR T-cells which go after two targets on B-cells, a protein named CD22 as well as CD19. It is hoped that the B-cells can’t survive if they lose both CD19 and CD22 and even if they evolve to lose one protein, they will still be susceptible to the therapy via the other.

Most CAR T-cell trials have currently been done in patients with few other options but the hope is that they will eventually be able to reduce, or even replace chemotherapy currently needed to achieve a cure, even in cancers with an excellent prognosis. For example, over 85% of children now survive leukemia long-term, but the cocktail of chemotherapies they receive come with considerable side effects, with many survivors experiencing serious health conditions later in life as a result of the treatment.

Although scientists are beginning to understand the short-term toxicities of CAR T-cells, their long-term side effects are largely an unknown and will remain so for several years. However, scientists are hopeful that CAR T-cell therapy will have fewer and less severe long-term side effects than conventional, old chemotherapies. A big ethical and moral question is how to justify replacing treatments which are undeniably very good at achieving the main goal of saving lives, with something which is currently less certain. For parents of children with leukemia who have failed conventional treatments already, the decision to try CAR T-cells is easy. For the first parents that consent to the inevitable chemotherapy-free CAR T-cell only trial, it will be a leap of faith. If all goes well, their children will be the first ever to survive leukemia without any chemotherapy treatment.

In May this year Emily, the first patient to get CAR T-cells celebrated the milestone of being five years cancer-free. Undoubtedly there are still numerous challenges to overcome before CAR T-cells become a mainstream therapy for multiple cancer types, but in five years from now, hundreds if not thousands of others will be joining Emily in this currently unique club.

The original article first appeared at https://www.forbes.com/sites/victoriaforster/2017/12/12/chemotherapy-free-cancer-treatments-move-closer-to-reality/?ss=pharma-healthcare#1486f28e6676

Nov
3
Sandeep Singh Dhillon
Amazon should buy these companies if it wants to get into selling drugs – CNBC News
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Christina Farr | @chrissyfarr

Amazon is considering getting into the business of selling prescriptions online.
CNBC asked five experts to pick the company that Amazon should buy first to help it compete in the $560 billion market.

Amazon should buy these companies if it wants to get into selling drugs, say experts Amazon should buy these companies if it wants to get into selling drugs, say experts. Amazon is already in a range of businesses that touch on the multitrillion medical sector, including selling surgical equipment and supplies, as well as its cloud software services which many big health-care companies use.

But if it wants to get into selling prescriptions, it might speed its time to market by making a buy-up of a company already in the space. There’s a precedent in Amazon’s $13.7 billion acquisition of grocery store chain Whole Foods earlier this year. Amazon has about $13 billion in cash and equivalents, as of September of this year.

So we asked 5 experts for their predictions on the companies that Amazon might look to acquire if it decides to move ahead with becoming an online pharmacy. Their answers have been edited for brevity.

Prediction: Premier

Scott Barclay, partner at venture fund DCVC and former vice president at e-prescription software provider Surescripts.

What is it? A group purchasing organization created to allow hospitals and other providers to pool their purchasing power to secure discounts on medical and other hospital supplies.

Why? According to Barclay, it’s a no-brainer for Amazon to buy a group purchasing organization or “GPO.” And Premier is the largest in the U.S., with a network of more than 3,750 U.S. hospitals and 130,000 other providers.

What’s in it for Amazon? “It’s a low-cost way to break into the (drug) supply chain that touches a large chunk of health care GDP,” Barclay said. “Amazon would quickly create a multibillion-dollar business without fundamentally taking on much risk or even being very innovative.”

Another benefit, according to Barclay, is that it buys Amazon time. It could pick and choose if it wants to go after more challenging opportunities like data and informatics, care provision and health insurance.

Any cons? Premier, which went public in 2013, wouldn’t come cheap — it’s currently got a market cap over $4 billion, and revenue jumped 15 percent to $1.16 billion in fiscal 2016. It has also been on a buying spree of its own of late to strengthen its market position.

Prediction: Express Scripts

Dr. Mark Frisse, Department of Biomedical Informatics at Vanderbilt University Medical Center and former chief medical officer at Express Scripts.

