Pharmaguy's Insights Into Drug Industry News
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Pharmaguy's Insights Into Drug Industry News
Pharmaguy curates and provides insights into selected drug industry news and issues.
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The Trouble With Rare-Disease Drugs... Price!

The average U.S. patient on an orphan drug last year relied on a $136,000 therapy, a figure that’s climbed 38 percent since 2010. A fraction of a teaspoon of Soliris [a drug by Alexion approved to treat a rare blood disorder called atypical hemolytic-uremic syndrome, or aHUS, and priced from $500,000 to $700,000 a year], administered in a single 35-minute treatment, costs more than $18,000, and patients might need 26 treatments a year for the rest of their lives. With this single drug accounting for almost all its revenue, Alexion has created enormous wealth out of an estimated 11,000 customers. It generated $3 billion in sales in 2016.

 

Having to rely for profits on a small number of customers who are each potentially worth millions of dollars causes side effects of its own. For years, the sales culture at Alexion was so pressure-packed that aggressive phone calls to doctors were among its milder transgressions. Ethical lines were routinely crossed, troubling many of its workers, according to interviews with more than 20 current and former employees and more than 2,000 pages of internal documents.

 

Further Reading:

Pharma Guy's insight:

I notice that price-gouging Alexion Pharmaceuticals was NOT ousted by PhRMA in its recent “purge” (read “@PhRMA Ousts Marathon #pharma et al to Appease Trump & His Campaign Promises”; http://sco.lt/8Zj01B).

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John Carroll, Editor-in-Chief of EndPoints, to Trump: Don’t Bet on a Faster FDA to Lower Drug Prices

John Carroll, Editor-in-Chief of EndPoints, to Trump: Don’t Bet on a Faster FDA to Lower Drug Prices | Pharmaguy's Insights Into Drug Industry News | Scoop.it

[John Carroll, comes down hard on failed Duchenne drug & Donald Trump’s criticism of FDA in a recent “Thumbs Up/Down” opinion piece.

 

Regarding a failed Duchenne drug, Carroll says:]

 

I never understood why the EMA would suddenly reverse itself in 2014 and allow PTC to sell ataluren as its only approved therapy on the continent for Duchenne muscular dystrophy. And it was even more perplexing to see the European regulator come back last year and decide to allow the drug to remain on the market as PTC lined up a new late-stage study over an expansive 5-year grace period.

 

At that point, not only did the EMA know full well that the drug had simply failed a Phase III trial for Duchenne, supposedly designed so that it would overcome the flaws in its failed Phase IIb trial, but the FDA had locked the door to PTC’s executive crew, unwilling to spend any time reviewing a drug that was woefully unacceptable for marketing.

 

This week, PTC put what should have been the last nail in ataluren’s coffin with fresh evidence that this drug is a dud. It failed the Phase III for cystic fibrosis. Case closed.

 

Except that it’s not and won’t be. This drug will continue to be sold despite the fact that it has repeatedly and decisively failed to clear late-stage trial hurdles. Even NICE was willing to endorse it for the UK after working out a price with PTC. PTC expects to earn more than $100 million this year from sales. And it has the backing of patient advocates — Duchenne families — who swear by it.

 

As we found out from Sarepta’s Exondys 51, though, regulators can be persuaded to overlook gaping holes in a Duchenne drug’s development program to make way for an approval (read “Path Taken by Sarepta NOT a Good Model for Other Rare Drug Approvals, Says FDA Official Who Should Know”; http://sco.lt/7stCs5).

 

There has to be a better way. If the FDA and EMA want to handle new drugs for Duchenne MD according to a different standard, then they should set up special groups inside the agencies that can help biotechs direct small but better designed studies that can offer real-world evidence of efficacy and safety. The path we’re on now is a disaster for patients, families and the societies forced to pay for these wildly expensive, unproven drugs.

 

[Regarding president Trump’s criticism of FDA’s approval process, Carroll says:]

 

The drug approval process, he says, is slow and burdensome (read , and he plans to make some big changes (here; http://sco.lt/8bbHJB). If you deregulate drug development, he has said before, drugs can speed through the FDA faster and the price will drop.

 

That, of course, is simply ridiculous. We’ve seen quite a few drugs speed through the review process in the last few years, and the price was in no way discounted as a result.

 

When Biogen, which has its own efficiency issues to address, got an approval for Spinraza last December just three months after it was filed, do you think the big biotech offered a discount as a result?

 

Not a chance. It priced the rare disease drug at $750,000. With its Tecfidera franchise on the wane, Biogen needs new revenue to satisfy investors. And how is that in any way unusual?

 

Gilead was a model of efficiency and expertise when it whipped through a development program for new hep C drugs. Sovaldi was the third drug approved under the FDA’s breakthrough drug designation, designed to help speed development timelines. It originally cost a small fortune. Cancer drug development has been revolutionized by the BTD program over the past few years. Have prices come down?

 

There are things that the FDA can do better that will lower some drug costs. But unless the president plans to simply gut development rules — a real possibility — don’t look for a faster FDA to bring down drug prices. Lower costs on drug programs do not lower prices.

