Safe Hands Across the Ocean

05/15/2014 06:29 AM |

Dear GO, CDER, and CBER staff:

We are pleased to announce an initiative to further our goal of ensuring that the public has access to quality pharmaceuticals. This initiative aims to deepen our reliance on trusted regulators outside of the United States who provide equivalent public safety and quality protection. This work is critically important and requires a dedicated FDA team to collaborate with our colleagues in European institutions and member states.

Managed jointly by GO, CDER, and CBER, this initiative will build upon our existing relationships with the European Medicines Agency (EMA) and member states of the European Union (EU), the European Commission (EC), and the European Parliament. It will complement the ongoing work of CDER, CBER, ORA, and OIP to enhance pharmaceutical quality through international collaboration, and also FDA’s work in implementing the FDA Safety and Innovation Act.  

Dara Corrigan to Lead Initiative as Senior Counselor for Global Operations and Policy

Effective May 18, Dara Corrigan will lead this mutual reliance initiative. She will leave her position as director of FDA’s Europe Office, but remain in Brussels, in the role of senior counselor for global operations and policy. Dara will report directly to Howard Sklamberg, deputy commissioner for global operations and policy.  She will provide strategic advice and counsel to the FDA Commissioner and other FDA senior executives. Don Prater, DVM, will serve as acting director, FDA Europe Office.

Dara brings a wealth of experience and knowledge of U.S. and EU laws and regulations that govern pharmaceutical products. Before serving as director of FDA’s Europe Office, she was FDA’s associate commissioner for regulatory affairs, overseeing one of FDA’s largest operating units and managing FDA’s inspection program. She also has held a variety of senior positions in HHS, and served as an attorney with the U.S. Department of Justice and at the law firm of Arnold & Porter.

CDER OCOMM’s Julie Zawisza and CDER OC’s Helen Saccone to Join Team

Dara will oversee a team that will be dedicated to mutual reliance activities and that will report to GO, CDER, and CBER. The team will include Julie Zawisza, who has served as director of CDER’s Office of Communications since 2008, and Helen Saccone, who has been a special assistant in the Office of Manufacturing and Product Quality (OMPQ) in CDER’s Office of Compliance since 2011. 

Julie holds a master’s degree in international science, technology, and public policy from the George Washington University and has more than 14 years of FDA experience. Before working in CDER, she worked in communications at CBER and was the head of FDA’s press office in the Office of the Commissioner. Julie also has worked for health professional and industry associations in communications and advocacy roles, and as a medical technologist at major medical centers in Washington, D.C. and Ann Arbor, Michigan.

Helen Saccone, Pharm.D., has worked in FDA for six years. Before coming to FDA, she worked as senior manager for education at the American Pharmacists Association and as a pharmacist for the D.C. Department of Health, conducting pharmacy and hospital inspections, as well as providing technical assistance on D.C. regulations. She holds a doctoral degree in pharmacy from Rutgers University. The team also will draw upon the work of other experts in CDER, CBER, ORA, OIP, and OCC.

We are delighted that these seasoned and accomplished individuals have agreed to lead the charge, and we have every confidence that the program is in very capable hands going forward. We invite you to congratulate Dara, Julie, and Helen, and offer them your support.

Building on Existing Pharmaceutical Quality Efforts

We want to thank our FDA staff members who continue to work so diligently on improving pharmaceutical quality and safety for the American public. The initiative announced today builds on the Agency’s ongoing efforts to enhance collaboration between FDA centers and ORA, and to bolster our resources around drug quality.

We are very excited about this programmatic direction and its potential to leverage our relationships with our European partners and capitalize on our shared interests as regulators to strengthen mutual reliance and ensure pharmaceutical quality for the U.S. market. 

In the coming months, you'll hear more about all of this as the team embarks on its first set of activities.  A special town hall will be scheduled soon to give FDA staff an opportunity to ask questions about this initiative and how it fits in with the Agency’s pharmaceutical quality efforts.

Howard Sklamberg, Deputy Commissioner for Global Regulatory Operations and Policy

Janet Woodcock, Director, Center for Drug Evaluation and Research

Karen Midthun, Director, Center for Biologics Evaluation and Research

  Read More & Comment...

A Biosimilar Hierarchy

05/14/2014 06:48 AM |

  Read More & Comment...

Spread the wealth and advance the health

05/13/2014 06:36 AM |

At the May 6th House Energy and Commerce Committee’s 21st Century Cures Initiative roundtable, participants were asked what steps Congress could take to expedite bringing new treatments and cures to patients.

Francis Collins, director of the NIH, said that what is needed is a “steady trajectory of support” so that scientists are willing to take risks. But what does that mean?

Collins said that over the past 10 years, the NIH has lost 20 percent of its purchasing power. His belief is that this loss has cost jobs and caused a lack of enthusiasm in our investigators, he said.

20%? That sounds like a statement worth investigating – because the NIH budget has grown significantly over the past decade.

“If we could have confidence of a stable trajectory for support, that would mean the world for an enterprise that is currently flagging,” he said. Not sure precisely what that means but, again, perhaps a study of how NIH is spending it’s current budget would help identify the strengths and weaknesses of its allocations – and the strategy behind them. After all, every dollar counts.

Janet Woodcock, director of the Food and Drug Administration's Center for Drug Evaluation and Research (CDER), said one issue affecting biomedical innovation is the current clinical trials system for drugs.

“The clinical trial system we have is not a system,” Woodcock said. “It takes years and it exhausts investigators.”