What is it? Express Scripts is the largest pharmacy benefits manager in the United States. These companies, known as PBMs, act as intermediaries between payers, like health insurers, and the rest of the health system.

Why? The culture fit. Amazon and ExpressScripts have a lot in common, according to Frisse. “These companies have the same business acumen, market discipline, and mastery of logistics and delivery.”

ExpressScripts could take on a job that Amazon might want to avoid, suggests Frisse. “Its team brings to the table the capabilities of managing a Byzantine and arcane process of pharmacy regulation,” he said. As Express Scripts CEO Tim Wentworth told analysts in July, it’s far more complicated than just dispensing drugs. Entering the business, “requires you to figure out how not to dispense drugs or to dispense the right drugs as much as it does to dispense them.”

Why not? It would be a massive purchase — Express Scripts has a market cap of more than $35 billion, more than twice the price Amazon paid for Whole Foods, and the company would have to borrow to take that on. It might be an easier option for Amazon to buy a small or midsized pharmacy benefits manager at a lower price, and still gain the national footprint and regulatory expertise.

Prediction: Glooko

Nina Kjellson, Canaan Partners’ biotech and health IT investor (note: Glooko is a Canaan portfolio company).

What is it? Glooko is a subscription-based diabetes management web and mobile app for patients and their health providers. It claims to have millions of users worldwide.

Why? Kjellson sees Amazon having a big impact in public health, especially in areas like obesity and diabetes. With Amazon already starting to sell medical supplies online, Kjellson sees the company carving out a big business in diabetes prescription medications and glucose meters/ strips, which is a $70 billion market and growing. “So Amazon should pick up a diabetes management platform like Glooko to have a consumer and health-care solution that ties the entire disease management ecosystem together in a great, consumer experience.”

Another bonus? As Amazon gets into the grocery business in the wake of its Whole Foods buy-up, it could consider personalized meal recommendations — a benefit for millions of people managing chronic conditions like Type 2 diabetes.

Why not? Privacy. Amazon already knows so much about its customers through their buying habits. Some users might be uncomfortable sharing their health status. Moreover, managing populations with chronic diseases is not Amazon’s core competency.

Prediction: GoodRX

Annie Lamont, managing partner at investment firm Oak HC/FT.

What is it? GoodRx offer coupons that consumers can take to the pharmacy to get discounts on prescription drugs. It claims to have lower prices than what a consumer would otherwise pay out of pocket.

Why? “It’s the most successful consumer-facing app for prescription drugs that is focused on comparing and giving the consumer the lowest cost,” said Lamont. And that’s an ideal fit for Amazon, which prides itself on transparency. GoodRx founder Doug Hirsch has also publicly stated that he would welcome Amazon’s long-rumored entry into the space.

Why not? “Valuation,” suggests Lamont. GoodRx hasn’t raised much in funding — $1.5 million in seed financing and an additional follow-on sum that hasn’t been disclosed — but has grown quickly since its 2012 launch and established itself as one of the biggest players in the space.

Any big risks? GoodRx has contracts with pharmacy benefits managers and retailers behind the scenes, explains Lamont. So there’s always a risk that they pull out, if they’re feeling threatened by Amazon.

Prediction: PillPack

Stephen Buck, co-founder of cancer-advisory site Courage Health and co-founder of GoodRx.

What is it? Pillpack is an online pharmacy start-up that delivers prescriptions by mail in personalized packets, based on when the user needs to take them.

Why? If Amazon got into the business of selling prescription drugs, PillPack seems like an obvious pick to Buck. “It’s a direct-to-consumer company that would fit nicely into Amazon’s Prime delivery service,” he said.

A good deal: PillPack also isn’t encumbered by a large retail operation, Buck said, meaning it doesn’t have a huge network of brick-and-mortar pharmacies. For that reason, it might also be “relatively cheap to buy,” he suggests, despite that it has raised more than $117 million in financing from venture investors to date.

Why not? The product might be too niche for Amazon, suggests Buck. “The whole experience including the packaging might only appeal to a certain segment of people who take multiple medications.”