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“Repurposing” Drugs as “Orphans” Drives Up Prices

“Repurposing” Drugs as “Orphans” Drives Up Prices | Pharmaguy's Insights Into Drug Industry News | Scoop.it

 

 Sen. Charles Grassley (R-IA) on Friday confirmed to Focus that he is gathering more information and discussing with his staff and interested parties a possible inquiry into the Orphan Drug Act’s abuses leading to high drug prices.

 

“Based on the reporting from Kaiser Health News [KHN] about how the orphan drug provisions appear to be stretched beyond their original intent, and the strong consumer concern about high drug prices, I'm interested in learning whether the unanticipated uses of the provisions are contributing to high prices for commonly used drugs,” Grassley said in an emailed statement to Focus. “My staff is meeting with interested groups and other Senate staff to get their views on the extent of the problem and how we might fix it. I also continue to work on other ways to help bring down drug prices, such as increasing competition with available products.”

 

Grassley’s comment follows a KHN investigation from January detailing how the incentives created by the Act’s provisions have led to more than 200 companies bringing almost 450 orphan drugs to market since the law took effect in 1983. The incentives are for diseases affecting fewer than 200,000 individuals in the US per year and include seven years of market exclusivity, tax credits for clinical trial expenses, user fee waivers and federal grants.

 

Dr. Gayatri Rao, director of the FDA’s Office of Orphan Products Development, told KHN that she would look into the investigation and pointed out that the “repurposing” of drugs does have scientific and patient benefits.

 

“We always talked about how we permit the second bite of the apple, third bite of the apple, as one small way to incentivize repurposing,” Rao told KHN, noting that industry and patient groups have been pressing the FDA for more incentives. “Now, all of sudden, it seems like, wow, this practice may be driving up prices.”

 

In 2015, researchers at Johns Hopkins called for reforms to the Act, saying loopholes have allowed drug companies to skirt its intent by taking advantage of its incentives for non-orphan conditions.

 

An article in PLOS Medicine in early January also noted: “Orphan-designated drugs to treat biomarker-defined subsets of common conditions have a number of characteristics that make them ill-suited to the orphan drug designation, including short development times and rapid expansion of off-label indications after approval. Application of the Orphan Drug Act in these cases risks wasting resources that might be better focused on truly rare conditions.”

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How "Patient Centricity" Helps Pharma Develop & Gain Approval for Orphan Drugs

How "Patient Centricity" Helps Pharma Develop & Gain Approval for Orphan Drugs | Pharmaguy's Insights Into Drug Industry News | Scoop.it

In rare diseases, true patient engagement is critical to every step of a drug's development, from clinical trials and regulatory approval, to patient diagnosis and adherence.

 

Why is that? Because patients with rare diseases (and their families) are essentially the thought-leaders of their condition. Usually these patients are very few in number, and have high unmet needs. Management of their condition is often fragmented, with knowledgeable healthcare professionals few and far between. Patients can feel isolated which begets active participation in both online and offline networks with other affected patients and families. Many rare diseases specifically affect children, which causes huge distress for parents and family members, who become very actively involved.

 

So despite often very challenging circumstances, patients with rare diseases and their families and caregivers are typically very well-informed about their condition, very connected and active in the space, and very willing to be involved when it comes to potential advances in the area.

 

Putting the patient at the centre is an absolute necessity for pharma companies involved in this space, and several companies have been very successful in doing so, for example Sanofi Genzyme, Novo Nordisk and Novartis, that were ranked the top three companies for corporate reputation according to rare disease patient groups (however, read, “Report: #Pharma Does Not Feel the Pain of Orphan Patients”; http://sco.lt/6RMFU1).

 

So how do companies successfully engage with patients and ensure the orphan drug they have in development will reach the patients who need it?

 

In both the US and Europe, a range of schemes to facilitate patient involvement in the regulatory process has been established (read, for example, “How a #pharma Funded ‘Grassroots’ Patient Advocacy Campaign Changed FDA's Approval Process”; http://sco.lt/4tqlbF). The FDA has introduced a Patient Engagement Advisory Committee and the EMA has patient representatives on a range of its committees, including its management board.

 

Orphan drug companies collaborate with patient advocates for example to demonstrate the burden of the disease, add it to the policy agenda and help to incorporate the rare patients' benefit-risk preferences into a structured evaluation process.

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Insurance Companies Accuse #Pharma of Gaming the Orphan Drug Approval System

Insurance Companies Accuse #Pharma of Gaming the Orphan Drug Approval System | Pharmaguy's Insights Into Drug Industry News | Scoop.it

Pharma companies may be exploiting a legal loophole that increases utilization costs of certain drugs, according to a study from AHIP (America's Health Insurance Plans).

 

"Orphan drugs" treat rare diseases impacting populations fewer than 200,000 annually that are otherwise lacking special treatment. Pharmaceutical companies can make tremendous profits by finding uses for those drugs outside of the original condition the medications were intended to treat. While the practice is legal, some experts say pharmaceutical companies are “gaming the system,” according to the brief.

 

Almost half of the orphan drug use in the study was for non-orphan diseases and conditions.

 

AHIP notes,"in a 2012 study on more than 350 orphan drugs approved through mid-2010, researchers found that 43 drugs, having at least one approved orphan indication, achieved global sales in excess of $1 billion in 2008."

 

Market exclusivity and extremely high prices have created such “blockbuster” orphan drugs, “a result that seemingly runs counter to the spirit” of the law which incentivizes rare disease research, the report adds.

