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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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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Thank You Martin Shkreli for Goosing FDA to Act More Expeditiously to Approve Generics!

Thank You Martin Shkreli for Goosing FDA to Act More Expeditiously to Approve Generics! | Pharmaguy's Insights Into Drug Industry News | Scoop.it

In a significant move, the Food and Drug Administration late last week made a policy change that may prevent companies from pulling a Martin Shkreli.

The agency plans to expedite reviews of applications for generic drugs where only one treatment is currently sold. The shift was prompted by public outrage that erupted last fall when Turing Pharmaceuticals, which Shkreli ran before he was indicted for securities fraud, bought a life-saving drug called Daraprim and promptly jacked up the price by 5,000 percent, from $13.50 a tablet to $750.

“We identified a gap and were able to identify a path forward,” an FDA spokeswoman wrote us. “The change being made (allows the agency) to capture circumstances when the only approved product on the market is a generic drug.”

Even though Turing does not hold a patent on the medicine, used to treat a rare parasitic infection known as toxoplasmosis, the company was able to increase the price as it did because there was no generic competition. The drug maker runs a closed distribution system, and as a result, Turing has a monopoly on Daraprim. Even if a generic drug maker wanted to enter what was suddenly a more lucrative market, any company would encounter a delay winning FDA approval since the agency faces a huge backlog of applications.

“This is a big deal,” said John Rother, who heads the National Coalition on Healthcare, a collection of insurers, employers, and unions, among others, that have objected to rising prices. “This should provide a faster way to inject competition in the marketplace, so that the price gougers can’t get away with what they’re doing.”

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FDA Report Touts Record Number of Novel Drugs Approved in 2015

FDA Report Touts Record Number of Novel Drugs Approved in 2015 | Pharmaguy's Insights Into Drug Industry News | Scoop.it

With life in the slow lane for a bit, it was a good time to catch up on reading. One of those was a recently issued FDA report on the novel new drugs that were approved in 2015. It is a very thorough and well organized report and while it is worth summarizing a few things from it here, you are encouraged to download the original here. It was a banner year in approvals on a number of accounts.


Moreover FDA met the PDUFA date for approval 96 percent of the time and about two-thirds of the approvals were made here in the United States before being approved in any other country.


  • 45 New Novel Drugs in 2015
  • 16 (36 percent) were First-in-Class
  • 21 (45 percent) had Orphan status
  • 14 (31 percent) had Fast Track status
  • 10 (22 percent) had Breathrough status
  • 24 (53 percent) had Priority Review
  • 6 (13 percent) were under Accelerated Approval


Need the primer on the difference between all those expedited approval terms?  Click here.


Pharma Guy's insight:

But FDA's Office of prescription Drug promotion (OPDP) sent out only 9 advertising enforcement letters (2 Warning Letters, 7 Untitled Letters) in 2015, which beats 2014's record low of 10 letters. For more on that, read "2015 Was a Golden Year for Pharma"; http://bit.ly/pmn150101p 

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GAO Report: FDA Expedites Drug Approvals, But Its Postapproval Oversight Stinks!

GAO Report: FDA Expedites Drug Approvals, But Its Postapproval Oversight Stinks! | Pharmaguy's Insights Into Drug Industry News | Scoop.it

FDA has supported efforts to shorten development and streamline the agency’s review of drug applications through expedited pathways. However, we found problems with the agency’s efforts to oversee and track potential safety issues and postmarket studies once those drugs are on the market. While internal control standards for the federal government specify that information should be recorded in a form and within a time frame that enables staff to carry out their responsibilities and that relevant, reliable, and timely information should be available for external reporting purposes, FDA’s data on postmarket safety issues and studies were found to be incomplete, outdated, to contain inaccuracies, and to be stored in a manner that made routine, systematic analysis difficult. 


FDA lacks reliable, readily accessible data on tracked safety issues and postmarket studies needed to meet certain postmarket safety reporting responsibilities and to conduct systematic oversight. Tracked safety issues are potential safety issues that FDA determines are significant and that it tracks using an internal database. Internal control standards for federal agencies specify that information should be recorded in a form and within a time frame that enables staff to carry out their responsibilities and that relevant, reliable, and timely information should be available for external reporting purposes. However, evaluations conducted by CDER of data in its database revealed problems with the completeness, timeliness, and accuracy of the data. These problems, as well as problems with the way data are recorded that impair their accessibility, have prevented FDA from publishing statutorily required reports on certain potential safety issues and postmarket studies in a timely manner, and have restricted the agency's ability to perform systematic oversight of postmarket drug safety. Although FDA has taken some steps to address the problems with its data, the agency lacks plans that comprehensively outline its efforts and establish related goals and time frames. Additionally, FDA does not have plans to use these data to inform its oversight of its expedited programs, such as determining if drugs that used an expedited program were subsequently associated with tracked safety issues at rates or of types that differed from drugs that used FDA's standard process.

