Some snippets from the National Council on Patient Information and Education’s report, Accelerating Progress in Prescription Medicine Adherence: The Adherence Action Agenda: A National Action Plan to Address America’s “Other Drug Problem”

At the same time that medical science has transformed HIV and many cancers into treatable conditions and significantly reduced the burden of chronic diseases like diabetes, many Americans are not benefiting from these treatment advances due to the persistent problem of poor prescription medicine adherence—a pervasive problem the leads to unnecessary disease progression, disease complications, a lower quality of life, preventable deaths, avoidable medical spending and lost work productivity.

Based on the latest estimates, half of the estimated 187 million Americans who take one or more prescription medicines—or up to 93.5 million patients—do not take these drugs as prescribed. In fact, studies show that 20% to 30% of prescriptions are never filled by patients, while 50%–60% of medications to treat chronic disease are not taken as prescribed. In terms of the toll in morbidity and mortality, lack of medication adherence is associated with poorer health outcomes, resulting in approximately 125,000 preventable deaths a year and many as 40% of nursing home admissions in people with type 2 diabetes. From the standpoint of the cost to the economy, new research estimates that $105 billion is wasted annually on medication therapy nonadherence of which 69%—or $72.5 billion—is spent on hospitalizations. Other findings project that poor medicine adherence, along with suboptimal prescribing, drug administration, and diagnosis, costs the health care system an estimated $290 billion per year in avoidable medical spending and lost work productivity, translating into 13 percent of total health care expenditures.

The future state of medication adherence will be affected by the move towards a patient-centered health care system through implementation of the Patient Protection and Affordable Care Act (ACA), including efforts to reduce avoidable hospital readmissions, which is driving innovative approaches to medication management.

A NEW ADHERENCE ACTION AGENDA

NCPIE’s Adherence Action Agenda advocates for an increased focus on the overlooked challenge of multiple chronic conditions, where the need for patient adherence is most acute, and lays out these ten policy and programmatic solutions to improve medication adherence:

1.              Establish medicine adherence as a priority goal of all federal and state efforts designed to reduce the burden of multiple chronic conditions. Because patient adherence is not viewed as an essential element of government initiatives to reduce the burden of multiple chronic conditions, the report calls for adherence to be integrated throughout the range of efforts now underway through a new HHS Multiple Conditions Strategic Framework to improve health systems change and facilitate new research efforts.

2.              Establish the role of the patient navigator within the care team to help patients with multiple chronic conditions navigate the health care system and take their prescription medicines as prescribed. Building on the patient navigator model now used in hospitals and cancer clinics nationwide, the action plan advocates for pairing patients treated for multiple chronic conditions with specially trained adherence navigators who will, in collaboration with patients and caregivers, obtain the patient’s medical records, create an accurate medication list, set up medication counseling as needed, schedule timely follow-up physician visits, and facilitate communication between the patient and his or her different physicians.

3.              Promote clinical management approaches that are tailored to the specific needs and circumstances of individuals with multiple chronic conditions. Since patients with multiple chronic conditions differ in the severity of their illnesses, prognosis, and functional status, the report encourages health professionals to adopt the American Geriatric Society’s guiding principles for treating older adults with three or more diseases, which calls for eliciting and incorporating patient preferences and choosing therapies that optimize benefits and minimize the harm for older patients.

4.              Incentivize the entire health care system to incorporate adherence education and medication support as part of routine care for MCC patients. With research showing that the interactions between patients and their healthcare providers affect how well patients manage their chronic conditions, the report advocates for an expanded investment in patient/provider education and engagement tools to help clinicians implement best practices for medication adherence and counsel their patients on the importance of following treatment plans.

5.              Eliminate the barriers that impede the ability of patients with multiple chronic conditions to refill their prescription medicines. One of the reasons patients fail to refill their prescriptions is the need to pick up prescriptions at different times and sometimes at different pharmacies, requiring numerous trips. T o reduce these obstacles, stakeholders support implementing the “pharmacy home” model, which gives patients a single pharmacy point of contact for filling prescriptions, and adopting refill synchronization, which allows patients to fill different prescriptions at one time and therefore, reduce the number of visits they must make to the pharmacy.

