There's a new Cop (Cha) in Town

• 09.25.2015

A savvy move from FDA – choosing someone who understands quality from the other side of the proposition. Only the most recent way that FDA is working to redefine 3D quality and pharmacovigilance.

For more on OPQ and the FDA's Quality Revolution, see here.

Here’s the official announcement from Janet Woodcock:

CDER Staff:

I am pleased to announce that Michael (Mike) Kopcha (“cop-cha”), Ph.D., R.Ph., a globally-recognized expert in product innovation and development, has been selected as the permanent director of the Office of Pharmaceutical Quality (OPQ). He will join CDER in November pending ethics clearance.

Mike is a leader in the development of innovative solutions to resolve scientific, manufacturing, and commercialization issues worldwide – and in standardizing and harmonizing global processes. With more than 25 years of pharmaceutical industry experience, his areas of expertise include formulation and process development, process validation, technology transfer, off-shoring/outsourcing, and change management.

OPQ – with close to 1,000 employees – was stood up in January to carry out new processes and policies to provide better alignment among review, inspection, and research functions. As OPQ director, Mike will take over the reins from me in matters relating to pharmaceutical quality regulation. These include submission review, manufacturing facility assessment, and quality surveillance.

Mike recently served as vice president, and global research and development franchise head, for cough, cold, and respiratory products at Novartis Consumer Health, Inc. in New Jersey. He joined Novartis in 2008 as the global head for pharmaceutical and analytical development, later serving as global head for new technologies and product innovation, and vice president and global head for global product development.

At Novartis, Mike has led the creation of a global R&D vision and strategies to drive innovation for the cough, cold, and respiratory franchise – through development, sourcing, manufacturing, and supply. He restructured and reorganized global product development to provide for a category-focused, globalized organization, and is credited for the creation of a successful strategic off-shoring/outsourcing program to increase project capacity and efficiency.

Before joining Novartis, Mike served as vice president for pharmaceutical development at KV Pharmaceutical, Inc. in St. Louis, where he directed and managed analytical research and development, product development, process development and technology transfer, stability, drug delivery, project management, and external alliance. His experience also includes related roles with Schering-Plough, J&J, and Ivax.

Mike earned his doctorate and master’s degrees in pharmaceutical science, and a bachelor’s degree in pharmacy, from Rutgers University in New Brunswick, New Jersey. He served as an adjunct assistant professor in the Department of Pharmaceutics, Ernest Mario School of Pharmacy at Rutgers.   

Mike will lead OPQ in planning and implementing strategies to drive pharmaceutical quality, with a goal of providing for an efficient pharmaceutical manufacturing sector that reliably produces high-quality medicines without extensive regulatory oversight.

I’d like to thank OPQ Deputy Director Lawrence Yu for his tremendous leadership, guidance, and support as we worked to stand up OPQ, and for assuming many additional responsibilities during my period as acting OPQ director.

Please join me in extending a warm welcome to Mike when he joins us later this fall. He will be a true asset to CDER and OPQ leadership. His broad knowledge and expertise in product development and technologies will allow him to play an essential role in our continued work to assure that the public has access to safe, effective, and high-quality medicines.

Janet Woodcock

Why people don't trust drug makers

• 09.25.2015

From today's edition of the New York Post ...

Why People Don't Trust Drug Makers

By: Peter J. Pitts

When former hedge-fund manager and current Turing Pharmaceuticals chief executive Martin Shkreli hiked the price of his generic anti-parasitic drug pyrimethamine from $13.50 to $750 per pill, he invited understandable national outrage. He also prompted presidential candidate Hillary Clinton to call for price controls on a wide variety of medicines.

The subtext here is clear: Americans think price-gouging on prescription medicines is rampant. This isn’t surprising, since the pharmaceutical industry has done a poor job of explaining why drugs cost what they do. More than three-quarters of Americans support mandatory limits on the price of certain drugs. And 86 percent want drug firms to disclose how, exactly, they set prices.

The best way for drug firms to quell this outrage is to give Americans what they’re asking for: more information. Specifically, the industry needs to shed light on the huge sums they spend on research, their rising research-and-development failure rates and the refusal of insurers and pharmacy-benefit managers to pass on manufacturer discounts to patients.