Pharma Guy's insight:

I reported on this problem back in 2013:


Many drug approvals were fast-tracked on the promise that pharma companies would meet study deadlines and supply the FDA with data about any adverse events discovered via observational postmarketing studies after market approval.

So, how well has the FDA held pharma companies feet to the fire regarding postmarketing commitments under FDAAA jurisdiction?

Not very well at all, according a Research Letter published in the July 10, 2013, issue of JAMA. The authors of the study found that NONE (zero) of the 865 studies under FDAAA jurisdiction from 2008 through 2011 have been completed. Of the 387 studies mandated in 2011, 271 (70%) have not even begun.


For more on that, read "FDA is Lax in Enforcing Law Regarding Prescription Drug Postmarketing Studies"; http://bit.ly/1PoqAsY 

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Pharma Guy's curator insight, March 28, 2016 2:41 PM

I reported on this problem back in 2013:

 

Many drug approvals were fast-tracked on the promise that pharma companies would meet study deadlines and supply the FDA with data about any adverse events discovered via observational postmarketing studies after market approval.

So, how well has the FDA held pharma companies feet to the fire regarding postmarketing commitments under FDAAA jurisdiction?

Not very well at all, according a Research Letter published in the July 10, 2013, issue of JAMA. The authors of the study found that NONE (zero) of the 865 studies under FDAAA jurisdiction from 2008 through 2011 have been completed. Of the 387 studies mandated in 2011, 271 (70%) have not even begun.

 

For more on that, read "FDA is Lax in Enforcing Law Regarding Prescription Drug Postmarketing Studies"; http://bit.ly/1PoqAsY 

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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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9, 10 Reasons Why FDA Is Approving Every Drug It Sees

9, 10 Reasons Why FDA Is Approving Every Drug It Sees | Pharmaguy's Insights Into Drug Industry News | Scoop.it

Last week I [Matthew Herper] wrote a post about new data from BioMedTracker that show that the Food and Drug Administration, which once approved as few as 40% of new drugs submitted by industry, has been on a green-light-almost-everything jag, approving 89% of drug applications.


What’s more, a closer look showed an even higher approval rate. This year so far, 96% of new molecular entities– industry jargon for drugs that have never been approved for any use before – that have been submitted to the FDA have reached the market. For anyone who was watching the FDA a decade ago, that’s just shocking. Good or bad, it’s a radical change.


It’s certainly true that there are a lot of factors that explain why the FDA approval rate is suddenly so high, and I only gave them a few paragraphs in the last piece. So here I’ll list nine (see pharmaguy's #10 in the comment section):


  1. The approval rate is much lower, because only 12% of drugs that enter clinical trials reach the market
  2. Drug companies are better at research, and they are simply producing better drugs
  3. Drug companies are picking areas where the chances of approval are higher
  4. The FDA is doing a better job communicating with companies ["and vice versa," says pharmaguy]
  5. The FDA has more power to restrict the use of an approved drug than it used to
  6. The FDA is taking a risk by taking strong stands against drug approvals right now
  7. The FDA is without a permanent commissioner
  8. It’s just random chance
  9. In the current political environment, the agency is approving drugs it shouldn’t


Pharma Guy's insight:

#10: It's no coincidence that this is happening as FDA sets its sights on PDUFA VI reauthorization: http://sco.lt/5nZ3p3

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FDA Sets Its Sights on PDUFA VI Reauthorization

FDA Sets Its Sights on PDUFA VI Reauthorization | Pharmaguy's Insights Into Drug Industry News | Scoop.it

The Prescription Drug User Fee Act (PDUFA) authorizes FDA to collect fees from pharmaceutical companies to help fund the agency’s drug review work. PDUFA’s intent is to provide additional funding for FDA to hire staff, improve systems, and establish a better-managed review process that enables us to do more timely reviews of human drug applications.