6.              Reduce the cost-sharing barriers for patients by lowering or eliminating patient copayments for prescription medicines used to treat the most common chronic diseases. The report makes clear that the cost of medications for some patients is a barrier to filling their prescriptions and taking their medicines as prescribed and advocates adopting policies that will reduce the out-of-pocket costs for medications, especially for patients on multiple prescriptions for chronic conditions.

7.               Accelerate the adoption of new health information technologies that promote medication adherence. Because significant innovations in health technology have the potential to improve the flow of timely and complete information on medicine use between patients and providers, the report calls for the swift adoption of new standards for using electronic health records, incentivizing providers to use health information technology to identify patients at risk for medication misuse, and the expanded use of electronic reminders and personal health records to improve medication adherence.

8.              Establish medication adherence as a measure for the accreditation of healthcare professional educational programs. Currently, the nation’s medical residency programs are moving towards an outcomes-based accreditation system, where medical residents will be evaluated on the basis of required core competencies, including interpersonal skills and communication. From the standpoint of medication adherence, this represents an opportunity to integrate medication management and e-prescribing into the curriculum of medical residency programs and paves the way for establishing medicine adherence skills as core competencies within the curricula of schools of pharmacy, nursing, and other allied health professions and as an accreditation measure.

9.              Address multiple chronic conditions and optimal medication management approaches in treatment guidelines. Clinical practice guidelines typically focus on managing a specific chronic condition and do not take into account the presence of multiple chronic conditions. The report advocates the accelerated development of updated treatment guidelines where information is included on the most common comorbidities clustering with the incident chronic condition, this can start with the most common combinations of multiple chronic conditions, called dyads and triads, which have already been identified by the Centers for Medicare and Medicaid Services (CMS).

10.          Stimulate rigorous research on treating people with multiple chronic conditions, including focused research on medication adherence to promote the safe and appropriate use of different medicines in this patient population. There is a paucity of evidence-based data on how to treat patients with two or more concurrent diseases who are taking drugs developed and tested in people who have a single condition. Accordingly, the report supports incorporating medicine adherence throughout the research agenda for multiple chronic conditions and advocates for increasing the budget for HHS research efforts examining the best ways to treat the most prevalent clusters of concurrent diseases. 

Because the stakes are so high, the report lays out a new Adherence Action Agenda to coalesce stakeholders around the common goal of improving patient adherence to reduce the burden of chronic disease. Ultimately involving the support and active participation of many constituencies—the federal government, state and local government agencies, professional societies and healthcare practitioners, health educators and patient advocates—it is hoped that this report will serve as a catalyst for action and provide a blueprint for accelerating progress.

This is thoughtful and actionable plan and the complete report is worth a careful read and candid debate.

From the pages of the San Jose Mercury News ...

Antibiotics in animals: In food safety, a noteworthy policy consensus

By Peter J. Pitts

It's always a surprise -- and therefore newsworthy -- when opposing groups in Washington, D.C., find common ground and a policy moves forward at the national level.

That's what happened recently when the Food and Drug Administration published documents implementing its policy that medically important antibiotics should only be used when there's a disease or a disease threat. As a result, "growth promotion" uses will be phased out over the next three years. An accompanying rule will require a veterinarian to oversee the use of medically important antibiotics in feed.

The policy ensures antibiotics that are similar to those used in humans will be used in animals in the same way: to address a specific disease or disease threat, and only under the supervision of a licensed medical professional. It affects farms nationally, making state-based efforts -- including those in California -- unnecessary.

This collaborative effort is noteworthy for animals, veterinarians and the millions of Americans who depend on those animals for food supply.

The FDA and animal health representatives share broad agreement on this national position. Animal health organizations, along with the companies that develop animal antibiotic medicines, have supported the policy since its inception and announcement in 2012. Consumer organizations that have criticized the use of antibiotics in agriculture asked for this policy in a letter to the White House in 2009 and supported the FDA's announcement.

While some have criticized the policy as being voluntary, the fact is that the FDA has succeeded because it pursued its agenda in a collaborative way. The agency met with everyone involved, including farmers and ranchers, the pharmaceutical industry and consumer groups, to understand and address concerns.

As a result of this collaboration, the agency has enacted change more quickly than with a regulatory or legislative approach. On the day the agency released the documents, the two largest companies selling these products publicly announced their support and cooperation. Other companies have 90 days to make their intentions known.