Of course, drug pricing is a complicated matter. Which is why the industry should focus on a few basic points when making its case.

First, the cost of research. Since 2000, drugs firms have spent over half a trillion dollars developing new medicines. And research costs for the last year alone totaled more than $51 billion. That’s up from $15.2 billion in 1995.

These are extraordinary spending levels, even compared to other research-intensive industries. In fact, the pharmaceutical sector spends five times more on R&D than aerospace, and 2 ½ times more than the software and computer industry. This is the kind of investment that pharmaceutical innovation demands, and it’s reflected in the economics of advanced drugs.

The industry also needs to do a better job explaining just how many failures firms endure searching for the next breakthrough medicine. Drug companies must develop hundreds of compounds until they find one suitable for testing on humans. Of those rare compounds that make it to phase-1 human trials, fewer than 12 percent win approval from the FDA.

That’s why bringing just one drug to market costs an average of nearly $2.6 billion and takes more than 10 years, according to researchers at Tufts.

If drug companies were open and honest about their frequent and expensive failures, they could quash the myth that pharmaceutical research is obscenely lucrative.

Drug prices aren’t merely the result of high development costs. The price of any given drug is the result of a lengthy process of meetings and negotiations between manufacturers and payers over a long period of time — often years before the FDA approves a product.

So when insurance companies demonize the pharmaceutical industry for high drug prices, they’re being more than a little disingenuous. Insurers and pharmacy benefit managers — not drug companies — are the ones who determine what patients pay for medications.

Consider the controversy surrounding the hepatitis C drug Sovaldi. When the medicine came on the market, it quickly became known in the press as “the $1,000 pill.” This may be a great sound bite, but it’s hardly accurate.

In reality, insurers and benefit managers negotiated discounts that reduced the price of Sovaldi by 20 to 50 percent. But they didn’t pass the full discount on to the consumer. Instead, insurers and pharmacy benefit managers pocketed the money to pad their bottom lines and executives’ wallets. Last year, CVS Caremark Corporation, one of the biggest benefit managers, paid its CEO over $32 million.

For many patients, particularly those without insurance coverage, Sovaldi’s manufacturer supplied a coupon ensuring that co-pays for the drug wouldn’t exceed $5.

In other words, the manufacturer went out of its way to ensure that few would have to pay full price for Sovaldi. In cases where patients had to shell out for “the $1,000 pill,” insurers were mostly to blame.

Most Americans, of course, are largely unaware of this information, which is why opinions have turned so sharply against the pharmaceutical industry. If they intend to win back the public, drug companies have some explaining to do.

Opioids and Sunshine at the CDC

• 09.24.2015

Turing is NOT Pharma

• 09.22.2015

It's easy to claim Turing Pharmaceuticals 5000 percent price hike of Daraprim is another example of how biopharmaceutical firms do business.

Easy, but untrue.


Here's Adam Feurstein of the Street.com explaining why he nominated Martin Shkreli as one of the worst biotech CEO's of 2014

Someone thought a smart but attention-addled man-child with a Don Bailey obsession and a Twitter account  could be a biotech CEO. They were wrong

In a previous post, Adam explains:

In the weeks before his ouster as the CEO of Retrophin (RTRX - Get Report) , Martin Shkreli was buying relatively small amounts of the company's shares on the open market and using his personal Twitter account to convince investors to do the same.

But while talking up Retrophin's prospects publicly and buying some of the stock, Shrekli was selling privately a much larger portion of his drug company holdings. Without public disclosure, Shkreli received almost $3 million in gross proceeds by selling "forward contracts" on his Retrophin stock in early September, according to a filing with the Securities & Exchange Commission made public Monday night.

Shkreli has not been accused of breaking any securities laws during his time at Retrophin. Last August, Retrophin's board admonished Shkreli for a series of inappropriate Twitter posts and the discovery that company employees were using alias Twitter accounts to promote Retrophin stock and make short-sale recommendations on other biotech stocks.

The new disclosure detailing Shkreli's selling of Retrophin shares comes at a delicate time. The former hedge fund manager has tried to restore his credibility and tamp down talk about his behavior and executive decision-making by blaming Retrophin's problems -- and his exit -- on the company's board and reporting by the media. At the same time, Shkreli is trying to raise outside investor money to finance a new company, Turing Pharmaceuticals.

