Latest Drugwonks' Blog
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 21st 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 20th 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 21st 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.
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.
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.
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.
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.”
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.
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.
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.
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.
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.
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