Woodcock said the FDA is starting to look at clinical trial networks that are already set up and funded. Testing a new drug in a clinical trial network is faster and “saves a lot of money,” she said.

Additionally, per Dr. Woodcock, since multiple new drugs are studied by the network, “you can do head-to-head comparisons of products.”

She also said there is a lot of innovation in drug manufacturing, and that the FDA will hold a meeting to pursue this avenue in a few weeks.

Another area that the FDA is focusing on is the Critical Path Initiative. Its goal is to bring innovative, high priority therapies to market quickly.

“There is a lot of research that needs to be done on things like biomarkers that would” speed the development of products, Woodcock said.

And, according to Margaret Anderson, executive director of FasterCures (an advocacy group aimed at improving the medical research system that is affiliated with the Milken Institute), “The appropriated dollars that go to the FDA are extremely valuable, and they are not enough.”

This past Sunday, I participated in a point/counterpoint in the pages of the Chicago Tribune with Dr. Kenneth Polonsky, Dean of Biological Sciences Division at Pritzker School of Medicine, and executive vice president, medical affairs, at the University of Chicago.

Dr. Polonsky’s perspective was, more or less, the same as Dr. Collins’, “more money equals more cures.” If only it was so simple. My view (IMHO) is somewhat more nuanced.

Here is Dr. Polonsky’s commentary.

And here’s mine:

Outside Opinion: Federal funds should go to medicine-makers

By Peter J. Pitts

Is more federal funding for the National Institutes of Health the best bang for the buck when it comes to using precious tax dollars to advance public health? No.

The NIH budget is about $30 billion. But what does that buy? Where do discoveries that advance public health really come from? Some do come from NIH-funded research — but not nearly the majority. The engine of innovation is the biopharmaceutical industry, which spends in excess of $50 billion annually on research and development. It's not a competition; the NIH and industry complement each other's efforts. But context matters.

The NIH focuses on basic research, the study of fundamental aspects of phenomena without specific applications. The biopharmaceutical industry addresses most of its R&D toward clinical research, science focused on the actual development of new medicines. The NIH provides grants to academic institutions. Industry employs the scientists who do the work and, increasingly, funds academic research.

But there's a problem. While universities love NIH dollars, they are less enamored of industry resources. Why? One reason is that NIH funding counts toward achieving tenure, while similar dollars from biopharmaceutical companies do not. Durbin's legislation would disincentivize more industry-academic partnerships. More government spending is not always the mother of invention.

As the Prairie State's great Sen. Everett Dirksen once (allegedly) quipped, "A billion here, a billion there, and pretty soon you're talking about real money." Some $10 billion annually could be allocated elsewhere to achieve broader access to newer, more targeted therapeutic medicines (and $5 billion could go toward the NIH's good work, hardly a paltry sum). The Food and Drug Administration should be No. 1 on the list to get more money.

The FDA regulates more than 25 percent of the U.S. economy, yet operates on an annual budget of $4.7 billion (about $2 billion generated by industry user fees). The budget's federal funding portion is about one-tenth the NIH's. Why hasn't Durbin proposed additional tax dollars for the FDA's programs on advancing regulatory science, expedited review pathways or more ready access to experimental medicines for desperately ill patients? The FDA doesn't even need $10 billion a year for 10 years to become our nation's leading force in health care innovation. Some $1 billion a year would do the job quite nicely. As to the remaining $9 billion, the line forms to the left.

Alas, headlines for hyped and misleading "NIH-funded cures" are far sexier than those for "more money for drug regulation." They may not be inversely important, but they are equally urgent in advancing 21st-century American health care.

  Read More & Comment...

What does Beijing know that Washington doesn't?

05/12/2014 07:15 AM |

China is adopting a free market solution to drug shortages.

China will scrap caps on retail prices for low-cost medicine and is moving toward free-market pricing for pharmaceuticals, after price controls led to drug quality problems and shortages in the country.

The Wall Street Journal reports that Chinese leaders want health care to be more accessible and affordable, but there have been unintended consequences in attempting to ensure the lowest prices on drugs. For instance, many pharmaceutical companies registered to sell the thyroid medication Tapazole have halted production in recent years after pricing restrictions squeezed out profits, experts say, creating a shortage.

The lesson for US lawmakers is clear – artificially low prices are the major cause drug shortages.

Perhaps its time for our lawmakers to revisit the legislative solution proposed in Senator Orrin Hatch’s Patient Access to Drugs in Shortage Act. There are three key codicils:

1. Price Stability

The Hatch bill would change the Medicare reimbursement rate for generic injectable products with 4 or fewer active manufacturers from ASP + 6% to Wholesale Acquisition Cost in order to achieve market price stability.

2. Medicaid/340B Rebate Exemption

The bill exempts generic injectable products with 4 or fewer active manufacturers from Medicaid rebates and 340B discounts in order to achieve market price stability.

3. Extended Exclusivity

Manufacturers who hold an approved application for a drug that would mitigate a shortage can extend by 5 years any period of exclusivity, even if the drug is eventually moved from drug shortage designation.

It’s embarrassing that the world’s leading free market economy (that’s us) hasn’t learned the Econ 101 lessons our friends in China are now implementing to solve the problem of drug shortages.

  Read More & Comment...

Protecting Sustainable Innovation

05/09/2014 09:20 AM |

HEP, HEP, Hooray!