Here's how Shkreli received almost $3 million in proceeds by selling Retrophin shares with delayed public disclosure:

On Sept. 9, Shkreli and an unnamed third party entered into a "prepaid forward contract" under which Shkreli promised to hand over 123,000 shares of Retrophin in two years. In exchange, the third-party buyer paid $1.07 million in cash to Shkreli -- an amount equivalent to 68% of the present value of the pledged Retrophin shares."


Again, courtesy of Feurstein on June 13 2011:

"As Neoprobe prepares to seek U.S. approval for its lymph node mapping agent, a New York hedge fund manager has shorted the stock and is taking an unusual step to prevent the company from getting a review by regulators.Martin Shkreli ofMSMB Capital Managementfiled a citizen petition with the U.S. Food and Drug Administration last week requesting Neoprobe's Lymphoseek be denied a review due to "severe deficiencies and flaws" in the conduct of two phase III clinical trials. Betting against FDA drug approvals is a well-worn (and often profitable) strategy in Wall Street's biotech trading canon. Shkreli, however, is pushing new boundaries by seeking to directly influence the FDA review process in his favor."

In 2013, Shkreli asked to speak before an FDA panel to argue against the approval of Lorcaserin, a weight loss drug made by Arena Pharmaceuticals Inc.  He had short positions in both Neoprobe and Arena

And here's an item that occured around the time Shkreli was raising $90 million for Turing.  

Retrophin Inc. founder Martin Shkreli, replaced last year as chief executive officer of the biopharmaceutical company, is under investigation by U.S. prosecutors in Brooklyn, New York, for possible securities law violations, according to a person familiar with the matter."

With this as background, it might be a better use of the Fourth Estate's time to ask the following questions:

How did Shkreli raise 100 percent of the $90 million Series A funding for a company with one product which, even at the highly inflated price of $750 a pill would generate less than $40 million in revenue?  

At Shkreli sought to drive down the share price of his company's stock in order to get exclusive marketing rights for a product at about a 90 percent discount. While Chief Investment Officer of MSMB He did the same thing in his effort to buy AMAG Pharmaceuticals in 2011 at a bargain basement price.  Here's what he said after badmouthing AMAG and driving down the share price:. “Over the past four years, the AMAG board of directors, presided over an 80% decline in the value of AMAG stock.   A letter the SEC sent to Shkreli reveals that the Turing CEO is very good a departing from the facts in explaining his investment activities.  
How do we know that Shkreli will not do the same with Turing?

A notorious short seller, is it possible that one way Shrekli is using to finance the offering is to use the price hike to short all other biotech stocks?

 Should the focus be on limiting the ability of short sellers and patent trolls to create drug companies that are essentially financed and designed to make money by a) bashing the core technologies of other companies as well as your own and b) buying up the marketing rights of one drug and using that, not the actual benefit of the drug, be the reason for huge price hikes?   

One other point worth further discussion.   In many cases health plans and PBMs have, by putting important medicines in the highest cost sharing tier, have in effect hiked drug prices by 5000%.   Isn't that wrong too? 

Finally, here's a good history of the way he wound up creating Turing. 

" Shkreli, fresh off Retrophin firing, says Turing Pharmaceuticals will be public by year-end The Deal Pipeline March 5, 2015 Thursday"
 

CDC gets it transparently wrong on opioids

• 09.21.2015

The pain community is not happy with the CDC. And who can blame them?

Per the CDC website, that agency is publishing new guideline (sic) “for prescribing opioids for chronic pain. The agency is working for timely release of the guideline while ensuring that the development process:

• Meets scientific standards
• Includes expert consultation
• Allows for appropriate stakeholders to provide input
• Facilitates partnership development to enhance dissemination and uptake

But the agency’s definition of “transparency” has many up in arms. It's a travesty. Here’s what the leading pain organizations had to say to CDC Director Tom Frieden and Debra Houry, Director of the National Center for Injury Prevention and Control:

Dear Drs Frieden and Houry:

As members of U.S. chronic pain management professional associations and patient advocacy organizations, we, the undersigned, are profoundly invested in any and all efforts undertaken that could impact either the positive or negative outcomes of pain treatment. We have been anxiously awaiting CDC’s draft opioid prescribing guidelines, now revealed September 16^th.