Read full article here https://www.cnbc.com/2017/11/02/amazon-pharmaceutical-move-acquisition-targets.html?lipi=urn%3Ali%3Apage%3Ad_flagship3_feed%3B%2BaGvcVckQQi8j8AOTm8tcg%3D%3D

Oct
23
Sandeep Singh Dhillon
Amazon Is About To Disrupt The Drug Industry, But Not The Way Most Think – Forbes
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By Steve Brozak, CONTRIBUTOR

It’s now a foregone conclusion that Amazon.com will enter the healthcare sector. Every day there is another article on how Amazon is planning to dominate some new corner of the American economy. One day Amazon is taking down Grainger and Home Depot. The next it’s single-handedly taking down not only FedEx, but also UPS and the United States Postal Service. No sector seems safe as Amazon sails its ship into new waters. But those expecting Amazon to cannon ball into healthcare may need to bide their time.

Two years ago I wrote in Forbes about how Amazon’s entry into healthcare could decimate CVS and Walgreens. I think it’s still possible, but not in the way the markets and media are anticipating today. Amazon’s entry into healthcare will not be sudden. It is likely to be similar to the acquisition of Whole Foods which developed over years as Amazon evolved its strategy in home grocery delivery, one part of the incredibly valuable consumables market. It was accomplished through trial, error and recognition that Amazon needed a bricks and mortar approach to the segment that led to the Whole Foods acquisition. Sudden and complete immersion into the healthcare system as it currently exists could taint the Amazon brand and will make the company vulnerable to regulatory risk. But there are segments of the healthcare market that offer an opportunity for Amazon’s unique capabilities, and there is evidence that Amazon has been making those slow and steady moves toward healthcare.

Becoming A PBM Could Be Amazon’s Healthcare Market Entry

Pharmacy Benefit Managers are the gate-keeping middlemen of the healthcare system. PBMs administer health plans for insurance companies and employers. They manage benefits and treatment costs for these organizations. The more organizations a PBM services, the greater number of patients (or lives, in insurance lingo) under its umbrella. With more lives comes more leverage when a PBM negotiates with pharmaceutical manufacturers for drug prices.

The three largest PBMs are Express Scripts, CVS and OptumRx, covering more than 80% of insured Americans. A PBM that manages tens of millions of lives has greater purchasing and negotiating power than a standalone company with 3,000 employees, or a pension plan with 20,000 lives, or even an independent insurance plan with just a few million lives. Among a slew of other services, PBMs primarily work with their clients to construct and administer a pharmacy benefit program, which is often referred to as a drug formulary. Formularies are just the lists of drugs that are approved or that have preferred pricing for the insurance company through a PBM’s negotiations with the drug’s maker. In exchange for placing its drug on formulary, a PBM receives a preferred price and a cash rebate from the drug’s manufacturer for every prescription it processes. Your insurance plan’s formulary is a major reason why your doctor prescribes you one drug company’s medicine over a competitor’s. Being the preferred drug is the goal. PBMs share a portion of the rebate it receives with their clients (the health plans) and patients receive the benefit of a drug at a preferred price. PBMs also collect fees for transactions and services it processes for its clients.

Target and Walmart learned that merely selling pharmacies in their bricks and mortar operations or through the mail doesn’t equal profits, although a case can be made that it certainly increases foot traffic and store loyalty. Margins are incredibly thin for the kinds of drugs they sell most routinely. In fact, Target sold its in-store pharmacy business to CVS, a pharmacy and a PBM, and Walmart has partnered with McKesson to source its drugs. Becoming a full service PBM would have allowed Target and Walmart to have greater control over margin, but it wasn’t in the cards for either company. While becoming a PBM could be the key to unlocking profits for Amazon, the current political and social environment is unfriendly toward PBMs and drug makers and comes with its own risks.