CDER Director, Dr. Janet Woodcock. responding to criticism about the high price of breakthrough drugs, said that the agency is working towards approving more effective treatments for diseases like cancer and hepatitis. Reacting to controversy around the price of the breakthrough Hepatitis C drug Sovaldi, Dr. Woodcock discussed the potential decrease in societal costs and increase in patients' quality of life.

"I think we have to in some ways think about this as a transition period," she said during a panel discussion about the breakthrough designation. "We may have to put a big down payment down now to get something really good."

She highlighted the cost to society and burden on patients in dealing with the side effects and morbidity of having Hepatitis C. "I really do believe we need to drive toward curing, but you have to have a transition period, she said. "We are driving toward a cure with hepatitis."

Woodcock further advocated getting these drugs on the market so they could be combined with other products to drive toward cures. "In cancer, I think we have to recognized this is version 1.0, but we're going to get there," she said. "And to get there we can't hold back. We can see that cure."

 The battle for the heart and soul of 21st century health care is the battle over innovation. And nothing short of victory is acceptable. To borrow an over-used adjective from the world of global climate change, we must protect “sustainable” innovation.

  Read More & Comment...

Timing is Everything

05/08/2014 09:20 AM |

Representative Anna Eshoo (D, CA) and the late Senator Edward Kennedy worked long and hard to write, lobby for, and pass the Biologics Price Competition and Innovation Act.

Unlike many of her congressional colleagues, Representative Eshoo has the creds to ask some tough questions of the FDA. She’s also smart enough to know the right questions to ask.

Unlike some of her fellow members who are swinging their political heft, trying to lobby the FDA on science-based questions, Ms. Eshoo is asking for clarification on the timing of a decision.

That is an appropriate and important question.

Representative Eshoo, in a letter to FDA Commissioner Hamburg asks (among other things) that the agency …

“Share with me the FDA’s timeline for the release of the draft guidance on naming and interchangeability.”

That’s a fair question from a member of Congress who deserves a prompt answer.

Observe due measure, for right timing is in all things the most important factor.

Hesiod

  Read More & Comment...

Putting "vigilance" back in pharmacovigilance

05/07/2014 06:46 AM |

I’ve just returned from Riyadh, Saudi Arabia where both the Saudi FDA and the Ministry of Health are embroiled in the current MERS (coronavirus ) crisis.

It’s already cost the Minister of Health his job and many citizens are questioning the ability of the government to protect their wellbeing. It’s also an international news story that does not reflect well on the Kingdom.

Another reason to reexamine the state of overall health preparedness – and not only in Saudi Arabia.

A good place to start is with a serious conversation on 21^st century pharmacovigilance. Let’s start with definitions.

According to the WHO, “Pharmacovigilance is defined as the science and activities relating to the detection, assessment, understanding and prevention of adverse effects or any other drug-related problem.”

Okay as far as it goes. But this is a very 20^th century perspective. Perhaps a more progressive view comes from our regulatory cousins at the MHRA, “Assessing the risks and benefits of medicines in order to determine what action, if any, is necessary to improve their safe use.”

The significant difference is that most regulatory officials still view PV through the lens of adverse events. The MHRA definition, however, broadens the conversation to include safe use. That’s 21^st century pharmacovigilance.

What does this have to do with the coronavirus?

It speaks to the need of public health officials to be both the guardians of the public health as well as the watchmen. And that takes more than vigilance – it takes resources. It also takes a village – a public health village – comprised not just of public officials, but physicians, nurses, pharmacists, hospitalists, patients, and caregivers.

And it’s got to be more than MedWatch.

How much sooner could the coronavirus been identified and dealt with if the Saudi Ministry of Health had a more robust mechanism for post-market surveillance that went beyond adverse events?

But it’s not just the Saudis.

The FDA is establishing an Office of Pharmaceutical Quality to improve the agency’s scrutiny of brand-name, generic and over-the-counter drugs, CDER Director, Dr. Janet Woodcock said at the Bloomberg health-care summit. The FDA is talking with the industry to develop data that may signal which manufacturing plants are straying from standards and need inspection, she said.

The agency now collects such information only during inspections. The thrust of the effort would be to head off potential concerns before the agency wields penalties such as banning products from troubled factories. “We want to use leading indicators. These people aren’t in trouble yet but they could be.”

Forewarned is Forearmed.

  Read More & Comment...

Compassion, it seems, is in the eyes of the beholder

05/06/2014 09:25 AM |

Yet another reason to stop calling it "compassionate use" and start calling it by it's proper name -- "expanded access."

The Wall Street Journal reports …

The FDA Says It’s More Compassionate Than You Think

Even as drug makers and desperate patients increasingly battle over access to experimental medicines for life-threatening conditions, Food and Drug Administration officials argue the agency is doing its part to make things right.

In the wake of some recent high-profile cases that made television news and prompted social media outcries, the FDA has released data showing that nearly every request it gets for “compassionate use”’ is approved.

The term refers to an FDA program in which individuals who are seriously sick and lack options are able to gain access to a medicine being developed, even though they are not enrolled in a clinical trial. For this to happen, a patient’s physician must first receive permission from the drug maker testing the medicine. From there, the physician must seek what amounts to a blessing from the FDA.

In recent years, the FDA has been very agreeable. In the fiscal year ended last October, the agency approved 863 requests, or 99 percent of all cases reviewed, according to FDA data. And one-third of those were approved on an emergency basis. In fact, the agency has approved 99 percent of all requests since October 2009. On average, 932 requests were endorsed annually since then.