As part of FDA’s agreement with industry during each reauthorization of the Act, the agency agrees to certain performance goals to enhance the process of drug review. The goals, now 30 in total, apply to many review processes, including the review of original new drug applications, resubmissions, and supplemental applications. Since the first user fee law was passed in 1992, PDUFA has been reauthorized four times. The current legislation, PDUFA V, is set to expire in September 2017.


On July 15, 2015, FDA gathered stakeholder perspectives during a meeting on what features the agency should propose in the reauthorization of PDUFA for fiscal years 2018 – 2022. Attendees included patient advocates, consumer advocates, representatives of professional health care associations, biopharmaceutical industry representatives, academic researchers, policy analysts, and others. FDA received mostly positive feedback on our progress under PDUFA V and helpful input about the future for PDUFA VI.


Highlights of progress noted during this meeting include:


  • FDA continues to meet or exceed most review goals.
  • The program is experiencing high rates of approvals for novel products during their first submission. This includes a historically high number of approvals for novel products treating rare diseases (17 orphan drug approvals in 2014).
  • There are improved and increased communication functions and practices between FDA and new drug companies, or sponsors. This includes implementation of a structured risk-benefit framework within the review process.
  • The Patient-Focused Drug Development program has been successful in systematically obtaining patient perspectives on certain diseases and related treatments.


Meeting participants also contributed a number of ideas for further enhancements, including:


  • Further efforts to involve the patient perspective in drug development processes;
  • Building on FDA’s Sentinel System for active surveillance of safety issues for medical products, including expanding its use as a source of data; and
  • Enhancing regulatory science initiatives, including the use of patient-reported outcomes and biomarkers.


More detailed information about the meeting is available at PDUFA Meetings, which includes a webcast of the one-day meeting, the agenda, access to the docket for online public comments, and (soon to follow) a complete written transcript of the public meeting’s proceedings.



Pharma Guy's insight:

It's no coincidence that FDA's drug approval rate is at an all-time high: http://sco.lt/5BKg6r 

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Patents Have Expired! Long Live the Patents! Floodgates Open for #Pharma Ad Agencies

Patents Have Expired! Long Live the Patents! Floodgates Open for #Pharma Ad Agencies | Pharmaguy's Insights Into Drug Industry News | Scoop.it
Three years ago, health-care agencies were starting to heal after a billion-dollar drop in annual pharma advertising. Today, they're charting a strong recovery.


Some called it pharmageddon. In 2012, health-care ad agencies were coming off a billion-dollar drop in annual drug advertising over five years with little hope in sight: A host of blockbuster medications were about to lose patent protection and there were pessimistic projections for the pipeline ahead.


The world's largest agency groups were feeling the pain. Interpublic CEO Michael Roth said during an earnings call then that the health-care sector was marked by "industry-wide softness."


But health care got a different kind of shout-out during IPG's most recent earnings call, when Mr. Roth called it the company's fastest-growing area and cited year-over-year growth of 13%.


Only a couple years after pharmageddon left agencies with the ruins of a stable and lucrative trade, budgets are back as health-care marketing defies earlier predictions and delivers one of the best comeback stories in advertising. The resurgence has been fueled by a combination of factors: shops' efforts to diversify their services in health care and the arrival of valuable new blockbuster drugs -- that demand elaborate digital content marketing to sell them."


Health care is one [area] where we're seeing strength; it was in a lull for a while as patents were expiring," Mr. Roth said on the recent earnings call. "We're seeing new products. And there in particular, I think content is a very important part of what we're doing with respect to the health-care clients."


The PR giant Edelman said its health-related revenue increased more than 20% in the year ending last June, after seeing "moments of leveling out" during pharmageddon."


The pharma industry is roaring back from pharmageddon," said Kym White, global sector chair of Edelman's health business.

Global spending on medicines is forecast to reach nearly $1.3 trillion by 2018, an increase of about 30% over 2013, according to the IMS Institute for Healthcare Informatics.

Pharma Guy's insight:


2014 Was a Good Year for FDA & Pharma

Banging Year for Drug Approvals, Wimpy Year for Enforcement Actions


The U.S. Food and Drug Administration (FDA) ended 2014 with a bang and a whimper.

The Agency approved a total of 41 new molecular entities (NMEs) and biologics (BLAs) in 2014, the greatest number since 1996. That was the bang. FDA, however, also ended the year with a whimper. The Agency sent out only a meager 10 advertising enforcement letters in 2014, the fewest ever. 