Despite this, a gamble in healthcare has to happen sooner rather than later. Amazon’s primary customer is between 30 and 45 years old, and that’s a demographic that will soon be looking to fill more and more prescriptions. And if you don’t think their customers’ life cycle milestones are part of retailers’ strategic plans, just look at the pace of acquisitions around baby startups just under a decade ago. Amazon bought diapers.com while Bed Bath & Beyond acquired Buy Buy Baby. Finding an algorithm that figures out when you’re going to have a child before you even think of having one yourself is the holy grail of E-commerce. The birth of a child is a significant milestone around which there is a burst of spending to capture. Likewise, as its consumers age and continue to change their habits through online shopping, healthcare has become the next frontier. Amazon has already made an entry and is setting prices for over the counter medications (OTC). On average, its OTC products, like Tylenol, are cheaper on its website than most bricks and mortar stores. There are several theories circulating around Amazon’s entry into healthcare. Here are my thoughts on three of the most common scenarios being discussed.

Scenario 1: Amazon Acquires Or Partners With A Large PBM

One of the more popular scenarios bandied about is that Amazon may acquire a large PBM player. This is unlikely for a few reasons. Foremost, while it would allow Amazon to quickly enter the healthcare market, integrating the processes and services from a large operation into Amazon would be complicated and chaotic. As the drug pricing debate continues in the mainstream, PBMs have been painted with a big target on their backs as more light is thrown on their opaque and very misunderstood segment of the healthcare system. Just this week, Senator Lamar Alexander (R-TN) wondered out loud why PBMs even need rebates, which is the lifeblood of the industry.

It will be very difficult to recreate the frustration-free consumer experience Amazon currently offers and replicate it through healthcare services as healthcare currently exists. Therefore Amazon will want to separate any healthcare services it offers from its main brand and ease into the business. You could argue that Amazon is already easing in by selling prescription drugs on their Japanese website, perhaps a test case for an American entry (supporting the case for Scenario 3). If you think Amazon can do no wrong, remember this is the company that thought it would be a good idea to emblazon Amazon.com on the back of its Fire Phone, a complete flop. Amazon can’t afford a flameout in healthcare, and making another transformative purchase that could easily eclipse the purchase of Whole Foods so soon, while entirely possible for a company the size of Amazon, would be wholly inadvisable.

As far as partnering goes, sure, Amazon could attempt to partner with a Prime Therapeutics or an Express Scripts. As others have pointed out, Amazon has a strong history of partnering with companies, leveraging its consumer reach and logistical magic to tap into and offer lifelines to companies like Circuit City and Toys R Us. But then what happened? The companies Amazon partners with tend to go out of business as Amazon upends the market.

Scenario 2: Amazon Re-engineers The Wheel With A Smaller PBM

It’s clear that Amazon needs to either re-engineer the PBM wheel or partner with a creative player who already is. While a partnership with a conventional middle market PBM will give Amazon a good entry point in healthcare, a better route would be to partner with an even smaller PBM to really learn the business, gain valuable insights on the industry and bring the PBM model into the 21st century. There are already reports that Amazon, with its 350,000 employees, is trying to administer its own health plan internally as its own PBM. While hiring executives from industry to construct its own virtual PBM is a good way for Amazon to start, it’s still just virtual. An even better path forward would be to acquire or partner with a smaller PBM that already manages millions of lives, self adjudicates (processes its own claims) and can easily manage an additional 350,000 Amazon employees. Through the smart acquisition of a creative PBM, Amazon would learn the business model and incorporate a PBM-like service into an expanding slate of healthcare programs and service offerings.

Scenario 3: Amazon Becomes A Bricks And Mortar Pharmacy In Order To Become A Mail Order Pharmacy

Amazon is well positioned to enter the mail order pharmacy business. Most mail order services are contracted out to a handful of other pharmacies that specialize in mail order delivery. For a while, insurance companies preferred their patients to enlist in a mail order pharmacy because the pricing was competitive compared to going to CVS or Rite Aid. Over the last several years that gap has narrowed. But mail delivery is what Amazon does very well, and combining a mail delivery strategy with a bricks and mortar strategy makes an incredible amount of sense for the company, especially now that it has acquired Whole Foods.