It was our expectation that the prescribing guidelines would have been

·      *  Developed in a transparent manner, consistent with best-known evidence

·      *  Or, where there is no evidence, by incorporating the common elements of long-accepted clinical expert consensus guidelines developed by a dozen organizations dedicated to the safe and effective treatment of patients with chronic pain

·      *  Consistent with those aspects of opioid medication prescribing and risk mitigation efforts that are actually supported with rigorous clinical trials, i.e. the FDA-approved indications for the full range of opioid formulations.

We were disappointed on all counts and now are deeply concerned that, not only are the CDC draft guidelines inconsistent with established best practices, they will potentially make it difficult for patients who rely on them for pain control to access them from clinicians who are clear on how best to use them.  

The CDC slides presented on Wednesday were not transparent relative to process and failed to disclose the names, affiliations, and conflicts of interest of the individuals who participated in the construction of these guidelines. The presenters refused to provide any information other than to read exactly what was written on the slides even when asked directly by audience members to disclose the processes and people who had developed these prescribing guidelines. Since CDC has traditionally not involved itself in developing and disseminating medication prescribing guidelines, and these recommendations are not consistent with current established guides for managing chronic pain with opioids, these process and participant questions are essential to understand.

A statement was made early in the presentation that it is acceptable and common practice to develop clinical recommendations even when there is a limited or non-existent base of evidence for (or against) the recommendation. In those cases, the standard is to convene expert clinicians with vast experiences in the condition and develop consensus around optimal approaches. CDC itself developed and published an analysis of existing guidelines that logically should have been the foundation for these new guidelines.

While there may be slight variances among organizations on specific guideline inclusions or recommendations, there is a broad conformity that extends beyond the focus of the new guidelines. By addressing only how to limit or avoid opioids, the new guidelines will inevitably result in fewer prescriptions overall - including those needed by patients with legitimate medical needs. Chronic pain advocacy organizations hear daily from increasing numbers of constituents who are not being able to access the opioid medications they’ve relied on to live with their chronic painful conditions. That is not an outcome that anyone involved in chronic pain and prescription opioid diversion and abuse wants but this guideline will produce.

In fact, it is CDC’s singular focus on prescription opioid diversion, abuse, addiction, and overdose over any improved understanding of chronic pain incidence, prevalence, trends, and optimal interventions that reveals within CDC an extreme imbalance in its own risk-benefit sensibilities when it comes to this class of medications. In evaluating new medications for approved uses, FDA requires safety and efficacy trials that all approved opioid medications have met. Detailed prescribing instructions are developed based on proven studies. Yet the new guidelines ignore the FDA’s prescribing expertise, recommending different maximum daily doses that appear in no guidelines or package inserts.

We call on CDC to immediately compile, analyze, and report any and all chronic pain data it possesses, managed with or without opioids. Certainly any health condition that impacts one third of our country’s population should, by definition, have a place of priority in CDC’s mission and mandate. CDC has a Center for Chronic Disease Prevention and Health Promotion that reports on arthritis as part of its annual chronic disease monitoring. Diabetes, interstitial cystitis, inflammatory bowel disease are all programs within this center that have, at their core, chronic pain components and may contain useful data. CDC also fields an Annual CDC/NCHS National Health Interview Survey that reports on severe headache or migraine, low back pain, and neck pain among adults. Obviously, a better understanding of how to effectively treat chronic pain should be an essential component to any treatment prescribing guideline. What CDC presented this week is not that.

If the new CDC guidelines reinforced existing recommendations of experts who agree there are tried and true approaches to safely and effectively using opioids to treat chronic pain, it would be a welcomed addition to clinical practices nationwide. Equally important, we need CDC to glean from its prescription opioid addiction and overdose data which cases actually involve chronic pain patients and which involve patients with active substance use disorder so we can help providers better differentiate the two.