 

[FDA approved 45 new drugs in 2015, four more than in 2014 and the highest number since 1996. Twenty (20) of those (43%) were "orphan" drugs. More on this here: http://sco.lt/7ZufVx]

Pharma Guy's insight:

Even CRESTOR is an “Orphan Drug”: http://sco.lt/4pauhN Also read “Orphan Drug Sales: An Enduring Prospect for #Pharma Profits”; http://sco.lt/5oCT1V and "Orphan Drugs Now Where the Money Is"; http://bit.ly/pgdaily111113-3A 

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Gang-Stalking - A Rare Disease Ripe for #Pharma Orphan Drug Solution?

Gang-Stalking - A Rare Disease Ripe for #Pharma Orphan Drug Solution? | Pharmaguy's Insights Into Drug Industry News | Scoop.it

Nobody believed him. His family told him to get help. But Timothy Trespas, an out-of-work recording engineer in his early 40s, was sure he was being stalked, and not by just one person, but dozens of them.

 

He would see the operatives, he said, disguised as ordinary people, lurking around his Midtown Manhattan neighborhood. Sometimes they bumped into him and whispered nonsense into his ear, he said.

 

“Now you see how it works,” they would say.

 

At first, Mr. Trespas wondered if it was all in his head. Then he encountered a large community of like-minded people on the internet who call themselves “targeted individuals,” or T.I.s, who described going through precisely the same thing.

 

The group was organized around the conviction that its members are victims of a sprawling conspiracy to harass thousands of everyday Americans with mind-control weapons and armies of so-called gang stalkers. The goal, as one gang-stalking website put it, is “to destroy every aspect of a targeted individual’s life.”

 

Mental health professionals say the narrative has taken hold among a group of people experiencing psychotic symptoms that have troubled the human mind since time immemorial. Except now victims are connecting on the internet, organizing and defying medical explanations for what’s happening to them.

 

The community of targeted individuals, conservatively estimated to exceed 10,000 members, has proliferated since 9/11, cradled by the internet and fed by genuine concerns over government surveillance. A large number appear to have delusional disorder or schizophrenia, psychiatrists say.

 

Yet, the phenomenon remains virtually unresearched.

 

For the few specialists who have looked closely, targeted individuals represent an alarming development in the history of mental illness: thousands of sick people, banded together and demanding recognition on the basis of shared paranoias.

 

They raise money, hold awareness campaigns, host international conferences and fight for their causes in courts and legislatures.

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Big #Pharma "Reinvents Innovation" by Investing in High-priced Specialty Drugs

Big #Pharma "Reinvents Innovation" by Investing in High-priced Specialty Drugs | Pharmaguy's Insights Into Drug Industry News | Scoop.it

Graham Lewis, IMS Health, VP Global Pharma Strategy, predicted that growth of the US pharmaceutical market will accelerate. He cited both the slow-down in developing markets and growing pharmaceutical company interest in specialty drugs as a major drivers.

Specialty drugs – those that costs more than $600 per month according to the Centers for Medicare & Medicaid Services – are hard to make and difficult to copy, which makes them attractive to manufacturers concerned about potential generic competition.

The challenge is finding patients and payers that can afford them, which is why many specialty drug developers focus on selling them in the US.

In fact, according to Lewis, 60% of the global growth in the specialty drug sales will be seen in the US over the next few years.

The drug industry’s interest in hard to make specialty drugs has reinvigorated R&D according to Lewis, who said: “The developed market, led by the US, has really reinvented innovation.

“In the past five years we’ve seen more innovation in pharma than in the previous 15,” he said, and these recent innovations are having “a gravitational effect.”

“If you’re in in innovation, this is the picture, and it’s all about the United States.”

Pharma Guy's insight:

More than $374 billion was spent on prescription drugs in the United States in 2014, according to a report from the IMS Institute of Health. That was a 13.1% jump from 2013, and the highest growth rate in more than a decade, says ExpressScripts. See http://sco.lt/8Eca6D I always said specialty and orphan drugs drugs were where the money is: http://bit.ly/orphanmoney 

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2015 Was Another Good Year for Orphan Drugs & Pharma Marketers

2015 Was Another Good Year for Orphan Drugs & Pharma Marketers | Pharmaguy's Insights Into Drug Industry News | Scoop.it

UPDATED 4 Jan 2016: FDA approved 45 new drugs in 2015, four more than in 2014 and the highest number since 1996. Twenty (20) of those (43%) were "orphan" drugs. This compares to 17 orphan drugs approved in 2014 or 41% of the total (see CDER New Drug Review: 2015 Update and "2014 Was a Good Year for FDA & Pharma").


That's good news for the pharma industry, which often submit drugs to the FDA as orphan drugs but once approved the drugs are "used broadly off-label with the lucrative orphan drug protections and exclusivity benefits," according to the authors of a study recently published in the American Journal of Clinical Oncology (read "#Pharma Welfare: 'Orphan' Blockbuster Drugs on Rise - Including Crestor!"). 

Pharma marketers also have good news regarding FDA marketing enforcement actions in 2015.


More about that here.

