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.

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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.

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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.”

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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Are free samples only skin deep?

04/17/2014 09:37 AM |

According to a new study published in JAMA Dermatology, dermatologists who receive free drug samples are more likely to give their patients prescriptions for expensive medicines.

Not surprisingly, that results in headlines such as “Free samples of prescription drugs are costly to patients, study says” (Los Angeles Times) and “Doctor’s free samples have a hidden cost” (NBC News). Sexy, but is that the real story?

The study reports that, for a single visit, the average retail cost of prescriptions for patients whose doctors received free samples from drug makers was about $465, compared with about $200 for patients whose doctors did not receive free samples.

What the study does not report (because they did not collect the data) was whether the free samples were the more therapeutically appropriate choice.

The reporting on the article (various news media) makes it sound as though generic and brand name drugs that treat the same condition are identical (and equally appropriate and efficacious) to each other. Not so. But why let important facts get in the way of a good story.

Most nauseating is the following study factoid:

In contrast, such drugs (sampled brand name drugs) only accounted for 17 percent of prescriptions written by doctors at an academic medical center that doesn't allow its doctors to accept free drug samples.

Well, duh. No samples in, no samples out. (Not to mention the fact that academic medical centers often dictate narrow formulary choices. Not surprisingly, that fact wasn’t mentioned in the study) It would also be interesting to match up patient outcomes of those derms who dispense samples vs. their ivory tower colleagues. Will any of these additional data sets be included in a follow-up study? Inquiring minds want to know.

The full study can be found here.

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Ad Comity

04/16/2014 11:25 AM |

As the saying goes, everything you read about in the news is true –except for those things you know personally. Case in point: coverage of the FDA’s approval of the pain medicine, Zohydro.

Yesterday, U.S. District Court Judge Rya Zobel granted a request by a drug manufacturer Zogenix for an injunction temporarily halting Gov. Deval Patrick’s attempt to ban the prescribing of the painkiller Zohydro.

Massachusetts Governor Deal Patrick and others who want to ban Zohydro argue that the FDA Advisory Committee voted against approval.

True, but not necessarily accurate.

At an FDA advisory committee, the agency is asked to defend its scientific thinking in public, before a panel of experts who can dissect results, challenge conclusions, and ensure no clinical stone goes unturned.  Seldom reported, however, is that advisory committee votes are recommendations. They aren’t binding on the FDA.

An analysis of advisory committee recommendations compared to agency actions shows FDA followed committee advice 74% of the time. Interestingly, the agency overruled “no” votes only three times: (Tarceva for maintenance therapy in lung cancer, Avastin for breast cancer, and Micardis to lower blood pressure.) Since their approval, these medicines have saved, extended, and improved hundreds of thousands of lives.

So, what about the Zohydro decision? The soundbite you’ve likely heard is that the vote was against approval of the drug. That’s true. What you probably don’t know is that, by a vote of 11-2, the experts affirmed that there was no evidence to suggest Zohydro had greater abuse or addiction potential than any other opioid.

When the committee voted, Dr. Bob Rappaport, Director of the FDA’s Division of Anesthesia, Analgesia, and Addiction, asked members to explain their votes. All but two said that while Zohydro had met their requirements for approval, their votes were meant to call greater attention to the agency’s regulation of opioids in general – not Zohydro specifically.

The FDA decided to approve Zohydro based on the agency’s judgment (and the advisory committee’s concordance) that the medicine is safe and effective. But the FDA also heeded the expert panel’s advice for better post-approval regulation of opioids.  Shortly before Zohydro’s approval, the agency strengthened opioid labeling and post marketing requirements to address the concerns raised by the advisory committee.

A report by the Institute of Medicine found that 100 million Americans now live with chronic pain. That’s a third of the U.S. population. Ten million of those have pain so severe they are disabled by the pain.

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D-structive

04/15/2014 07:01 AM |

As many as 36 million Americans narrowly dodged a recent proposal from the Centers for Medicare and Medicaid Services (CMS) that would have advanced unprecedented government intrusion within the Medicare Part D prescription drug program.

CMS never offered a satisfactory reason for its proposed tinkering with something that's working. Part D is a real rarity -- a genuinely popular and cost-effective federal health program. The proposed reforms were not only unnecessary; they would have compromised affordability, limited treatment options for patients, and stifled innovation in the insurance industry.

Though the misguided proposal has been shelved, Part D is still at risk. The Obama administration has pledged to push through a similar plan in the near future.

By all accounts, Medicare Part D is overwhelmingly successful. The program offers drug coverage to seniors for a monthly average of $31 and enjoys a 90 percent approval rating among beneficiaries.