“I think the numbers speak well for the program,” says Richard Klein, director of the patient liaison program in the FDA Office of Health and Constituent Affairs.

In reality, though, the process is not working as some patients would hope. Drug makers sometimes deny requests for compassionate use because they want to stick with strict trial criteria needed to win FDA marketing approval for their medicines. An unexpected patient reaction, for instance, might jeopardize a drug’s chance of success. In some instances, a company claims not to have sufficient supplies to handle a large number of requests.

This is what happened recently when a small biotech company called Chimerix denied a Virginia family. Their 7-year-old son, a cancer survivor, developed a viral infection after a bone marrow transplant and they hoped he would benefit from an antiviral drug Chimerix is developing. But repeated rejections became a publicity debacle as criticism was directed at the company and the FDA program as well.

The FDA said it intervened by working with Chimerix to design a pilot study to include the boy. That pilot study is now underway, according to a Chimerix spokesman. But an FDA spokeswoman says this was not the first time the FDA took such a step. Meanwhile, the FDA’s Klein says the agency is developing a new draft guidance, or blueprint, for handling expanded access cases.

“The agency only provides the pathway,” he says. “If a company is not willing to entertain a request [for compassionate use], then it doesn’t come to us. Most of the time, we only know about the applications where companies are willing to make drugs available. The numbers we have don’t include the times when companies say no. And there’s not a lot of data to say what the outcomes of these situations are.”

Maybe not. But perhaps FDA officials should find a way to require drug makers to report their compassionate use decisions – approvals and rejections – so the public can see the extent to which the program is having its intended effect.

  Read More & Comment...

It only hurts when I click

05/05/2014 02:29 PM |

According to Robert Jamison, PhD, professor of anesthesia and psychiatry at Harvard Medical School and pain psychologist with Brigham and Women’s Hospital, mobile medicine is helping chronic pain patients cope with and manage their condition thanks to new smartphone apps, which can track patients from a distance and monitor pain, mood, physical activity, drug side effects, and treatment compliance.

Per Jamison, smartphone apps are helping the shrinking ranks of pain specialists treat and monitor rapidly increasing populations of chronic pain sufferers. “Today the ratio is one pain specialist for every 10,000 patients, but mobile technology allows for easy time-effective coverage of patients at a low cost, offering significant opportunities to improve access to health care, contain costs, and improve clinical outcomes,” he explained.

At the American Pain Society annual meeting, Jamison presented results of his research on smartphone apps, developed at Brigham and Women’s, for monitoring pain patients. He found that internet-based cognitive behavioral therapy could significantly decrease pain levels, improve function, and decrease costs compared to standard care.

“Online networks, for example, can promote communication, distraction, information sharing, self expression and social support,” Jamison said. “We also believe online networks decrease feelings of withdrawn behavior and instill a greater willingness to return for treatment.”

Jamison added that electronic diaries maintained by patients are more effective than paper diaries for evaluating pain levels, daily activities, treatment compliance and mood.

Jamison said that while few studies have been conducted on text messaging as a pain management tool, texting has proven to be effective for managing patients with diabetes, hypertension, asthma, smoking cessation and weight loss.

In his ongoing research, Jamison is studying 60 patients with chronic cancer and non-cancer pain who use a pain management smartphone app. “We hypothesized that the pain management smartphone app will help providers track patients and reduce emergency department visits and hospitalizations by 50 percent,” said Jamison.

A key feature of the pain management app is daily pain tracking in which patients are asked five questions about their pain, activity interference, sleep, mood, and overall status on a sliding scale of 1 to 10, and compare these with baseline ratings. Should pain ratings significantly increase from baseline or reach 9 or 10, the patient gets an immediate response that the pain specialist has been contacted.

“The pain management smartphone app can deliver non-pharmacological, cognitive behavioral treatment as well as prompt patients to stay active, comply with therapy, and develop pain coping skills,” Jamison reported. He added that the smartphone data can be summarized and transmitted every day into the patient’s electronic medical record.

Jamison noted that the average response rate to text messages to pain patients is 70 to 90 percent and that high responders show improved pain levels.

  Read More & Comment...

The Tao of Pain Management

05/01/2014 08:50 PM |

From the op-ed pages of the Washington Times ...

PITTS: America’s pain and opioid problem

Millions risk overdose death as they seek relief from suffering

Former Canadian Prime Minister Pierre Trudeau once said, "There's no place for the state in the bedrooms of the nation." What's the appropriate place for the state in our nation's pharmacies and medicine chests — particularly regarding for opioid pain medications?

Earlier this month, the Food and Drug Administration (FDA) took a big step in making sure that the number of opioid drug-related deaths doesn't continue spiraling out of control by approving a drug called Evzio. A take-home, one-time-use autoinjector, Evzio is the first drug of its kind. It releases a narcotic antagonist called naloxone to reverse the effects of an opioid overdose until emergency medical help arrives.

Fast-tracking drugs like Evzio, which will help dramatically reduce the number of opioid-overdose deaths, is just one part of a complex solution. Improved provider and patient education is crucial. Caregivers need to know how to properly prescribe based on an individual's pain-management needs, and patients need to know how to properly follow their treatment plans. If not, addiction will continue consuming lives.

Opioids work by targeting the same receptors in the brain as heroin, resulting in feelings of euphoria. The numbers speak for themselves: People are hooked.

According to the National Institute on Drug Abuse, 5.1 million people reported abusing prescription painkillers. Teenagers account for a large share of the rising prevalence of opioid abuse — they report abuse or dependence problems at six times the rate of folks 50 and older. Eight percent of high school seniors report using Vicodin nonmedically. Curtailing the frequency of opioid-related abuse — and at an early age — must remain a priority.