Pharma Guy's insight:

Meanwhile, FDA is slow to approve generic drugs, which helps boost drug prices; see http://sco.lt/5mHdZ3 

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Report: #Pharma Does Not Feel the Pain of Orphan Patients

Report: #Pharma Does Not Feel the Pain of Orphan Patients | Pharmaguy's Insights Into Drug Industry News | Scoop.it

As patients and caregivers are confronted with a dramatically changing healthcare landscape, American patient advocacy organizations are evolving the work they do for these communities and would like to see their relationships with pharmaceutical companies also change to keep pace.


This report from inVentiv Health Public Relations Group is based on more than two months of interviews with nearly four dozen patient advocacy organizations representing patients with a variety of diseases and conditions, including cancer, mental health and rare diseases. It highlights areas in which the patient advocates would like to see change in their relationships with pharmaceutical partners, as they work together to ensure that new medications arriving on the market meet the needs of people with devastating diseases.


From the report:


"To cite just one flashpoint, rising co-insurance in many health plans under the ACA, as well as narrowing formularies and physician networks, have exposed patients with rare cancers and other orphan diseases to more burdensome costs. Pharmaceutical partners aren’t always able or willing to alleviate their pain. When drug companies fail to respond to cost concerns, patient groups feel abandoned by their pharma partners."

Pharma Guy's insight:


The #1 item on the patient advocacy wish list: "Improve transparency and authenticity"

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"Venture Philanthropists" Bet Big on Orphan Drugs for Rare Diseases

"Venture Philanthropists" Bet Big on Orphan Drugs for Rare Diseases | Pharmaguy's Insights Into Drug Industry News | Scoop.it
Venture philanthropy risks benefiting companies, not patients.


ROBERT J. BEALL, the president and chief executive of the Cystic Fibrosis Foundation, called his recent decision to sell the royalty rights to his organization’s research a “game changer.” Indeed: Deals like this, in which an investment company paid the foundation $3.3 billion for its future royalties from several cystic fibrosis drugs it helped finance, could revolutionize the way medical research is funded. Rather than the staid model of government-funded institutions handing out grants to academic research facilities, a new breed of “venture philanthropies” like the Cystic Fibrosis Foundation could corral private investment into developing lifesaving drugs quickly and cheaply.


The problem is that venture philanthropy is, essentially, another term for privatizing scientific research. Instead of decisions about the fate of scientific funding being made by publicly oriented institutions, those decisions are being put in the hands of anonymous philanthropists and ostensibly benevolent nonprofits.


So far, there is no effort to extend government price controls to venture-philanthropy-derived research. The Cystic Fibrosis Foundation did little to lobby for lower prices on the drugs that were developed from the research it funded. As a result, Kalydeco, a cystic fibrosis medication it funded, is one of the most expensive drugs available, at $300,000 a year.


One argument in favor of venture philanthropy is that it creates a way to sustain small foundations that study rare diseases that, from a for-profit point of view, aren’t worth investigating.


But while Big Pharma might be faulted for funneling billions of dollars into erectile-dysfunction drugs and off-label drug marketing, researching extremely rare diseases may also represent a misuse of public and private funds. Efforts to cure, rather than treat or prevent, obscure diseases can be expensive, diverting investment from more common afflictions. The high costs of focusing on rare diseases are then eventually pushed onto the health care system by way of egregiously high drug prices. Such a choice involves an incredibly complex moral calculus, one that is best processed by democratic public institutions.


To make medical advancements truly philanthropic, the profit motive needs to be removed from the equation. If the intent is to cure rare diseases, then we should be increasing the budget for the National Institutes of Health and other research initiatives. Instead of gala balls and donor drives, higher taxes on the same rich benefactors could be used to fund the research that isn’t already being supported. Biotech patents developed through venture philanthropy should not have exclusive rights attached to them.


This would allow generic versions of drugs onto the market, which would go a long way toward keeping health care costs down and not driving the uninsured into debt.


Pharma Guy's insight:


When a dumb reporter asked "Willie" Sutton, the famous bank robber, why he robbed banks, he is reported to have said "Because that's where the money is." Duh!

Until a few years ago, if you asked a pharmaceutical company executive why his or her company developed and marketed an "orphan drug" -- ie, a drug for a disorder affecting fewer than 200,000 people in the U.S. -- you would likely have gotten a response such as "because there is an unmet medical need" or something similar.

Today, however, orphan drugs also have the potential to turn into blockbusters; ie, be where pharma's money is at (see "Analyst says smart money is on drugs for certain orphan diseases" and "New Big Pharma Economies of Scale: Less Patients Needed to Reach Blockbuster Sales").


From a Feb 5, 2015, editorial by a CF patient in Cystic Fibrosis Today:


Despite the incredible success of the CF Foundation in the sale of its royalty rights through the venture philanthropy model, concerns have been raised about collaboration between non-profits and pharmaceuticals.


“There is some concern that a profit motive could divert the organizations from their primary mission — helping patients — and create a conflict of interest,” Andrew Pollack comments in his New York Times article. “For instance, the price of the main drug developed through Cystic Fibrosis Foundation’s investment is $300,000 a year.”

The article addresses a serious issue surrounding the high cost of CF modulators that can be cost prohibitive for patients. The high costs begin to enter the ethical dilemma, which asks, how much would you pay to be healthy?