The other benefit of this collaborative approach is that it should avoid the unintended consequences that resulted when Europe legislated a ban on growth promotion uses of antibiotics. That ban resulted in increased animal disease and death. The FDA's collaborative approach gives farmers and ranchers the chance to adjust to these changes more gradually and avoid these negative consequences.

The policy is a significant change in the way antibiotics are used to keep food animals healthy. It is unfortunate that many seem to think that antibiotics are used only "to fatten animals." That's not true, but now no medically important antibiotics will be used to promote growth. In addition, no antibiotic will be used in feed unless a licensed veterinarian verifies that it is needed to treat or prevent a disease.

Consumers should be heartened by this development. While eliminating what the FDA believes to be unnecessary uses of antibiotics, the limited and important uses needed to protect animal health with continue.

That's important, because animals get sick -- just like humans do.

Farmers and veterinarians work hard to prevent disease and avoid the use of medicines to treat disease. We all know that there's a nexus between animal health and human health, and the food supply is one of the key areas of that nexus. Farmers and veterinarians need a variety of tools to keep animals healthy because healthy animals help produce safer food.

If someday it should happen that draft guidance must be sound                                              
I’ve got a little list. I’ve got a little list                                                                            
Of social media platforms where user content can be found                                   
And if a product’s disssed it never will be missed.

Some further thoughts on the FDA’s latest social media operetta, the draft guidance entitled, “Fulfilling Regulatory Requirements for Postmarketing Submissions of Interactive Promotional Media for Prescription Human and Animal Drugs and Biologics” – or as fans of Gilbert & Sullivan might prefer to call it, “Patience.”

The real nugget is to be found between lines 188-193:

However, a firm generally is not responsible for UGC that is truly independent of the firm (i.e., is not produced by, or on behalf of, or prompted by the firm in any particular). FDA will not ordinarily view UGC on firm-owned or firm-controlled venues such as blogs, message boards, and chat rooms as promotional content on behalf of the firm as long as the user has no affiliation with the firm and the firm had no influence on the UGC.

In it’s September 21, 2009 Federal Register notice (the one that announced the now famous November 12, 2009 Part 15 hearing), the FDA asked about the issue of property owner vs. property user and user-generated content more broadly:

When should third-party discussions be treated as being performed by, or on behalf of, the companies that market the product, as opposed to being performed independent of the influence of the companies marketing the products?

As I testified at the hearing, “Would letters to the editor be liable for an FDA warning letter? What about radio call-in comments? What about freedom of speech? Relative to intended to promote -- how can this be differentiated from intended to share and educate? And whose job is it to define such differentiation? As Don Draper said, I'm enjoying the story so far, but I have a feeling it's not going to end well.”

(For my complete Part 15 testimony, see here.)

Four plus years later, the property owner vs. property question is asked and answered. So far so good on the UGC front. Better late then never.

Now the question is, does regulated industry really want uncontrolled, unfiltered, and unpredictable user-generated comment on their sites? Because, let’s be honest, it ain’t all gonna be pretty. Is Big Pharma ready to mix it up in real time with real people?

On another note, there’s a peculiar little codicil in the draft guidance that appears on lines 246-249, to wit:

Once every month, a firm should submit an updated listing of all non-restricted sites for which it is responsible or in which it remains an active participant and that include interactive or real-time communications. Firms need not submit screenshots or other visual representations of the actual interactive or real- time communications with the monthly updates.

Hugely cumbersome? To be sure.  But what’s really troubling is the good folks at OPDP think such a “running list” is even plausible. Do they really think that regulated healthcare companies are centralized to such a degree that any one person or department knows the full extent of social media participation? And even if this was the case, is this information really any business of the FDA? Are companies currently required to submit their media plans along with creative for agency review – on a running basis no less?

And just who is going to review these lists? What are the qualifications of such reviewers? Since OPDP isn’t hiring, where in the review queue will these lists reside? Will they be made public? This is mission creep extraordinaire. Danger, Will Robinson. Danger.

Like Old Man River, social media keeps on rolling along with or without FDA guidance (draft, bottled, or otherwise). List making isn’t going to limit it. And no amount of hoping/wishing/praying is going to make it static. Social media jes’ keeps on rolling along.