It's not just a user problem. It's a provider problem, too.

In the United States, the use of opioids as first-line treatment for chronic pain conditions doesn't follow either label indications or guideline recommendations. Fifty-two percent of patients diagnosed with osteoarthritis receive an opioid pain medicine as first-line treatment, as do 43 percent of patients diagnosed with fibromyalgia and 42 percent of patients with diabetic peripheral neuropathy. Payers often implement barriers to the use of branded, on-label non-opioid pain medicines, relegating these treatments to second-line options. The result is a gateway to abuse and addiction.

How do we fight the twin dangers of opioid misuse and addiction?

In her statement on opioid abuse last month, FDA Commissioner Margaret Hamburg explained that the FDA will continue to work collaboratively with "state and local governments, public health experts, health care professionals, addiction experts, researchers, industry and patient organizations" to help reduce the risks of opioid misuse, abuse, addiction and overdose.

The first step is providing physicians with extensive clinical guidelines and educational opportunities for prescribing opioids. For example, continuing medical education classes are offered by the Substance Abuse and Mental Health Services Administration to make sure that physicians are up to date on the importance of creating individualized patient treatment plans.

Last year, the FDA announced labeling changes and post-market study requirements for opioids in an attempt to begin more accurately prescribing and treating patients requiring opioid pain medications. Labeling changes mark one important step in highlighting the value of physician-to-patient communication and individualized patient pain-management programs.

In addition to providing patients with materials on proper use of opioid medications, patients should also be educated about proper drug disposal. The National Survey on Drug Use and Health found that painkillers used for nonmedical reasons were obtained from friends or family members 70 percent of the time.

Prescription-drug monitoring programs offer another solution. The programs are state databases that make sure patients are filling prescriptions belonging only to them, are taking the right dosages, and are not receiving prescriptions from multiple physicians.

Continued research into drugs that can reverse the symptoms of opioid overdose and ensuring patients have access to naloxone agents such as Evzio will also reduce the occurrence of overdose-related deaths. Mitigating the occurrence of opioid overdose could also save our health care system upward of $70 billion per year.

The FDA took a big step this month in approving a take-home drug that temporarily reverses the effects of opioid overdose. However, the battle over misuse, addiction and overdose is far from over. Health care providers and policymakers need to continue working toward a solution — or the line between the orange bottle in the medicine cabinet and the syringe in a back alley will become increasingly blurred.

Peter Pitts, a former Food and Drug Administration associate commissioner, is president of the Center for Medicine in the Public Interest.  Read More & Comment...

USTR, IPR, and Special 301 Progress

04/30/2014 11:40 PM |

Washington, D.C. – The Office of the United States Trade Representative (USTR) has released its annual “Special 301” Report on the adequacy and effectiveness of U.S. trading partners’ protection and enforcement of intellectual property rights (IPR).  Significant elements of the 2014 Special 301 Report include the following:

•       USTR highlights growing concerns with respect to the environment for IPR protection and enforcement in India and other markets. 

•       The Report expresses ongoing, serious concerns about the protection and enforcement of trade secrets with respect to China, and emerging concerns in other markets.

•       USTR announces that it will conduct Out-of-Cycle reviews to promote engagement and progress on IPR challenges identified in this year’s reviews of India, Kuwait, Paraguay and Spain.

“The United States is an innovation economy.  We are the global leader in research and development.  We have given rise to some of the most creative, inventive and entrepreneurial businesses in the world, contributing significantly to advances in global health, the development of the digital economy and the education and entertainment of billions of people worldwide.  More than 30 million Americans owe their jobs directly to these and other innovative industries.  USTR is fully committed to unlocking opportunity for those Americans to share their inventions and creations with people all over the world without their work being infringed or misappropriated,” said Ambassador Michael Froman. 

“Release of the 2014 Special 301 Report marks 25 years since USTR published the first Special 301 ‘Fact Sheet.’  In that time, we have achieved dramatic changes in the international intellectual property landscape.  The Obama Administration is committed to meaningful and sustained engagement with trading partners -- from China to India to Canada -- with the goal of resolving intellectual property-related concerns so that Americans and American firms can compete on a level playing field in those markets.”

“I would like to congratulate the Governments of Italy and the Philippines on their removal from the Watch List.  Both were named in the first Special 301 Fact Sheet and in many annual reports since, but today we acknowledge their accomplishments and encourage them to continue their progress,” Ambassador Froman concluded.  “Likewise, we congratulate Israel on its removal from the Watch List earlier this year.”

  Read More & Comment...

Baa Humbug

04/30/2014 10:08 AM |

The Times of India quotes Pfizer's senior vice-president for global policy and international public affairs, Justin McCarthy, that "a series of regulatory initiatives and legal challenges have frayed relations between the Indian government and global pharmaceutical firms,” and urged the government to "shift from using destructive IP policy as an access strategy.”

Mr. McCarthy and Pfizer ought to be applauded for bringing to light this important policy issue where the Indian government wants to bury the truth.  However, I would add one nuance to the policy discussion. India’s IP grabs aren’t an access strategy – it’s a doomed effort at domestic industrial policy.

A wolf in sheep’s clothing shouldn’t be called a sheep.

  Read More & Comment...