The argument presented in the article is that if non-profits such as the CF Foundation are receiving royalties from the sale of drugs, there is less incentive for them to lobby the company to reduce the costs for the patients. However, Dr. Beall of the CF Foundation has contended that despite their concerns for the high priced ivacaftor (kayldeco) there was little they could do to reduce the cost, which was at the discretion of Vertex.


I am the first to argue that it is an imperative that every person with CF who is eligible for novel CF modulator therapies have access to the drugs. However, it is misplaced to discredit the venture philanthropy model as a result, since the discussion surrounding the affordability of the drug only exists because the CF Foundation invested the capital to have it created.


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Because of Potential Abuse, Orphan Drug Act Might Need to Be “Repealed & Replaced”

Because of Potential Abuse, Orphan Drug Act Might Need to Be “Repealed & Replaced” | Pharmaguy's Insights Into Drug Industry News | Scoop.it

Building on weeks of mounting pressure to address high prescription drug prices, three influential U.S. senators have asked the government's accountability arm to investigate potential abuses of the Orphan Drug Act.

 

In a letter to the U.S. Government Accountability Office, Sens. Orrin Hatch, R-Utah, Chuck Grassley, R-Iowa, and Tom Cotton, R-Ark., raised the possibility that regulatory or legislative changes might be needed "to preserve the intent of this vital law" that gives drug makers lucrative incentives to develop drugs for rare diseases.

 

"While few will argue against the importance of the development of these drugs, several recent press reports suggest that some pharmaceutical manufacturers might be taking advantage of the multiple designation allowance in the orphan drug approval process," the letter published Friday states.

 

In January, NPR published a Kaiser Health News investigation that found the orphan drug program is being manipulated by drug makers to maximize profits and to protect niche markets for medicines being taken by millions (read “Orphan Drug Sales Offer a Glimpse of ‘Sheer Greed’”; http://sco.lt/9ErY1p).

 

Congress overwhelmingly passed the 1983 Orphan Drug Act to motivate pharmaceutical companies to develop drugs for people whose rare diseases had been ignored. Drugs approved as orphans are granted tax incentives and seven years of exclusive rights to market drugs that are needed by fewer than 200,000 patients in the U.S.

 

In recent months, reports of five- and six-figure annual price tags for orphan drugs have amplified long-simmering concerns of abuse of the law. The senators' call for a GAO investigation reflects that sentiment.

 

The senators asked the GAO for a list of drugs approved or denied orphan status by the Food and Drug Administration. It also asked whether resources at the FDA, which oversees the law, have "kept up with the number of requests" from drug makers and whether there is consistency in the department's reviews.

 

The Kaiser Health News investigation found that many drugs that now have orphan status aren't entirely new. More than 70 were drugs first approved by the FDA for mass-market use. Those include cholesterol blockbuster Crestor (read “#Pharma Welfare: ‘Orphan’ Blockbuster Drugs on Rise - Including Crestor!; http://sco.lt/4pauhN), Abilify for psychiatric disorders and rheumatoid arthritis drug Humira, the world's best-selling drug.

 

Others are drugs that have received multiple exclusivity periods for two or more rare conditions. About 80 drugs fall into this category, including cancer drug Gleevec and wrinkle-fighting drug Botox.

 

Further Reading:

 

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Orphan Drug Sales Will Outpace All Other Meds, Thanks to High Prices

Orphan Drug Sales Will Outpace All Other Meds, Thanks to High Prices | Pharmaguy's Insights Into Drug Industry News | Scoop.it

As payers continue to foot the bill for orphan drugs, sales of these rare disease treatments are forecast to grow 11 percent over the next five years — to $209 billion. And this is more than twice the expected increase in sales of all other prescription medicines, according to a new analysis.

 

By 2022, orphan drugs are predicted to account for more than 21 percent of worldwide brand-name prescription drug sales, up from 6 percent in 2000. Last year, sales of orphan drugs, which are used to treat rare diseases for patient populations numbering less than 200,000, climbed more than 12 percent, to $114 billion. By comparison, sales of other brand-name drugs rose 2.4 percent, to $578 billion.

 

The figures indicate that orphan drugs remain a lucrative revenue stream for the pharmaceutical industry, especially the largest drug makers. Based on worldwide orphan drug sales, seven of the 10 largest players are global companies, including Novartis, Roche, and AbbVie, not upstart biotechs, according to the analysis, which was conducted by EvaluatePharma, a market research firm.

 

Here are a few other nuggets from the report:

 

  • In the US, the biggest-selling orphan drug last year per patient was Soliris, an Alexion Pharmaceuticals medication used to treat two rare diseases, at more than $400,000 per patient.
  • Also in the US, the biggest-selling orphan drug, based on total revenue, was Revlimid, a Celgene treatment for cancer, at $4.4 billion.
  • And among companies selling orphan drugs for diseases other than cancer, Shire has the largest sales volume on a global basis with $5.3 billion.
Pharma Guy's insight:

Further Reading:

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Orphan Drugs Are “Wicked Hot” & Profitable for Pharma to Boot!

Orphan Drugs Are “Wicked Hot” & Profitable for Pharma to Boot! | Pharmaguy's Insights Into Drug Industry News | Scoop.it

More than 30 years ago, Congress overwhelmingly passed a landmark health bill aimed at motivating pharmaceutical companies to develop new drugs for people whose rare diseases had been ignored.