There may be a single explanation for both phenomena: the Prescription Drug User Fee Act (PDUFA) payments to FDA from pharma. These payments now account for more than 65% of FDA's budget for the approval and regulation of drugs. 

Topics (partial list):
  • Orphan Drugs Are "Where the Money Is"
  • Banging Drug Prices
  • Wimpy Enforcement
  • Busy, Busy FDA
  • PDUFA Fees vs. Enforcement

Access this article here. NOTE: A free subscription may be required.


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U.S. Drug Approvals Hit 18-year High

U.S. Drug Approvals Hit 18-year High | Pharmaguy's Insights Into Drug Industry News | Scoop.it

U.S. drug approvals in 2014 hit their highest level in 18 years and recommendations in Europe also came at a rapid rate, driven by expensive new treatments for cancer and rare diseases.


After suffering a wave of patent losses on blockbuster products, which peaked two years ago, drugmakers are recovering their ability to bring new medicines to market and productivity is improving.


The U.S. Food and Drug Administration's Center for Drug Evaluation and Research approved 41 novel medicines in 2014, 14 more than a year earlier, according to its website. That tally is second only to the all-time high of 53 approvals reached in 1996.


The European Medicines Agency, which includes generic drugs in its list, recommended 82 new medicines last year, up from 79 in 2013 and 57 in 2012.

Pharma Guy's insight:


Treatments for ear infections, insomnia and fungal infections of the toe were among less heralded advances for medical science in 2014.

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FDA approves cancer drugs without proof they’re extending lives

FDA approves cancer drugs without proof they’re extending lives | Pharmaguy's Insights Into Drug Industry News | Scoop.it

For decades, researchers have focused ondeveloping new cancer drugs that save lives or improve the quality of life. But when the U.S. Food and Drug Administration allowed Inlyta, a $10,000 a month drug, on the market in 2012, there was no proof that it did either.


Inlyta is not an exception to the rule.


A Milwaukee Journal Sentinel/MedPage Today analysis of 54 new cancer drugs found that over the last decade the FDA allowed 74% of them on the market without proof that they extended life. Seldom was there proof of improved quality of life, either.


Nor has the FDA demanded companies provide such evidence.

Instead, the agency approved the drugs based on surrogate measures, such as a tumor shrinking, rather than the gold standard and most reliable measure of cancer research, patients actually surviving longer. The problem is cancer is complicated — a tumor might stop growing or shrink in one spot, then reappear somewhere else, or even in multiple places.


Inlyta, manufactured by Pfizer, was allowed on the market based on a commonly used surrogate known as progression-free survival, which means patients survived longer before doctors detected a tumor worsening.


Patients given Inlyta saw no noticeable progression of their disease for an average of 6.7 months, two months longer than those who got a control drug. But patients who got Inlyta did not live any longer.

Before Inlyta was approved, an FDA reviewer noted it would be the seventh drug for advanced kidney cancer approved by the FDA since 2005. Only one of them, a drug known as Torisel, had actually proven to help people live longer.


Prompted by politicians, pharmaceutical companies and advocacy groups seeking to speed up drug approvals, the FDA has allowed shortcuts to make it easier for companies to get products on the market.


A big part of that has been the use of surrogate measures.


For instance, diabetes drugs usually are approved based on lowering blood-sugar levels, rather than clinical benefits such as fewer amputations and heart attacks.


Heart drugs may be approved based on tests measuring various fats in the blood rather than fewer heart attacks, strokes or cardiovascular deaths.


Surrogate measures, which allow for shorter, smaller and cheaper clinical trials, have opened the gates to a steady stream of costly drugs of dubious value.


"The whole paradigm is broken, and it is an unmitigated disaster," said Peter F. Thall, a biostatistician at MD Anderson Cancer Center in Houston who designs clinical trials for cancer research.

Pharma Guy's insight:


Remember this comment by Marijn Dekkers, "outspoken" head of Bayer Pharmaceuticals, in which he not only disses patients but claims to have cured cancer?


"If you have cancer, you get a pharmaceutical product, and your cancer goes away. You're quick to call the doctor and [say] the staff at the hospital was great. But the pill that did it gets forgotten. We struggle with getting society to put value on what we do, and it becomes particularly important as we get under more pressure to develop the next pill."


For more on that, read Bayer's CEO Accuses Patients of Being Ungrateful B*stards! We Cured Cancer, Dammit!