The unmet challenge in chronic pain management with opioid treatment is to identify the conditions for which, and patients for whom, opioid use is most appropriate; the regimens that are optimal; the alternatives for those who are unlikely to benefit from opioids; and the best approach to ensuring that every patient’s individual needs are met by a patient-centered health care system. We need CDC to provide some context around the incidence and prevalence of undertreated pain and the related adverse consequences of undertreated chronic pain on all body systems. With these insights it may actually be possible to improve pain care rather than restricting one treatment based on perceived, not quantified, harms to legitimate patients.

We believe the most important outcome of treatment for chronic pain is pain control sufficient to enable people with pain to maintain a high level of patient-determined quality of life and functionality while avoiding or minimizing potential negative outcomes that could outweigh pain control benefits. We believe it is up to the patient, informed by a comprehensive and ongoing discussion with his or her providers, to decide if risks of opioid use apply to them and are acceptable to bear, when compared with the adverse physical, psychological, and quality of life impacts if pain cannot be adequately controlled without opioids. Our concern is ensuring that further stigma and physical harm to patients does not result from policies and guidelines that address only the risk of harm from inappropriately prescribed or used opioids, when what healthcare providers most require are affirmative strategies to help patients manage their pain.

Sincerely, 

Alliance for Patient Access*

American Academy of Pain Management*

American Cancer Society, Cancer Action Network, Inc.*

American Chronic Pain Association

Center for Lawful Access and Abuse Deterrence

Chronic Pain Research Group at the Clinical Pharmacokinetics Laboratory, University at Buffalo School of Pharmacy and Pharmaceutical Sciences*

Hospice and Palliative Nurses Association

Interstitial Cystitis Association

Massachusetts Pain Initiative

National Fibromyalgia & Chronic Pain Association

Pain Connection

Project Lazarus

The Pain Community

US Pain Foundation

Virginia Cancer Pain Initiative

                                                                                                  
* Participant in the FDA August 6 meeting

This unfair, unjust, and ill-considered CDC action must not stand.

The Califf Regime and the Critical Path to Confirmation

• 09.17.2015

Some good quotes from A Califf Regime: The Disruptor FDA, Industry Need, by the very savvy 
Donna Young of Scrip Intelligence --

The complete story (subscription only, sorry) can be found here.

… If Robert Califf is confirmed as the next commissioner of the FDA – which most in the biopharmaceutical community are anticipating is all but assured – he'll be taking over an agency that's in the midst of some of the most complicated challenges it's ever faced, the resolutions of which have broad implications for drug and device makers.

… The FDA is dealing with an inability to attract and retain the best and the brightest scientists, noted Ellen Sigal, chair of the nonprofit advocacy group Friends of Cancer Research (FOCR). "The salaries are terrible, hiring practices are convoluted and divestiture is complex," she told Scrip.

… Greg Daniel, managing director for evidence development and innovation at the Brookings Institution's Center for Health Policy and a fellow in economic studies at the Washington think tank, said another problem confronting the FDA is its need for consistent resources and funding to maintain and build on the advances the agency has made in developing new tools and refocusing on patients through new expedited development and review programs.

… Peter Pitts, president of the Center for Medicine in the Public Interest, a Washington think tank, and a former FDA associate commissioner during the George W. Bush administration, insisted Califf is the right leader and "precisely the right guy" to handle the knotty challenges ahead for the US regulatory agency – declaring the former Duke University professor's nomination was "the most important health care act the president has undertaken," even more so than getting the Affordable Care Act enacted.

President Obama’s nomination of Califf, announced on Sept. 15, "really is the biggest thing he's done to advance the public health," Pitts told Scrip. "I really believe that."

If confirmed as commissioner, Pitts said he thinks Califf, who he called an "action-oriented guy," will have an "immediate impact for many issues that need immediate attention" at the FDA – most specifically, precision medicine.

The fact that the past few times a new commissioner was being sought by Democratic and Republican presidents and Califf's name kept coming up as a potential contender demonstrates "he's highly qualified" and capable of doing the job, Pitts said, adding that it also shows that "occasionally, there is bipartisan sanity when it comes to health care issues."

What's most important, Pitts said, is that Califf is viewed as a well-respected scientist – both by regulators and industry alike.

Before joining the FDA this past February as deputy commissioner for medical products and tobacco, Califf had worked closely with the agency on several matters, including serving on its Cardiovascular and Renal Drugs Advisory Committee, "so his learning curve is not steep," Pitts said.