Unlike other healthcare benefits, Part D is also cost-effective for taxpayers. The nonpartisan Congressional Budget Office (CBO) reports expenditures are now 45 percent below original cost projections.

And because Part D provides access to vital medicines, the program helps seniors avoid expensive stays in hospitals and nursing homes. Over half of beneficiaries report they would be "more likely to cut back or stop taking medicine altogether" without Part D coverage. In this way, Part D saves our healthcare system $12 billion annually, according to a study in the Journal of the American Medical Association.

In light of the program's remarkable track record, it's inexplicable why CMS would attempt to tamper with important aspects of Part D.

As part of the proposed changes to Part D, CMS wanted to eliminate rules that provide "protected class" status to mental health drugs and drugs used for autoimmune diseases and organ transplant patients.

If the reform had taken effect, private plan providers may no longer have covered many of the medicines included in these designated categories. Countless Part D beneficiaries would have lost access to the drugs they were currently receiving and need to stay healthy.

CMS claimed that the proposal would lower prices by giving private plan providers better negotiating leverage with pharmaceutical companies. But that rationale ignores the fact that costs for these drugs have already been falling for years. From 2006 to 2010, in fact, prices for protected-class drugs dropped by two percent, despite an explosion in overall healthcare costs.

The idea that federal regulators can intervene to help negotiate better prices is dubious at best. And CMS didn't offer any quantitative evidence that restricting vital medications would benefit either seniors or the federal government.

Indeed, Part D works specifically because it encourages open competition between private insurers who are eager to serve Medicare's huge prescription drug market.

Nevertheless, federal regulators sought to reinterpret certain parts of Part D's "non-interference clause" -- the part of the law that forbids regulators from distorting the market by intervening in drug price negotiations. The proposed changes would have allowed the government access to all agreements struck by drug manufacturers, pharmacies, and insurers.

The new rules would have also limited the number of bids an insurance plan may offer in a region to two. By reducing the number of plans available, CMS would have weakened competition between insurers while also preventing plan providers from experimenting with new policies. Uncle Sam wants to reduce competition? What would the Federal Trade Commission have to say about that?

On top of all that, the agency suggested changes that would encourage nearly every employer who offers health insurance for retirees to drop prescription drug coverage.

There was one common thread running through these proposed changes to Part D: they all restricted choice and discouraged competition. The Obama administration, it seems, isn't content with a healthcare entitlement that relies on market forces -- no matter how cost-effective or popular the program may be.

When the Obama team shelved its plan, CMS Administrator Marilyn Tavenner only promised to pull it only "at this time." In fact, she committed to "advancing some or all of the changes in these areas in future years." So Part D isn't yet safe. In the future, if regulators want to sabotage the most successful federal health program in the country, the least they could do is explain why.

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Pharmitzvah 2014

04/14/2014 07:00 AM |

Last week’s 14^th annual meeting of Pharmaceutical Research and Manufacturers of America (PhRMA) was a memorable pharmitzvah. Smart folks, old friends, challenging panels, and a bevy of good speeches on challenging topics.

And while there was the appropriate serving of rah-rah, there was significant substance alongside the Beltway style.

This was best summed up by Ian Read (Chairman and CEO of Pfizer) in his inaugural remarks as the incoming Chairman of PhRMA. It wasn’t a Morning in America oration. Read shared his concern about the industry’s failure in getting the message out about “the value we generate.” His key message, “We need to fix the misperception gap.”

Specifically he talked about the industry’s need to broaden the conversation from the economic performance of biopharmaceutical companies to the value that accrues to society and called for a “dialogue with society.” Bravo.

He asked, “Where are the headlines?” They’re not about societal value – and they need to be. There’s a strong story to tell. It’s not happening. And it needs to, because minus that narrative, nothing the industry wants to make happen (with government being a focus since the meeting was in Washington, DC) will be possible.

Read called for “industry speaking for itself.” After all, if you can’t be your own best advocate, you’re suspect in the minds of many – and rightfully so. He spoke to “better ideas and clarity” versus “more tactics.” That’s a foundational shift and a timely one. Innovators win when the discussion is about the future.

Read called for transparency in data sharing and physician relationships. Doing it is one thing – and most members of the pharmaceutical brethren are doing the right thing. But it’s equally important to tell the industy’s various constituencies what programs are in place, what they are designed to do, and how they work. Transparency mustn’t be allowed to be a cudgel used to beat innovators but a tool in it’s own communications arsenal.

Value to society. It was Read’s mantra. It needs to be the industry’s manifesto.

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Kathleen Sebelius

04/11/2014 06:52 AM |

Mission Accomplished?  Read More & Comment...