 

By the drugmakers' calculations, the markets for such diseases weren't big enough to bother with.

 

But lucrative financial incentives created by the Orphan Drug Act signed into law by President Reagan in 1983 succeeded far beyond anyone's expectations. More than 200 companies have brought almost 450 so-called orphan drugs to market since the law took effect.

 

Yet a Kaiser Health News investigation shows that the system intended to help desperate patients is being manipulated by drugmakers to maximize profits and to protect niche markets for medicines already being taken by millions. The companies aren't breaking the law but they are using the Orphan Drug Act to their advantage in ways that its architects say they didn't foresee or intend. Today, many orphan medicines, originally developed to treat diseases affecting fewer than 200,000 people, come with astronomical price tags.

 

"Orphans are wicked hot," said Dr. Tim Coté, a former FDA official who now runs a consulting firm that advises drugmakers on orphan drugs.

 

And many drugs that now have orphan status aren't entirely new. More than 70 were drugs first approved by the Food and Drug Administration for mass market use. These medicines, some with familiar brand names, were later approved as orphans. In each case, their manufacturers received millions of dollars in government incentives plus seven years of exclusive rights to treat that rare disease, or a monopoly.

 

Drugmakers of popular mass market drugs later sought and received orphan status for the cholesterol blockbuster Crestor (read “#Pharma Welfare: ‘Orphan’ Blockbuster Drugs on Rise - Including Crestor!; http://sco.lt/4pauhN), Abilify for psychiatric conditions, cancer drug Herceptin, and rheumatoid arthritis drug Humira, the best-selling medicine in the world.

 

More than 80 other orphans won FDA approval for more than one rare disease, and in some cases, multiple rare diseases. For each additional approval, the drugmaker qualified for a fresh batch of incentives. Botox, stocked in most dermatologists' offices, started out as a drug to treat painful muscle spasms of the eye and now has three orphan drug approvals. It's also approved as a mass market drug to treat a variety of ailments, including chronic migraines and wrinkles.

 

Altogether, KHN's investigation found that about a third of orphan approvals by the FDA since the program began have been either for repurposed mass market drugs or drugs that received multiple orphan approvals.

 

"What we are seeing is a system that was created with good intent being hijacked," said Bernard Munos, a former corporate strategy adviser at drug giant Eli Lilly and Co. who reviewed the KHN analysis of several FDA drug databases. It's "quite remarkable that it has gone on for so long."

 

And the proportion of new drugs approved as orphans has ballooned. In 2015, 21 orphan drugs were approved, accounting for 47 percent of all new medicines, up from just 29 percent in 2010; in 2016, nine more orphans won approval, 40 percent of the total.

 

Orphan drugs now account for seven of the 10 top-selling drugs of any kind, ranked by annual sales, according to EvaluatePharma.

Pharma Guy's insight:

Further Reading:

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Orphan Drug Sales Offer a Glimpse of “Sheer Greed”

Orphan Drug Sales Offer a Glimpse of “Sheer Greed” | Pharmaguy's Insights Into Drug Industry News | Scoop.it

Rising concerns about spending on prescription drugs that treat rare diseases are overblown, according to an analysis published Wednesday in the journal Health Affairs.

 

"We wanted to focus on the true impact of orphan drugs," said Victoria Divino, a senior consultant at IMS Health and an author of the study. Researchers at IMS Health, a health care analytics firm, and drugmaker Celgene Corp. looked at U.S. pharmaceutical spending from 2007 to 2013 on more than 300 drugs that had orphan approval under the 1983 Orphan Drug Act.

 

The Orphan Drug Act has long been considered a success for encouraging the production of hundreds of drugs for rare diseases. But the law's very success has raised economic concerns as the number of high-priced drugs for rare diseases has grown.

 

The review found that orphan drug spending in the United States totaled $15 billion in 2007 and $30 billion in 2013, an increase from 4.8 percent of total pharmaceutical spending to 8.9 percent. The current study projects orphan drug spending will remain fairly stable as a proportion of total drug spending. That stands in contrast to other published reports that estimate orphan drugs will account for 20 percent of worldwide spending on drugs (other than generics) by 2020 (read, for example, “Orphan Drug Sales: An Enduring Prospect for #Pharma Profits”; http://sco.lt/5oCT1V and "Orphan Drugs Now Where the Money Is"; http://bit.ly/pgdaily111113-3A).

 

The rise in orphan drug spending since 2007, Divino said, was caused by an increase in the number of orphan drugs approved by the Food and Drug Administration. The number of orphan drug approvals increased from 16 in 2007 to 33 in 2013, the analysis' time period.

 

The paper, published in the American Journal of Clinical Oncology, noted that seven of the top-10 selling drugs worldwide in 2014 had received orphan status. Those listed included popular drug such as Crestor, a cholesterol fighter, and Humira, for rheumatoid arthritis and Crohn's disease (read “#Pharma Welfare: "Orphan" Blockbuster Drugs on Rise - Including Crestor!; http://sco.lt/4pauhN).

 

"There are a lot of factors I think that have come up in pricing in general, but what people are getting a glimpse of is the sheer greed," said Dr. Martin Makary, an author of the 2015 paper and professor of health policy at Johns Hopkins School of Public Health.