A House Divided

04/29/2014 08:27 AM |

California may be the poster child for the Medical Home but, when it comes to pharmacy synchronization, it’s a healthcare house divided: the House of hospital pharmacists on the one side, patients, physicians, and community pharmacists on the other.

 

Currently pending in front of the State Assembly is AB2418, legislation that would promote “promotes policies designed to improve patient medication adherence.”

 

Specifically the bill addresses pharmacy synchronization, a tried and true methodology for significantly improving compliance and adherence. The bill is being opposed by hospital pharmacists or better known as the “anti-adherence” organization.

 

Adherence is a problem of behemoth proportions. According to a report in the report conducted by the New England Healthcare Institute, not taking medications as prescribed leads to poorer health, more frequent hospitalization, a higher risk of death and as much as $290 billion annually in increased medical costs. There isn’t any one way to solve the problem. Education? Sure, but that only gets you so far. Apps and other social media interventions? Yes. Phone call reminders from physicians and pharmacists? Absolutely. But, alas, there is no one magic bullet.

 

Pharmacy programs seem to be the best way forward, and there’s hard data to back that up. Case in point – the successful Appointment-Based Model program being used at Thrifty White, a Midwest chain of pharmacies. (For more information on the Thrifty White program, see the article, Adherence and persistence associated with an appointment-based medication synchronization program, from the December 2013 edition of the Journal of the American Pharmacists Association.) Patients enrolled in the program have experienced 3.4 to 6.1 times greater adherence

 

Medication synchronization is a pharmacy service that improves patient adherence to prescribed medications by coordinating the refill dates for all of a patient’s chronic prescription medications so these can be picked up on the same date each month. It is estimated that 76 percent of Americans aged 60 and over use two or more medicines and 37 percent take five or more medicines. Patient and caregiver lives are simplified by eliminating multiple trips to the pharmacy each month. It also minimizes confusion over when a prescription is due to be refilled, and minimizes disrupting treatment through delayed or missed refills.

 

The Appointment-Based Model (ABM) is a voluntary medication synchronization prescription refill program that aims to improve patient adherence as well as the efficiency of the pharmacy operation. The ABM provides enhanced patient access through a streamlined process, opportunity for patient education on medication use, and greater pharmacist oversight to address potential contraindications, duplicate drug therapy, and errors.

 

What outcomes of medication synchronization?

 

·      Improved adherence and time on therapy.

·      Enhanced patient quality of care resulting from better adherence and understanding of medication therapy.

·      Increased patient and pharmacist engagement allowing for greater monitoring and oversight of patient multiple-medication regiments.

·      More efficient pharmacy operations. Medication synchronization has the potential to reduce administrative costs incurred by pharmacists and physicians long term as it streamlines medicine refill processes and helps pharmacies manage their inventories. 

 

The American Medical Association adopted Resolution 801(I-12) in 2013, which encouraged relevant organizations, including insurance companies, to implement prescription refill strategies for patients with multiple prescriptions to reduce travel barriers to access to medicines.

 

The National Community Pharmacists Association (NCPA) and National Association of Chain Drug Stores (NACDS) have endorsed medication synchronization language for states to use when considering legislation. The  American Pharmacists Association (APhA), American Cancer Society, Stroke Foundation, Epilepsy Foundation, American Academy of Pain Managemet and State Pain Policy Network are also educating their members on the benefits of medication synchronization.

 

According to CMS, The estimated cost to Part D sponsors is $0.5million and the savings to Part D sponsors and beneficiaries is $1.8 billion. And there will be an overwhelming reduction in overall health care costs because of improved patient outcomes due to enhanced medication adherence, which should lead to higher quality ratings for a health insurance plan.

 

AB 2418 should be a no-brainer.

  Read More & Comment...

PC Healthcare

04/28/2014 07:08 AM |

The other evening I attended a dinner hosted by National Journal and AstraZeneca on the topic of “patient centricity.” Those around the table were payers, patient advocates, providers, pharmaceutical executives, IT geeks, and policy wonks.

Patient Centricity is a phrase we hear a lot and it’s lost a lot of it’s meaning – kind of like “personalized medicine.” But (in the immortal words of Jim Croce) “It doesn’t have to be that way.” "PC" is the new Healthcare.

In fact, when you think about it, both phrases really mean the same thing – driving positive therapeutic outcomes and, not surprisingly, the assembled diners agreed. After all, payers want (and are willing – mostly) to pay for treatments that work, physicians want treatments that provide the maximum therapeutic benefit, pharmaceutical companies want their products to work as well as they possibly can (so that physicians want to prescribe them to more patients and payers want to pay for them), and patients (last but not least) want to get well as swiftly and completely as possible.

(And, it’s no accident that many in Pharmaland have found religion in the wake of the intense focus on the prices of certain specialty medicines. It is now more urgent than ever to protect “sustainable innovation.”)

The point to remember is that a patient who is diagnosed earlier and treated with the most efficacious treatment as soon as possible is the least expensive over time. Alas, this means we must think long-term – not a trait the healthcare system is known for.

Patient Centricity = Driving Outcomes. Personalized Medicine, the right medicine in the right dose for the right patient at the right time, drives outcomes.  Patient Centricity is Personalized Medicine.

QED.

  Read More & Comment...

Who bilked the AHRQ?

04/25/2014 07:04 AM |

One day before her resignation, HHS Secretary Kathleen Sebelius sent a letter to Congressman Bill Cassidy (R, LA). One of the subjects was academic detailing.

According to Secretary Sebelius:

AHRQ no longer conducts an academic detailing program as of September 2013.