Before acquiring Whole Foods, it never made sense for Amazon to build or to acquire a network of independent pharmacies. Since none of the Whole Foods stores have a pharmacy, Amazon has an opportunity to establish in-store pharmacies that are powered by mail order fulfillment centers. A bricks and mortar strategy should be based around having an in store pharmacist fill as few prescriptions as possible. Rather, a regional prescription fulfillment center could fill and deliver the majority of prescriptions to both the store and to the patient’s doorstep. This is not unlike the current pharmacy model whereby local bricks and mortar pharmacists fill immediately needed prescriptions in the store, while chronically needed medications are filled and delivered to the store by a regional fulfillment center for monthly pickup by the patient. A model in which Amazon combines its mail delivery capabilities with its Whole Foods bricks and mortar would begin to draw two-way traffic from the stores and onto Amazon’s website. As patients sign up and manage their medications, they would create an essential link between the virtual and bricks and mortar Amazon. Shopping for eggs at Whole Foods isn’t going to compel me to go to Amazon.com no matter how many signs Whole Foods hangs in its stores to remind me that they’re now owned by Amazon. But managing my pick up/drop off of medications at my local Whole Foods through my Amazon health portal would draw me into both. Amazon seems to understand this and has already begun to blend its online business in Whole Foods by placing its innovative pick up/return locker system in Whole Foods lobbies.

Of course, while a current generation of Americans prefer to have the personal touch of interacting with and picking up their prescriptions, the trend will soon be to have most drugs delivered by mail. I have even evaluated companies that are working on fill-on-demand drug dispensing vending machines, which may also one day make their way into Whole Foods stores (let’s call them Locker 2.0). To date the one area of true weakness for mail order pharmacy services has been providing essential prescriptions immediately, say, on the day a patient is discharged from the hospital. That’s a weakness Amazon could easily topple with its tests of Prime Now, its same-day two-hour delivery service, another incremental step as Amazon inches toward the healthcare market.

Amazon’s customer base may be young, averaging 35 years old, but they are aging and in the midst of the child-rearing cycle, which requires frequent trips to the pharmacy. Amazon’s customers will need more prescriptions for themselves and for their children. Soon an entire generation will be even more used to buying things online. Right now Amazon has announced it is preparing to make two very big decisions public. The first is whether or not it will get into healthcare, to be announced, supposedly, sometime around Thanksgiving. The second is where it will locate its second world headquarters. If Amazon wants to capture as much of the purchasing a customer can do, it will need to mature with its consumers and formally enter into the healthcare market.

And as for my prediction for Amazon HQ2: It’s Boston.

Oct
13
Sandeep Singh Dhillon
NIH And 11 Pharmaceutical Companies Announce $215 Million Collaboration – Forbes
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By Ellie Kincaid , FORBES STAFF

The U.S. National Institutes of Health and 11 pharmaceutical companies today announced the launch of a five-year cancer immunotherapy research collaboration as part of the Cancer Moonshot. In total the partners will contribute $215 million to what they are calling the Partnership for Accelerating Cancer Therapies (PACT).

PACT’s fundamental question, NIH director Francis Collins told reporters in a press conference, is, “why doesn’t immunotherapy work for all patients in all types of cancer, and what can we do about that?”

Top priority in answering that question is testing immune- and cancer-related biomarkers in clinical trials to understand the mechanisms of how cancers respond to or resist immunotherapy. With standardized biomarkers to look at and harmonized assays to test them across many different trials, data from different studies will be more easily compared, Collins said.

Developing standardized biomarkers for immuno-oncology will be “extremely enabling,” like standardizing IP addresses in the early days of the web, Thomas Hudson, vice president for oncology discovery and early development at AbbVie, said at the press conference.

He expects having standard biomarkers and sharing data will help provide a scientific basis for deciding which cancer drugs to try in combination. Given the sheer number of potential combinations, that’s “one of the key reasons we got involved in PACT,” he said. “We don’t think random combinations are the way to go.”

The data for researching biomarkers will come from four National Cancer Institute-funded Cancer Immune Monitoring and Analysis Centers at Dana-Farber Cancer Institute, Stanford Cancer Institute, Precision Immunology Institute and the Tisch Cancer Institute at Icahn School of Medicine at Mount Sinai and University of Texas MD Anderson Cancer Center. The centers will support adult and pediatric immunotherapy trials with tumor and immune profiling.