Don't politicize FDA decisions on pain medications

04/10/2014 08:46 AM |

Our elected officials should act to protect medical progress in chronic pain relief. One hundred million people in the United States deal with debilitating chronic pain and need access to approved medications every day to stay productive and attain some measure of quality of life.

We must protect medical access for those afflicted with the most severe pain
The FDA did its job correctly by approving a unique treatment option based on its safety and efficacy data.

Do not let politicians overturn the FDA’s legitimate and expert approval of a pain medicine option that can bring relief.  Do not infringe on patient access to approved pain medication options in any State. Help us send a message to Washington and state capitals across the nation to do the right thing.

Stand up and show your support for people with chronic pain.  Please sign (and share) our petition today.

The on-line petition can be found here.

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Expedited Reviews? Context Matters

04/09/2014 06:55 AM |

I was recently interviewed by Context Matters on Expedited Reviews and the Current State of Drug Development. Here’s Part One of the conversation

Regardless of what you want to call it, whether it’s expedited review or special protocols, limited use medicines, the theory is, “How can the FDA help bring important therapies to the market faster?” And, there are a lot of pieces to that puzzle.

At the end of the day, the agency still has to meet with the sponsor, who still has to have data. They have to meet and decide how to move forward.

There are two issues that also have to come into this conversation:

1. The patient voice
2. Benefit / risk profile

Those are two things that really are not usually discussed. Generally speaking, in this type of conversation what comes about in the discussion is time. How quickly can something happen? Obviously faster is better than slower, but you don’t want to ever sacrifice proper review.

Blood From a Stone: Expedited or Standard, It’s All the Same FDA Resources

A key issue not being widely talked about is that the FDA isn’t hiring a whole new team to deal with expedited reviews. It’s the same people. So does an expedited review take precedence over a non-expedited review? How does the agency determine who gets the resources at what point in time?

Obviously, if you’re going to file a regular old-style NDA, you get a PDUFA (Prescription Drug User Fee Act) date and the agency is responsible for that. How do you weigh that against “critical medical need”?

The answer is it’s on a case-by-case basis. You can’t say, “Hey, we’re not going to make the PDUFA date because we’re reviewing someone else’s drug,” that’s a non-starter. You have to view expedited review situations in the broader context of everything else the agency has to do.

And then the agency is then going to define (on a case-by-case basis) what it feels is most important to the public health. Here’s an example: MS (multiple sclerosis) or IPF (Idiopathic pulmonary fibrosis) – Are either of those more or less important from an expedited review / resources perspective than a new anti-infective? It depends who you talk to. If MRSA issues are on the top of your agenda, then it’s one thing. If it’s orphan diseases, it’s another or if it’s pain-mitigation it’s something else again. Where you stand on these issues depends on where you sit.

I don’t really think having people specifically tasked to working on expedited reviews is the way to go, unless you have more people. And then the question is, where do they come from and do you take the best and brightest away from standard review, to put them there? How do you weigh the general importance of staff time and agency resources? Do you want it to be for a disease that is not an orphan disease that’s an important therapy, or do you want it to be on an orphan disease that can have a profound difference on people’s lives? Those are really philosophical questions, but it comes down to resources. I don’t think you can have dedicated teams when you don’t have people that can dedicate their time exclusively, otherwise it is just rhetoric. It’s just another committee on which the same people sit.

Comparing the Agencies

What might be interesting (and is generally not part of this conversation) is a move towards greater FDA / EMA harmonization on certain disease categories. There’s been a lot of chatter on this issue, but at the end of the day nothing really happens. I think the EMA would be interested in that, probably more than the FDA, because one of the main differences is the type of data accepted by the EMA. The EMA casts a wider net in terms of the types of data they accept for any given application.

One of the reasons the EMA has gone beyond the FDA is that their national economies are so much more financially strapped than we are here. It is important to remember that EMA decisions are only the first part of gaining market access. After the EMA, drugs go on to be evaluated by different country Health Technology Assessment (HTA) agencies to determine reimbursement and pricing in various markets. And from a reimbursement perspective, the HTA agencies are moving away from the QALY (quality-adjusted life year) towards a more value-based insurance design.

From a review proposition, from the FDA’s perspective, obviously they don’t get involved in cost issues, only clinical issues. I think what innovator companies are going to find out (especially with the expedited review pathways) is that the risk of getting a drug yanked quicker (meeting a quick death) rises. So they are going to have to understand, from a business perspective, the risk of asking the FDA for these special medical use reviews – it could backfire unless you understand what you are asking the FDA to do. The FDA is trying to signal that there are multiple ways to get an important new therapy to market quicker, but that has to be based on a public health need and not on a marketing strategy.

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