He said Califf also is well respected among regulators at agencies outside the U.S. – an asset that could aid the FDA and other regulatory bodies in harmonizing their review processes and rules, which could broadly benefit the drug and device industries.

But one of the greatest qualities Califf possesses, Pitts said, is his ability to innovate.

"He can take a theory and make it a practical reality," Pitts said, adding that he anticipates Califf to lead the FDA to be "partners in innovation" with industry through "entrepreneurial regulation."

"What that means is that Rob Califf can put the FDA at the center of the health care innovation ecosystem," he declared.

… "He's well known to be an innovator," Steven Nissen, head of cardiology at the Cleveland Clinic in Ohio, said about Califf. "He's been innovative in the design of clinical trials and has long been an advocate of finding ways to do clinical trials that are better, cheaper, faster. He's been somebody who has been outspoken about the fact that the approach we use for drug development is too costly and in some cases too long." Nissen said the current FDA is suffering from "years of stagnation."

"The FDA has not moved forward on any important initiatives during this period of stagnation," he argued. "And it needs a new look. It needs somebody to really come in and really shake things up."

Califf, Nissen said, is "capable of doing that … We really actually see the world pretty similarly."

Industry Ties

But while Nissen and Pitts showered praise on Califf, Public Citizen's Carome asserted President Obama's candidate to run the FDA was "bad news for patients and public health" because of the former Duke University professor's "long history of extensive financial ties to multiple drug and medical device companies."

But Pitts argued that "in my mind, the commissioner of the FDA should have an extremely up close and personal knowledge of industry to understand what's happening … I think anybody who knows Rob Califf knows he's not swayed by any financial interests," he said.

… Pitts, Nissen and Sigal all advocated for keeping Califf in the commissioner's role long after President Obama leaves office – declaring the FDA chief's job should be a five- or six-year term.

"I've long been an advocate for this not to be a political appointment," Nissen said.

The next critical path is the path to confirmation.
 

RE: Rob Califf -- CQ on the QT

• 09.16.2015

Good story in Congressional Quarterly on President Obama’s nomination of Rob Califf to be the next FDA Commisisoner.

Unfortunately, since CQ is a subscription-only service, I can only provide some snippets:

Califf, according to several former FDA officials, is a believer in new technology and involving the voice of the patient in the drug development process.

"He understands what the future of health care looks like," said Peter Pitts, who led FDA external relations during the George W. Bush administration.

Califf previously was a top official at Duke University's School of Medicine. During a 30-year career at Duke, he was in charge of the Translational Medicine Institute, whose goal is to speed up the development, testing and approval of drugs and devices.

At the time of his appointment, the FDA hailed Califf, a cardiologist, as "one of the top 10 most cited medical authors." Shortly before taking his FDA position, Califf oversaw a vast long-term study that revealed that a cholesterol-lowering drug could help prevent heart attacks in some patients who cannot tolerate a more common cholesterol-reduction method, statins.

But during his years at Duke, Califf developed close ties to players in the pharmaceutical industry. Some believe these relationships will be an asset for someone who needs industry buy-in for his agency's policies. If Califf is confirmed, Pitts said, it "means the FDA can be both the regulator of industry and its colleague in innovation."

The Senate Health, Education, Labor and Pensions Committee would hold a hearing on Califf's nomination.Sen. Lamar Alexander, R-Tenn., the committee chairman, said in a statement that the panel will move promptly on the nomination.

"Dr. Califf has impressive credentials," said Alexander. "The FDA affects nearly every American and needs the certainty of a strong leader. I look forward to hearing from Dr. Califf specifically on how we can move medical discoveries more rapidly through the FDA and other agencies and get safe treatments and drugs into Americans' medicine cabinets."

If Califf is confirmed, he may only have a little more than a year on the job. But FDA observers say that he's already worked hard to earn a rapport with different branches of the agency and policymakers.

Former FDA official Frank Sasinowski, now a consultant, said Califf will be able to "go into congressional hearings and people will understand that he has an appreciation of what it takes to drive innovation and get new therapies out to patients."

If you are a CQ subscriber, the full article can he found here.