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As Drug Approvals Dive in 2016, R&D ROI Retracts

As Drug Approvals Dive in 2016, R&D ROI Retracts | Pharmaguy's Insights Into Drug Industry News | Scoop.it

The global pharmaceuticals industry is set to win the lowest annual number of new drug approvals this year since 2010 (read “New Drugs Approved by FDA in 2016 is Half the Number Approved by This Time in 2015”; http://sco.lt/9CtktV) and a new report on Tuesday suggests drugmakers' returns on research investment are deteriorating (read “Consultants Advise Pharma to Act More Like Biotech to Revive ROI for New Drugs”; http://sco.lt/8qLTCT).

 

Only 19 new drugs have been approved in the key U.S. market so far in 2016 and, with less than three weeks to go, it is clear the full-year tally will be well down on 2015 and 2014's bumper haul of 45 and 41 new products respectively.

 

At the same time the profitability of drug research is being squeezed by steadily rising costs and increasing political pressure over the high prices of many modern medicines.

 

As a result projected returns on investment in research and development (R&D) for the top 12 pharmaceutical companies have fallen to just 3.7 percent this year from a high of 10.1 percent in 2010, according to consultancy Deloitte.

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Promoting Therapeutic Innovation: What Do We Do About Drug-Device Combinations?

Promoting Therapeutic Innovation: What Do We Do About Drug-Device Combinations? | Pharmaguy's Insights Into Drug Industry News | Scoop.it

Legislation proposed in the Senate, the Combination Product Regulatory Fairness Act, which is intended to promote innovation among products that contain both a drug and device (http://1.usa.gov/1KFLZg9).

 

The Combination Product Regulatory Fairness Act—introduced in July 2015—would broaden the ability for combination products to be classified as devices. The bill requires that jurisdiction as drugs or biologics not be assigned “solely because” of chemical action in the human body and orders the FDA to provide scientific support to the sponsor whenever the agency does not classify a combination product as a device.

 

In addition, the bill requires the FDA and sponsor to establish early in development a combination product review plan, an inflexible agreement on all aspects of clinical studies needed to establish its effectiveness and safety, including any postapproval studies. For example, if the FDA and sponsor agree on a clinical trial design that would test the product against placebo based on its effect on an intermediate outcome intended to predict clinical benefit or harm—a surrogate end point—the FDA would be highly restricted in its ability to later seek additional clinical data to inform its decision making.

 

However, the bill’s aims also put it at odds with certain important public health interests. The classification changes it proposes would inevitably increase the number of combination products reviewed under the more lenient 510(k) pathway, even though in 2014, almost two-thirds of combination products submitted for approval were already classified as devices (http://1.usa.gov/1KbpOUa). The 510(k) process provides important flexibility in making small alterations to moderate risk devices, but it has been criticized for being a highly limited premarket screen for a product’s safety and effectiveness and for permitting products to evolve substantially over time without adequate oversight, based on numerous seemingly innocuous updates (http://bit.ly/1SjUH9W). In addition, unlike for drugs and biologics, the FDA does not have authority to require postmarket studies as a condition of clearing a 510(k) device.

 

The bill’s restrictive combination product review plan compounds these potential negative patient safety implications. This proposed attempt to restrict FDA flexibility is reminiscent of a similar provision in the 21st Century Cures bill that limits the FDA’s ability to alter study plans for drugs in the accelerated approval pathway.

 

Balancing faster patient access to innovative agents with sufficient proof of the therapeutic product’s clinical benefit continues to be a difficult and dynamic process, but the FDA should not have to forfeit its current authority and flexibility in overseeing the development of therapeutic products before they reach patients. And patients and the medical community gain little from forcing more combination products to be classified as devices so that they can be sped to market with limited evidence of their safety and effectiveness.

Pharma Guy's insight:

Related articles: "99% of Medical Devices Approved by FDA with Shockingly Little or No New Research"; http://sco.lt/7W7p0D and "NYT Editorial: F.D.A. Won't Be Fixed by 21st Century Cures Bill"; http://sco.lt/6PLcGX and "Anecdotal Evidence Good Enough to Approve Medical Devices According to Lawmakers"; http://sco.lt/58AwLp 

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Pharma Guy's curator insight, March 31, 2016 6:11 AM

Related articles: "99% of Medical Devices Approved by FDA with Shockingly Little or No New Research"; http://sco.lt/7W7p0D and "NYT Editorial: F.D.A. Won't Be Fixed by 21st Century Cures Bill"; http://sco.lt/6PLcGX and "Anecdotal Evidence Good Enough to Approve Medical Devices According to Lawmakers"; http://sco.lt/58AwLp 

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FDA Defends Expedited Oncology Drug Approvals, Neglects GAO Criticism

FDA Defends Expedited Oncology Drug Approvals, Neglects GAO Criticism | Pharmaguy's Insights Into Drug Industry News | Scoop.it

Richard Pazdur, M.D., Director of the Office of Hematology and Oncology Products, highlights some of his office’s 2015 approvals and discusses a few of the expedited review programs that are used by the office.