Getting access to data from clinical trials NCI supports was “very attractive,” Hudson said.

Which biomarkers the collaboration will focus on first is still to be discussed at a meeting the companies will likely have next month, said Douglas Lowy, NCI’s acting director.

The 11 pharmaceutical companies participating are AbbVie, Amgen, Boehringer Ingelheim Pharma GmbH & Co. KG, Bristol-Myers Squibb, Celgene Corporation, Genentech, Gilead Sciences, GlaxoSmithKline, Janssen Pharmaceutical Companies of Johnson & Johnson, Novartis Institutes for Biomedical Research and Pfizer. Each will contribute up to $1 million per year for the five-year partnership, totaling $55 million.

The NCI will make up the rest with $160 million in funding mostly from the Cancer Moonshot, with some coming from regular appropriations, Lowy said.

Read the full article at https://www.forbes.com/sites/elliekincaid/2017/10/12/nih-and-11-pharmaceutical-companies-announce-215-million-collaboration/#287c1aef7b54

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/

Aug
23
Sandeep Singh Dhillon
MMA urges MOH to clarify process for prescribing branded medicines to patients, especially pensioners – MIMS Malaysia
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The Malaysian Medical Association (MMA) urged on 18 August, for a more efficient and transparent process for prescribing branded medicines to patients, amid the Ministry of Health’s (MOH) “generic first” policy.

MMA president Dr Ravindran R. Naidu said approval for giving patients drugs outside the ministry’s formulary – at public clinics or hospitals – should not rest solely with the Health director-general.

“It’s important to have a transparent process, so everyone knows exactly what is needed for approval,” asserted Dr Ravindran.

“If a safe and effective generic is available in MOH formulary, every public-sector patient should receive the same drug, except where the patient cannot tolerate the generic (e.g. due to allergy). In case of intolerance of generic, the government should supply original drugs ― it’s not the patient’s fault,” he added.

Dr Ravindran said that MOH should instead bring in original drugs with good cost-to-benefit ratio should generics be unavailable, pointing out that “many” drugs do not have generic versions as well.

“Generic first” policy applies to all now

This comes after Health deputy director-general Datuk Dr S. Jeyaindran confirmed, that as part of the “generic first” policy, approximately 700,000 retired government servants will now no longer be reimbursed if they buy brand-name medications that are unavailable at public hospitals – unless they fulfil certain conditions.

Instead, they will receive generic medicines, like other patients, he added.

The policy is part of MOH’s management of medical treatment of federal government retirees and pensioned military veterans, together with the Ministry of Higher Education (MOHE) and the Defence Ministry (MOD), which took effect on 1 June this year.

The change occurred after the contract for the supply of medicines with Oratis Rx Sdn Bhd ended early this year. Pensioners are now told to pay for their purchases first and submit claims subsequently – a process which takes an average of three to six months.

“The process has been made difficult, so people opt for the cheaper generics,” said a doctor who requested anonymity.

Dr Jeyaindran assured that the standard operating procedure (SOP) has been fine-tuned and put into place in the last six weeks, with the Public Service Department (PSD) transferring funds to MOH for pensioners’ healthcare and the Retirement Fund (Inc) (KWAP), handling all dialysis and dialysis-related claims.

Change in SOP causes confusion
However, pensioners lamented that with the change, hospitals needed to approve of the prescriptions first. If approval is declined, pensioners would have to directly pay out-of-pocket for medicines that were previously dispensed for free through Oratis Rx Sdn Bhd.

According to a circular published on 30 June, approval was also needed from the Health director-general to get drugs outside the MOH formulary. This was based on three conditions: drugs in the formulary were ineffective in treating the patient; the patient suffered adverse effects from drugs in the formulary; and the application was not intended to continue medical treatment that had been started at a private hospital or a hospital from another ministry.

This has generated confusion and unnecessary backlog as the rule took effect on 1 June, but only notified relevant parties on 30 June.

The MOH however, remained firm and Dr Jeyaindran cautioned, “If doctors want to use a drug outside the Blue Book, they must justify why the patients need it.”