But what happened to tax dollars already spent?

In October 2010, AHRQ awarded five grants for a program on academic detailing and the “communication of CER results to physicians”.  This program to disseminate CER findings is supported by $29.5 million from the American Recovery and Reinvestment Act (aka, “the stimulus package”).

* One contract, for $11.7 million, went to Total Therapeutic Management (TTM) and is specifically intended for physician outreach and education

The goal of this contract was to integrate AHRQ’s comparative effectiveness research, products, and tools into clinical practice through 9,000 on-site, face-to-face visits with clinicians, nurses, health plan formularies, benefit managers, and other healthcare professionals. 

According to TTM’s Barry Patel, “the AHRQ AD project to disseminate CER findings was a 3 year program that was scheduled to end in September 30th. The project has been over since Sept 30th 2013 as scheduled.”  

Other recipient’s of the $29.5 million were:

* Ogilvy Public Relations Worldwide, Healthcare Division: $18 million to create a publicity center and another contract for $8.6 to create regional dissemination centers.

* Prime Education (an educational design and accreditation company focused on continuing medical education programs): $4 million as a “continuing education award.”

* IMPAQ International (a social science research and consulting firm that specializes in impact evaluations for a client base of predominantly U.S. government agencies): $2.4 million to evaluate the impact of the other four contracts.

Maybe it’s time for Congressman Paul Ryan to call a hearing to discuss the, um, outcomes of that $25.9 million.

  Read More & Comment...

Pain Politics

04/24/2014 07:01 AM |

Many of the claims about Zohydro are incorrect, and efforts to trump FDA's approval threaten the integrity of science-based regulation, FDA Commissioner Margaret Hamburg said in an interview with Steve Usdin of BioCentury This Week television. Edited excerpts follow.

BCTV: Why did FDA approve the drug?

Margaret Hamburg: I certainly understand the broader concerns that are motivating some of this in terms of the terrible public health burden of opiate addiction, abuse, misuse and overdose. But there is a need for adequate pain treatment of patients. And Zohydro, in fact, does represent an important pain medicine for people that respond better to hydrocodone - or if you need to rotate pain medicines because of chronic use - who don't want to be exposed to acetaminophen, which is a leading cause of liver toxicity, often even fatal liver toxicity.

And all of the other hydrocodone products currently in the marketplace are combined with acetaminophen.

BCTV: The governor of Massachusetts and several members of Congress have said Zohydro is superpotent and more addictive than other opioids.

MH: One of the common misperceptions is that this is the most potent opiate out there. And that simply isn't true. The second is that the other opiates out there have abuse deterrent formulations. And we wish that that were so. But in fact, almost without exception, the abuse deterrent claims of some of the products really don't hold water when tested.

And the one abuse deterrent formulation that has met the FDA criteria for labeling as abuse deterrent actually only provides deterrence against immediate injection or nasal intake - snorting. But it does not prevent oral abuse. And the majority of opiate overdose and abuse is through the oral route, not injection.

BCTV: Is there any deterrent now or even conceivable that's going to prevent somebody who's determined to abuse an opioid from doing that?

MH: Certainly not in terms of the formulation of the drug. There is nothing that can, at the present time, deter a determined abuser.

BCTV: Massachusetts and Vermont have tried to ban Zohydro. Other states are talking about it. What are the consequences of having states take that unprecedented action of overruling FDA approval?

MH: I do think that it's quite troubling. And I understand the motivation behind some of these actions in terms of the pressing need to reduce the burden of opiate addiction and preventable disease and death. But I think people should step back and really think very carefully about what it means for states or the Congress to start dictating what drugs should be approved and which ones should be withdrawn from the marketplace.

You can easily look at scenarios that are increasingly worrisome where, because of ideological beliefs or dislike for certain patient categories or other criteria, drugs that could make a difference in people's lives get banned.

BCTV: Some members of Congress have written to the HHS secretary and asked her to overrule FDA's decision. What happens if we have a political system where political appointees can overrule FDA on decisions that members of Congress don't like?

MH: I think it's very worrisome. FDA has a very carefully defined legal regulatory framework for our decision-making. Most importantly, we are driven by the science, and there is a set of scientific database standards for product review and approval. When individuals who have very different backgrounds and are in the political environment, not the scientific environment, start making the decisions for the public, you can end up in some very worrisome places.

The full video interview is available at www.BioCenturytv.com.

  Read More & Comment...

Duchenne Muscular Dystrophthe and the Special Medical Use Super Highway

04/22/2014 08:27 AM |

By now you’ve surely seen the news (the good news) about Sarepta Therapeutics and it’s investigational drug for Duchenne muscular dystrophthe. The FDA (in a reversal of it’s original position) has indicated a willingness to consider the treatment for accelerated approval.

Much made of the high-pressure tactics employed by patient groups. But, if this decision is viewed as “the uncaring FDA caving in to desperate patients,” we’re being unfair to the agency, misrepresenting the intellectual potency of the patient voice, and missing a very important nuance. 

In it’s Page One coverage of this story the Washington Post, after much FDA bashing (and towards the very end of the article), writes,

Not everyone faults the FDA for taking so long to decide how to proceed. Eric Hoffman, director of the Center for Genetic Medicine Research at Children’s National Medical Center and a longtime Duchenne researcher, said valid questions remain about eteplirsen’s dystrophin-producing abilities and how effective Sarepta’s drug really is.

“I’ve personally been very impressed with the FDA,” he said. “There’s every indication they are taking this extremely seriously.”