In 2015, the Office of Hematology and Oncology Products (OHOP) approved 16 new molecular entities (NMEs). The most notable were drug approvals in disease areas such as non-small-cell lung cancer, colorectal cancer, breast cancer, melanoma, renal cancer, and diseases that are particularly difficult to treat like pancreatic cancer.


FDA reviews new drug applications according to timeframes established by the Prescription Drug User Fee Act (PDUFA). There are also programs in place to expedite the drug development and review timeline, and many of the innovative therapies that were approved by OHOP this past year received an expedited designation. Generally, these designations are given to therapies that we consider to be better than what is currently on the market or that fulfill an unmet medical need. The accelerated approval, priority review, and breakthrough therapy programs are frequently used with new oncology drugs, and often a single drug receives multiple designations.

Pharma Guy's insight:

Dr.. Pazdur notes that "Following an accelerated approval, companies conduct additional confirmatory clinical trials with the drug to further examine its clinical benefit."


He does not mention the GAO report that found "FDA’s data on postmarket safety issues and studies were found to be incomplete, outdated, to contain inaccuracies, and to be stored in a manner that made routine, systematic analysis difficult.": http://sco.lt/898BUn  

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Drug Approvals at 19-year High, But...

Drug Approvals at 19-year High, But... | Pharmaguy's Insights Into Drug Industry News | Scoop.it

2015 was a good year for innovation in medicine with the U.S. Food and Drug Administration approving 45 novel drugs, four more than in 2014 and the most since the all-time record of 53 set in 1996.


Across the Atlantic, the European Medicines Agency recommended 93 new products, including generics, up from 82 in 2014.


But despite the rosy statistics and the prospect for further progress in 2016, the pharmaceuticals industry faces challenges, with increased political focus on drug pricing having punctured both biotech and specialty pharma valuations in recent months.


The prospect of Hillary Clinton becoming U.S. president could further undermine confidence in the sector's profitability in 2016, given her pledge to rein in drug costs. But any changes in the U.S. pricing model are likely to be gradual, according to Bernstein analyst Tim Anderson.


Big pharmaceutical companies, meanwhile, are still struggling to get a decent return on the billions of dollars spent annually on research and development, since many new medicines are forecast to yield relatively modest sales.


Moreover, securing a strong launch for new drugs, which must compete with a growing roster of cheap generics, is often an uphill battle as healthcare providers push back against the high prices being charged.

Pharma Guy's insight:

Meanwhile, the number of FDA enforcement letters sent in 2015 is the lowest EVER! For more on that, see http://bit.ly/1JIY2bU 

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Greg Judd's curator insight, January 4, 2016 9:13 AM

I like John's insight, and I think how this line plays out will be worth following:


Big pharmaceutical companies, meanwhile, are still struggling to get a decent return on the billions of dollars spent annually on research and development, since many new medicines are forecast to yield relatively modest sales.

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FDA Slow to Approve Generic Drugs - Helps Boost Drug Prices

FDA Slow to Approve Generic Drugs - Helps Boost Drug Prices | Pharmaguy's Insights Into Drug Industry News | Scoop.it

With anger soaring over high drug prices, 4,300 cheaper generic drugs are awaiting approval by the Food and Drug Administration.


The FDA acknowledges it has had problems in the past. Chronically underfunded, its generic drug office was long the poor stepchild of the agency. But the agency says it has made significant strides, in part because of measures known as the Generic Drug User Fee Amendments.


How much progress has been made since then depends on how you interpret the data.


The FDA acknowledges that drug applications completed in fiscal 2015 took an average of four years to review, and generic drug companies continue to complain about the lack of progress.



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FDA's Unbridled 96% New Drug Approval Rate: A New Record!