Efficiency compromised with change

MMA past president Dr Milton Lum said generics are sometimes ineffective in cases of dialysis, high blood pressure and arthritis. There is also an increased resistance to generic antibiotics and common drugs, and there are also no generics for many cancer drugs.

“The Blue Book is based on essential medicines,” explained Dr Lum, referring to the MOH formulary. “They cover the majority of common illnesses, but not everything.”

However, local generics pharmaceutical manufacturers have asked Dr Lum to back up his statement that generics are ineffective in certain cases on 21 August.
In addition, Dr Lum also questioned how long patients and doctors must wait for approval of applications to use branded medicines.

“Is there a process to fast-track it, particularly when it’s needed urgently? What happens if the DG is not in the country? Who approves it then?” he asked.

“It has to be an efficient process with certain timelines for decision to be made, for approval to be given. If you give approval, there is a time lag before you obtain the medicine. So, there has to be efficiency in the process and timelines to be complied with,” he added. MIMS

The full article first appeared at https://today.mims.com/topic/mma-urges-moh-clarify-process-for-prescribing-branded-medicines-to-patients-especially-pensioners?country=Malaysia&channel=GN-Local-News-MY&elq_mid=18726&elq_cid=24884

Aug
22
Sandeep Singh Dhillon
Samsung Wants to Become a Drug Company – Investopedia.com
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Samsung Electronics, the South Korean consumer electronics company is expanding efforts to diversify into the pharmaceutical business, landing its first deal to develop drugs for diseases that are hard to treat.

While the company is best known for churning out mobile phones, semiconductors and other consumer electronics, it recently started to move into the drug market. In late July, The Wall Street Journal reported the company was gearing up to launch a generic version of Johnson & Johnson’s rheumatoid arthritis drug Remicade. The newspaper is now reporting that Samsung is forming a partnership with Japan’s Takeda Pharmaceutical Co. to fund and develop several drugs over the next few years with the first effort focused on a treatment for severe acute pancreatitis. Terms of the deal were not disclosed.

The Path Less Risky
According to the report, the company’s move to get into novel treatments of hard-to-treat drugs is a big enhancement of its drug efforts, but one that is also risky. Only one in 10 drugs that end up with human trials gets approval while companies could spend hundreds of millions of dollars and years working on formulations at the experimental level. Developing a generic version of a popular drug, as with Remicade, has proved to be a less risky strategy for many drug companies. According to the Journal, citing data from EvaluatePharma, the biologic market is on track to have $214 billion in sales in 2017 alone and sales are projected to hit $276 billion by 2020.

With saturation in the smartphone market, and Samsung seeing increased competition from U.S. and Chinese technology firms, the company is seeking ways to diversify beyond consumer electronics, and the pharmaceutical industry is one area it has set its sights on. The generic version of Remicade is expected to sell for 35% less than the current list price for the popular drug.

Samsung got approval from the Food and Drug Administration for the generic version in April, and the launch marked the company’s first push into the pharmaceutical market under its new Samsung Bioepis Co. unit. The biotech arm is going after generic versions of branded drugs that are made from living cells and will address complex diseases like arthritis and cancer, reported the Journal. The brand-name treatments can often cost as much as tens of thousands of dollars a year, presenting a big opportunity for lower-cost players to grab market share. And it is now adding novel drug therapies to its focus, which has always been a goal of the Bioepis unit. “At this stage of our company’s development, we believe this is the next logical step,” said Mingi Hyun, a Samsung spokesman, to the Journal, noting that the unit has held talks with other pharmaceutical and biotech companies to pursue similar deals to its partnership with Takeda.

Read more: Samsung Wants to Become a Drug Company | Investopedia http://www.investopedia.com/news/samsung-wants-become-drug-company/#ixzz4qUjBsxLc
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Aug
17
Sandeep Singh Dhillon
Goldman Sachs breaks down how Amazon can jump into health care – CNBC
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By Christina Farr, CNBC

Amazon is speeding its efforts to crack the health care market, hiring a number of high-profile executives, testing Echo technology in top hospitals and creating a secret “1492” team dedicated to health-technology opportunities like telemedicine and electronic medical records.