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How Endo Pharmaceuticals Got Caught Promoting an Orphan Drug "Off Label"

How Endo Pharmaceuticals Got Caught Promoting an Orphan Drug "Off Label" | Pharmaguy's Insights Into Drug Industry News | Scoop.it

The Truth In Media Project has released the last segment of of its newest series Truth In Media: Big Pharma, The FDA & Health Propaganda. Part 4, titled How Big Pharma Uses Off-Label Drugs, illustrates how pharmaceutical companies have made massive profits in spite of being ordered to pay enormous settlements related to harmful side effects of certain drugs.

Truth In Media’s Ben Swann first discussed the case of Peggy Ryan, a pharmaceutical sales representative for Endo Pharmaceuticals, who spent a decade undercover gathering information and ultimately blowing the whistle on corruption within the company.

Ryan, described as a “rising star” at Endo, said that she had been pressured and directed to push a product called Lidoderm, which was initially created to treat a specific condition called post-herpetic neuralgia. Since Lidoderm was approved to be prescribed only for this condition, the drug was classified as an “orphan drug,” which qualifies a drug to be subsidized by taxpayer dollars in its approval and production to offset low profits related to producing a product that is prescribed to a limited number of patients.

Swann pointed out that the FDA does not monitor orphan drugs for side effects once approved.

Ryan said she was instructed to sell the drug “off-label,” meaning to sell the drug to be used conditions outside of its intended use. Lidoderm ended up being prescribed for a plethora of unapproved health issues including back pain and carpal tunnel syndrome. Many of these prescriptions were covered by Medicare and Medicaid.

“She had access to a lot of information, and she provided that to the government. But then they asked her to wear a wire and obtain audiotapes of the people that she reported to telling her and other employees to essentially to break the law,” Judy Hoyer, a whistleblower attorney whose firm represented Ryan, told Swann.

Endo was ordered to pay $193 million in penalties in a settlement; $21 million was allocated to settle criminal charges, and $172 million was paid out under the False Claims Act. While $193 million is certainly a large sum of money to be paid, Endo made at least $1 billion selling the drug off-label; the penalty paled in comparison to the profits made.

In a separate case that also involved massive drug sale profits in light of little government oversight, highly-cited researcher, doctor and Emory University Professor Doug Bremner discussed the acne drug Accutane with Swann. Bremner, who has spent a great deal of time researching PTSD and depression and the brain, served as an expert witness in litigation proceedings associating depression and suicide with Accutane.

Pharma Guy's insight:

Also read: “How Big Pharma Courts Physicians & Makes Them Look Like They Have a Lot of Money!”; http://sco.lt/6w3HWL

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Drug Spending Growth Surges Again in 2015

Drug Spending Growth Surges Again in 2015 | Pharmaguy's Insights Into Drug Industry News | Scoop.it

Total spending on medicines in the U.S. reached $310 billion in 2015 on an estimated net price basis, up 8.5 percent from the previous year, according to a new report issued today by the IMS Institute for Healthcare Informatics.

Invoice spending is based on IMS Health reported values from wholesaler transactions measured at trade/invoice prices and exclude off-invoice discounts and rebates that reduce net revenue received by manufacturers. Net spending reflects company recognized revenue after off-invoice discounts, rebates and price concessions are applied.


According to IMS, the increase was driven primarily by a wave of "innovative" new medicines. Take a closer look at the data here.

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ThePlanetaryArchives - BlackHorseMedia - San Francisco's curator insight, April 16, 2016 10:53 AM
"Innovative" drugs? There are no drugs that actually cure any disease, and America is sicker and fatter than ever. One day these people will be revealed for the snake-oil salesmen that they are. Except snake-oil actually works.....
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Orphan Drug Sales: An Enduring Prospect for #Pharma Profits

Orphan Drug Sales: An Enduring Prospect for #Pharma Profits | Pharmaguy's Insights Into Drug Industry News | Scoop.it
Check out an infographic to learn more about orphan drugs, rare diseases and the top-selling drugs in the category.
Pharma Guy's insight:

You may also want to read this: "Orphan Drugs Now Where the Money Is"; http://bit.ly/pgdaily111113-3A 

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#Pharma Welfare: "Orphan" Blockbuster Drugs on Rise - Including Crestor!

#Pharma Welfare: "Orphan" Blockbuster Drugs on Rise - Including Crestor! | Pharmaguy's Insights Into Drug Industry News | Scoop.it
An FDA rule created to spur drug companies to develop treatments for rare diseases is being used far beyond its original scope, critics say.

 

[Would you believe that Crestor is an "orphan" drug? Also read: "How AstraZeneca Hopes To Use 'Orphan Drug' Designation To Extend Patent Life Crestor"; http://sco.lt/77Pyfx]

 

A new study from Johns Hopkins University School of Medicine questions whether some of the biggest drug companies and their blockbuster medications are taking advantage of a decades-old act meant to increase research, development and drug approval for people suffering from rare diseases.

 

The new study, published in the American Journal of Clinical Oncology,argues that while the ODA has fostered drug development for patients with rare cancers and other diseases, current data suggest that companies are "gaming the system to use the law for mainstream drugs." 