FDA's Unbridled 96% New Drug Approval Rate: A New Record! | Pharmaguy's Insights Into Drug Industry News | Scoop.it

Remember when the FDA rejected drugs?


We just got treated to a whole lot of drama this week as to whether Addyi, a drug to boost women’s libidos, would be approved. But based on the data, that approval was probably a foregone conclusion.


As recently as 2008, companies filing applications to sell never-before-marketed drugs, which are referred to by the FDA as “new molecular entities,” faced rejection 66% of the time. Yet so far this year the FDA has rejected only three uses for new chemical entities, and approved 25, an approval rate of 89%.


In reality, the FDA approval rate is more like 96%. Eliminating BioMedTrackers counting of multiple uses for the same drug means FDA approved 23 drugs and rejected 1, Merck ’s anesthesia antidote, Bridion. Again, that means 19 of 20 new drug applications were approved.

Pharma Guy's insight:

It's no coincidence that this is happening as FDA sets its sights on PDUFA VI reauthorization: http://sco.lt/5nZ3p3

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No #Pharma EVER Made Public Full Text of FDA Rejection Letters

No #Pharma EVER Made Public Full Text of FDA Rejection Letters | Pharmaguy's Insights Into Drug Industry News | Scoop.it

Seven times between August 11, 2008, and June 27, 2013, the Food and Drug Administration declined to approve a new medicine, in part, because patients were more likely to die when taking the drug than in a control group. Yet only one of those companies told investors – and the public – about that concern.


That’s perhaps the most shocking detail from a sweeping FDA analysis, published in the new issue of the British Medical Journal, of what pharmaceutical companies tell the world when the agency rejects a drug. The conclusion: at best, the industry’s communications about rejections are insufficient and misleading. At worst, they sound close to outright lies – and investors faced with an FDA rejection should never believe anything that executives have to say unless those executives make public the full text of the rejection letter – something that, in the time span of this analysis, no company has done.


Companies say the letters should remain confidential because they contain competitive information. But  the reality is that companies are not served by a system where only a limited number of people know why a drug was rejected. One of the best ways to understand what you need to do to get your drug approved is to know where others went wrong.


An example? The same one I used in 2008: The FDA has now rejected Merck’s sugammadex, hailed by many anesthesiologists are a breakthrough antidote to anesthetics, three times. Wouldn’t it be nice to know exactly why, in the FDA’s own words? It’s time to change the FDA regulations so that investors know why drugs get rejected.

Pharma Guy's insight:


Hear! Hear!

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FDA's 2014 Scorecard: Drug Approvals Up, Advertising Enforcement Actions Down!

FDA's 2014 Scorecard: Drug Approvals Up, Advertising Enforcement Actions Down! | Pharmaguy's Insights Into Drug Industry News | Scoop.it

In 2014, the FDA approved 41 new drugs -- the most since 1996 (see here). That's quite a "record" even if 37% of the approvals were made in December alone.

But FDA wasn't as anxious to set any advertising enforcement records in 2014. The Agency sent out only a meager 9 Untitled Letters and 1 Warning Letter last year (see chart on left).

Could the reason for this be that pharmaceutical marketers are getting better at complying with FDA regulations?

Or is the FDA afraid of being sued by the pharmaceutical industry in the wake of the 2012 Caronia decision in the U.S. Second Circuit that found off-label promotion was protected by free speech?

Or perhaps FDA depends more and more on "user fees" paid by the drug industry and does not want to "bite the hand that feeds it"?

I think the latter is the most likely explanation. Read on to learn why.

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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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Software Glitch May Have Lead to Some Drugs Being Approved Based on Incorrect Data!

The reliability of clinical tests used to win approval for some medicines -- particularly generic copies of original drugs -- could be in doubt due to an apparent software glitch that may mean data was calculated incorrectly.


An official at the London-based European Medicines Agency (EMA) told Reuters that the issue, involving Thermo Fisher Scientific's Kinetica package, would be discussed by European regulators at a meeting next week.


Thermo Fisher -- a U.S.-based maker of laboratory equipment and life science research tools with an annual turnover of $17 billion -- said it was looking into the matter, which was first raised by independent experts in a scientific paper.


The problem could mean some medicines have been approved on incorrect data. Others may have been rejected, or never submitted, even though they might have been good enough for use.


The scale of the potential problem is unclear, but may extend to medicines submitted for approval in Europe, the United States and beyond.

Pharma Guy's insight:


Holy smoke, Batman!

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