Hoffman said he understands the families’ sense of urgency, and he shares optimism about exon skipping. But he said the public campaign to pressure the FDA has divided the community more than united it.

“It becomes this form of bullying that puts everybody between a rock and hard place,” he said. “Unless you’re supporting kicking out the head of the FDA and granting accelerated approval as quickly as possible, then you’re supporting killing Duchenne kids.”

He said that the reality is more nuanced and that FDA reviewers do want to help patients but are wary about setting a precedent by approving high-priced drugs they aren’t yet convinced will work.

Precisely.

The Duchenne muscular dystrophthe case also serves as an important example as to why the FDA’s nascent Special Medical Use program shouldn’t be limited to anti-biotics/anti-infectives. There’s much work to be done – and the more options that are open to bring important new therapies to patients the better.

Accelerated Approval is good – but, as a recent blog from Context Matters points out, the FDA’s previously established programs to expedite therapies (including Fast Track, Accelerated Approval, and Priority Review) have not produced lasting improvements in approval time. “Based on our preliminary analysis, in 2008 the average cycle time for ‘Priority’ was 10.1 months, versus the ‘Standard’ which was 21.2 months. In 2011, the average cycle time for ‘Priority’ was 19.5 months, versus ‘Standard’ which was 17.5 months,” the blog authors wrote. “What’s interesting here is that over time the fast lane has apparently become just another standard lane; in 2011 it was actually even slower.”

What’s standing in the way of the FDA Special Medical Use Super Highway?

The FDA needs to have the proper infrastructure and support to successfully reduce approval times. Clearly, when you’re looking at products that have less data behind them, you need more senior people who can devote greater resources to studying them, and that also takes time away from other programs that the agent needs to review. It’s one thing to give the FDA more authority, it’s another thing to give the FDA greater responsibility, but if you don’t give them the resources to get it done, to a large degree it is just rhetoric.

And there’s more than enough of that to go around.

  Read More & Comment...

Weber on Apps for Adherence

04/21/2014 10:27 AM |

Dr. Michael Weber, hotshot cardiologist, Chairman of the Center for Medicine in the Public Interest, and all-around good guy weighs in on medication adherence and the value of apps.

The AP reports:

Medicine only helps if you take it properly. And adhering to an exact schedule of what to take, and when, can be challenging for patients who are forgetful or need to take several medications.

Doctors warn about the consequences and urge patients to use various techniques, such as using divided pill boxes or putting their pill bottles beside their toothbrush as a reminder to take their morning and bedtime medicines.

Still, only about half of patients take medication as prescribed, resulting in unnecessary hospital admissions and ER visits that cost the U.S. health care system an estimated $290 billion a year.

To help combat the problem, many doctors are trying a more high-tech approach: They're recommending smartphone apps that send reminders to patients to take their medications and record when they take each one.

"I think it's going to become pretty standard" for doctors to recommend them, said Dr. Michael A. Weber, a cardiologist at SUNY Downstate Medical Center. Weber began recommending apps to patients a few months ago and already has seen better lab results from a few using them.

"Some people say, 'That's a great idea,'" Weber said. "Even ones who claim they're conscientious, like the reminders."

He said the apps are particularly helpful for patients with symptomless conditions, such as high blood pressure or high cholesterol. Those patients are less likely to regularly take their medications than someone with pain or an infection.

"I don't think they're going to change the world," Weber said, though he recognizes benefit of apps. Even so, he said smartphone apps won't do much to help people who simply don't like taking medicine, fear side effects or can't afford their prescriptions.

The full AP story can be found here.

  Read More & Comment...

Admitted Mistake

04/21/2014 07:12 AM |

In a March 25, 2014 tweet and blog post, I inaccurately referred to Gabriel Levitt as an “admitted felon” and to his business, PharmacyChecker.com, as having “felonious business interests.” Mr. Levitt’s attorney has assured me that Mr. Levitt has never been charged with or convicted of a felony, nor has PharmacyChecker.com. Per Mr. Levitt’s attorney, PharmacyChecker.com provides pharmacy verification and drug price comparisons.

  Read More & Comment...

The Dangers of QALY-Fication

04/18/2014 09:30 AM |

In today’s New York Times, Andrew Pollack reports:

Saying they can no longer ignore the rising prices of health care, some of the most influential medical groups in the nation are recommending that doctors weigh the costs, not just the effectiveness of treatments, as they make decisions about patient care.

And further:

In practical terms, new guidelines being developed by the medical groups could result in doctors choosing one drug over another for cost reasons or even deciding that a particular treatment — at the end of life, for example — is too expensive. In the extreme, some critics have said that making treatment decisions based on cost is a form of rationing.

This is an urgent discussion that has been going on for a while minus any real imput from providers. It’s good to have them join the conversation. But here’s the bad news:

The cardiology societies, for instance … plan to rate the value of treatments based on the cost per quality-adjusted life-year, or QALY — a method used in Britain and by many health economists.

It’s important to point out that NICE in England (not Britain – that’s an important distinction) has publicly stated that it is moving away from using QALY-based reimbursement decisions, moving to a value-based insurance design (VBID) strategy.

Here’s the important difference – QALY is based on cost, VBID is based on outcomes.

Everyone should welcome physicians to the crucial discussion over reimbursement. But the focus should be on paying for what works, not for what’s cheapest.

As for an open and honest debate over the costs/benefit of end-of-life care – it’s important, but politically problematic.

  Read More & Comment...