 

The authors found what they deem "a pattern of pharmaceutical companies submitting drugs to the Food and Drug Administration (FDA) as orphan drugs but once approved, the drugs are used broadly off-label with the lucrative orphan drug protections and exclusivity benefits."

 

The study contends that some big drug companies submit a drug for FDA approval with a narrow enough indication that would qualify it for orphan drug benefits. After FDA approval, however, the drug can be used more broadly.

 

For example, Rituxan (rituximab), which is made by Roche and was initially FDA approved for use in the treatment of follicular non-Hodgkin's lymphoma, is the No. 1 selling medication approved as an orphan drug. 

 

"It is currently used to treat a wide variety of conditions, ranks as the 12th all-time bestselling medication in the United States, and generated over $3.7 billion in U.S. sales in 2014," the report states. 

 

The researchers' concern is not only the "corporate welfare" that is being afforded to blockbuster drugs and highly profitable drug companies, but that "patients with rare cancers and other diseases may suffer due to dilution of the tax incentives and other benefits" offered by the rule to spur the development of niche drugs.

Pharma Guy's insight:

As I said back in 2011 when I channeled Will Sutton's ghost: Orphan Drugs Are Now "Where the Money Is." I said: Until a few years ago, if you asked a pharmaceutical company executive why his or her company developed and marketed an "orphan drug" -- ie, a drug for a disorder affecting fewer than 200,000 people in the U.S. -- you would likely have gotten a response such as "because there is an unmet medical need" or something similar. Today, however, orphan drugs also have the potential to turn into blockbusters. Read more here: http://bit.ly/pgdaily111113-3A 

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Otsuka to FDA: No Thanks... Orphan Status for Abilify is More Profitable. Generics Can Pound Sand!

Otsuka to FDA: No Thanks... Orphan Status for Abilify is More Profitable. Generics Can Pound Sand! | Pharmaguy's Insights Into Drug Industry News | Scoop.it

In a closely watched gambit, a drug maker has taken the highly unusual step of going to court to prevent the FDA from widening the market for its best-selling pill. And the move may not only embolden other companies to follow suit, but also threatens to raise health-care costs for consumers.


The dispute turns on the complexities of regulatory law, but has a familiar refrain—a brand-name drug maker is trying to thwart generic competition and protect a product that rings lots of registers. In this case, Otsuka Pharmaceutical Co. hopes to defend the market for its Abilify antipsychotic, which generated $4.9 billion in U.S. sales last year, but faces the loss of a crucial patent next week.


Here’s the back story: Last December, the FDA approved Abilify for treating children with Tourette syndrome, a neurological disorder that causes tics.


Since Abilify had a so-called orphan designation, which refers to a drug used to treat a rare malady like Tourette’s, Otsuka won another seven years of exclusive marketing rights—through late 2021—before low-cost generics could appear.


But in February, the FDA surprised Otsuka by approving Abilify to treat adults with Tourette syndrome. This widened the market and kicked off the legal battle, because Otsuka contends FDA law would trigger a labeling change that could usher in generics.


In a lawsuit, Otsuka contends the FDA isn’t allowed to approve an indication for which a drug maker didn’t apply and charged the agency was actually attempting to “clear a blocked path” for generics. An FDA spokeswoman wouldn’t comment.


One Wall Street analyst suggests Otsuka may have overreached. “Under Otsuka’s interpretation, having one orphan indication protects the entire [Abilify] franchise from generic entry,” wrote Sanford Bernstein analyst Ronny Gal in an investor note. “Otsuka seized on a putative loophole as a way of protecting its blockbuster franchise.”


He maintains, however, that “Congress did not intend to allow [a] single pediatric orphan indication to provide patent protection for [a drug] beyond the targeted orphan use, and to argue otherwise is shamelessly audacious.” An Otsuka spokesman declined to comment.

Pharma Guy's insight:


As one person familiar with the matter notes, “if Otsuka prevails, you’ll see other [drug makers] look to try something similar.” This just proves how profitable orphan drug status is. For more on that read: Orphan Drugs Now "Where the Money Is," Says Willie Sutton's Ghost- http://bit.ly/pgdaily111113-3A

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37% of New Drugs Approved by FDA in 2014 were for Treatment of Rare Diseases

37% of New Drugs Approved by FDA in 2014 were for Treatment of Rare Diseases | Pharmaguy's Insights Into Drug Industry News | Scoop.it

New data from the FDA (here) shows that 2014 was a banner year for orphan drugs, which are drugs that treat "rare" diseases/disorders affecting fewer than 200,000 people in the U.S.


Orphan drug approvals represent 37% of all new drug approvals in 2014. Today, orphan drugs have the potential to turn into blockbusters with annual sales over $1 Billion.


Read more here.


Pharma Guy's insight:


When a dumb reporter asked "Willie" Sutton, the famous bank robber, why he robbed banks, he is reported to have said "Because that's where the money is." Duh!

Until a few years ago, if you asked a pharmaceutical company executive why his or her company developed and marketed an "orphan drug" -- ie, a drug for a disorder affecting fewer than 200,000 people in the U.S. -- you would likely have gotten a response such as "because there is an unmet medical need" or something similar.

Today, however, orphan drugs also have the potential to turn into blockbusters; ie, be where pharma's money is at.


Read, for example, "New Big Pharma Economies of Scale: Less Patients Needed to Reach Blockbuster Sales")

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