FDA puts up its Dukes

• 09.15.2015

President Obama has made the most important healthcare decision of his administration. (No, not that.)

The nomination of Rob Califf to be the next Commissioner of the FDA signals the beginning of a new era at the agency that regulates more than a quarter of the US economy. Welcome to the era of entrepreneurial regulation.

Entrepreneurial Regulation means that the FDA can be both regulator of industry and its colleague in innovation. Expedited pathways are Entrepreneurial Regulation. Breakthrough Designation is Entrepreneurial Regulation. The Super Office of Pharmaceutical Quality is Entrepreneurial Regulation. More aggressive use of the Reagan/Udall Foundation is Entrepreneurial Regulation. Patient Focused Drug Development is Entrepreneurial Regulation.

It’s more than time for the FDA to take its place in the center of the healthcare ecosystem.

There are many issues facing the FDA and not just in drugs. Food safety, device reform, dietary supplement regulation. It's a long list. Califf knows the issues and the players -- most importantly those inside the agency. His nomination is both timely and savvy -- particularly with PDUFA reauthorization under way.

Califf’s nomination is an n-of-1 opportunity. When considering who could replace Peggy Hamburg, Rob Califf was the short list.

Put up your Dukes? In more ways than one.

The Merlion in Summer

• 09.14.2015

I've just returned from Singapore where I spent some quality time with Raymond Chua (the director of the Singapore HSA -- their version of the FDA) and his senior staff. We covered a wide variety of issues ranging from "what's new at the USFDA?" (Answer -- a lot!), global regulatory convergence, and much in between (such as Singapore as a regulatory “third way” in addition to the FDA and EMA).

The HSA directorate was particularly interested to discuss the USFDA's expedited review pathways (breakthrough therapy designation, accelerated approval, priority review, etc.) and some of the ways they’re impacting regulatory policy (i.e., adaptive clinical trial design, senior staff commitment, earlier sponsor/agency interaction). Per Raymond, the HSA is "pathway agnostic" -- an interesting concept that deserves more discussion and consideration in White Oak. He was also keen to learn more about the USFDA's new "Super Office" of Pharmaceutical Quality. (For more on this, see here.)

When it comes to 21^st century drug and device regulation, Singapore is a place to study for best practices as well as innovation in both cogitation and action.

The Merlion roars loudly and proudly on behalf of it’s nation’s public health. And there's much the rest of the world can learn from both it's practices and world view.

(And the eats are pretty spectacular too. I recommend the Chili Crab.)

Zeke Emanuel's Final Solution For Drug Prices

• 09.10.2015

In his NYT oped "The Solution to Drug Prices" Zeke Emanuel claims price controls are a good thing as long as they are connected to the value of the drug.  I assume that he thinks longer life and reduced health care spending are measures of value. 

He thinks Australia and Switzerland are good examples of how to do price controls right. 

In fact, there are several studies of both pricing systems.  In Australia life expectancy increased by 11 months for people who had access to new drugs faster.  mean age at death increased more for diseases with larger increases in mean drug vintage.  Life expectancy for people who had older medicines didn't budge.In  Australia’s Pharmaceutical Benefits Advisory Committee recommended only  two of twenty-six drugs  with a cost per life-year saved greater than $57,000. Only one of twenty-six submissions with a cost per life-year saved less than $32,000 was rejected. Australia ranked 18th out of 20 comparable OECD countries in access to new medicines, with cancer patients waiting over two years after a drug is approved

In Switzerland a study concluded that 17000 life years were gained in 2012 due to the kind of cancer drugs Emanuel claims are NOT cost-effective.  In fact the cost per life-year before age 75 gained from cancer drugs was $21228.  Moreover, the study found that by delaying access to these medicines as part of the price control program, Swiss cancer patients died sooner than they had to.  Finally, another study concluded that Swiss heart patients  who  used  newer  cardiovascular  drugs  in 2003 lived longer than  people  who used older cardiovascular drugs. (Meanwhile, in the US generic versions of patent medicines are less expensve than the price controlled brand products in Europe.)

Americans with heart disease and cancer live longer than patients in other countries even though many spend more on on these diseases.  The price controls Emanuel pushes would save less than .5 percent of total US health spending.  They would drive up total costs and make life shorter and more painful for millions.