Goldman Sachs is now out with a 30-page report from five research analysts on Amazon’s likely ambitions in the $560 billion prescription drug market. The note cites CNBC’s reporting on the 1492 group and Amazon’s hiring of a general manager to lead its pharmacy unit.

Here are some of the key insights from the report:

  • Rather than replacing pharmacies right away, Amazon might start by partnering with a pharmacy benefits manager (PBM), which acts as an intermediary between payers, like health insurers, and the rest of the health system. That would provide “access to patient data and the potential to cross-sell related products.”
  • Amazon could ultimately improve price transparency for the consumer and reduce out-of-pocket drug costs. But it would likely start by speeding up the drug delivery process and facilitating at-home delivery.
  • Amazon could also become an online pharmacy, retail and online pharmacy, integrated PBM and online pharmacy, or handle drug distribution to pharmacies.
  • One potential — and overlooked — challenge for Amazon might be the so-called “age gap.” Amazon’s customers tend to be younger and healthier than people who typically take prescription drugs.
  • Amazon could move into digital health by using the Echo in clinical settings and developing tools for telemedicine and remote patient monitoring. “Imagine seeing a virtual doctor on your Amazon app, having it prescribe you a certain medication, and then tapping a ‘buy now’ button — all without leaving your home.”

Jul
5
Sandeep Singh Dhillon
US Pharma Looks at Blockchain Tech to Track Prescription Drugs – Cryptocoinsnews.com
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U.S. Pharmaceutical companies and the Food and Drug Administration (FDA) could soon track prescription drugs over an interoperable blockchain as a means to detect and put an end to counterfeit medicines in the market.

The solution comes from San Francisco-based blockchain startup Chronicled and its partnership with LInkLab, a life sciences supply chain. The two companies have launched a blockchain “track and trace” pilot at a recent one-day event in San Francisco. Developed for the pharmaceutical industry, the solution could prove a viable means to curbing counterfeit drug distribution and sales in the United States and beyond.

Attended by representatives from global pharma manufacturers, wholesalers and hospitals, the blockchain pilot event also saw participation from enterprise IT giants and blockchain tech companies. The event, according to Chronicled, marks the first phase of a pilot to develop an interoperable blockchain platform for the pharmaceutical industry.

Chronicled co-founder and chief performance officer Samantha Radocchia stated in a release:
We will be working closely with teams at leading enterprise blockchain projects over the coming months to identify the most suitable enterprise blockchain to serve as a data utility for the pharmaceutical industry.

The blockchain platform will adhere and satisfy the FDA’s Drug Supply Chain Security Act, signed into effect by former US President Obama in November 2013. The act was and signed to ‘develop an electronic, interoperable system to identify and trace certain prescription drugs as they are distributed in the United States.’

With this in mind, the blockchain solution will developed to support and comply with DSCSA protocols. Further, the pilot solution will focus on data privacy while adhering to GS1 standards, a global standardized system for traceability that can be implemented by all participants in a supply chain across borders globally.

Altogether, the blockchain tech solution aims to standardize a less expensive, less cumbersome and more efficient and secure approach to facilitate prescription drug distribution.

“The first phase of this project is to prove that one global pharmaceutical manufacturer can comply with their DSCSA regulatory obligations and meet the 2017 and 2023 requirements,” stated Susanne Somerville, co-founder of the LinkLab. “We are excited to be partnering with a major pharmaceutical player in this first phase.”

Future phases will foster the involvement of other participants in a typical pharma supply chain process, straight from the manufacturer to the pharmacy and hospitals.

One of blockchain technology’s core offerings that make it a no-brainer for supply chains across industries is its immutable, time-stamped, tamper-proof ledger, accessible by its all or pre-approved participants. Last week, Australia Post, the country’s postal service operator announced a partnership with Alibaba to curb the rise of counterfeit food exports from Australia to China, using blockchain technology to improve traceability.

Jul
4
Sandeep Singh Dhillon
Did You Grow Up Thinking You’re Allergic To Penicillin? Guess Again – Forbes
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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.”