And regulated industry jes’ keeps falling further and further behind the curve.

How can the FDA help to facilitate, encourage, and expedite more activity on the part of regulated industry? After all, as Janet Woodcock has said, “Social media is where the people are.”

The answer isn’t “more process.” It's more prowess.

And maybe it's time for another Part 15 hearing.

Per a press release, Med Ad News (www.medadnews.com) will return “under new parent company Outcomes LLC, founded by Med Ad News Director, Daniel Becker. Many familiar faces will continue playing a critical role in both the production of the publication and the awards ceremony itself. Med Ad News continues the 30 year tradition of the highly anticipated 25th Anniversary Manny Awards along with the April release of the Annual Healthcare Communications Agency Issue.”

"Despite the growing uncertainty and hype surrounding the future of pharma B2B media, these past few years, Med Ad News and the Manny Awards had its best year ever. Outcomes LLC was developed to continue this momentum and is dedicated to providing our customers with access to a valuable suite of integrated marketing opportunities, events, and content marketing services. We have nothing but hope and optimism for the New Year and future and look forward to continuing to serve the pharmaceutical market with the forward-looking expertise and industry professionalism our customers, both readers and partners, have come to rely on," said Founder, Brand Director, Daniel Becker.

And editor Chris Truelove too!

FDA won't hold companies responsible for independent user content on their social media properties

By Virgil Dickson

The Food and Drug Administration won't hold pharmaceutical manufacturers and distributors accountable for information patients and clinicians post about their products through social media channels that the companies support.

The industry has been reluctant to fully embrace the platforms because they lacked direction from the FDA on how to use them without veering into prohibited promotion. The FDA held a public hearing on the issue in 2009 but until now had not offered any guidance.

According to Mental Health America, a new rule proposed by the Centers for Medicare and Medicaid Services (CMS) to remove anti-depressants and antipsychotics from its “protected” status on Part D drug plan formularies would be harmful to consumers.

The proposed rule revises long-standing prior agency policy that required Part D plans to include on their formularies “all or substantially all” drugs within six classes: anti-depressants, antipsychotics, anticonvulsants, antineoplastics, and immunosuppressants. This policy, known as the “six protected classes” policy, has been in effect since the inception of Part D, and has strong congressional support.

David L. Shern, Ph.D, president and CEO of Mental Health America, said the organization is a strong supporter of six classes policy and would join with other advocacy groups in submitting comments opposing the rule, which are due by March 7.

“For many mental health consumers, access to the full range of the most effective medications is a crucial component of successful treatment and recovery,” he said. “Such medically necessary psychotropic medications, and their combination with other services and supports, are often essential to permit people with mental health and substance use conditions to recover and to lead healthy and productive lives in their communities. “

Dr. Shern said policies that restrict access to medically necessary medication not only fail to achieve their intended purpose of reducing overall health care costs but prolong human suffering, and reduce the potential for an individual with a mental health or substance use condition to achieve full recovery.

Per Shern, “These policies fail to acknowledge that physicians and consumers should make individualized treatment decisions, recognizing the unique and non-interchangeable nature of human beings and psychotropic medications, and acknowledging that lack of access to medications has both human and fiscal consequences."

In case you missed it – here's the FDA's latest addition to the social media draft guidance compendium. Nothing earth shattering, but more guidance is better than less.

Some useful tidbits …

* A firm is responsible for product promotional communications on sites that are owned, controlled, created, influenced, or operated by, or on behalf of, the firm.

* A firm is responsible for promotion on a third-party site if the firm has any control or influence on the third-party site, even if that influence is limited in scope. For example, if a firm collaborates, or has editorial, preview, or review privilege, then it is responsible for its promotion on the site and, as such, that site is subject to submission to FDA to meet postmarketing submission requirements. However, if a firm provides only financial support (e.g., through an unrestricted educational grant) and has no other control or influence on that site, then the firm is not responsible for information on a third-party site, and has no obligation to submit the content to FDA. Furthermore, if a firm is merely providing promotional materials to a third-party site but does not direct the placement of the promotion within the site and has no other control or influence on that site, the firm is responsible only for the content it places there and, thus, is responsible only for submitting to FDA promotional content that was disseminated on that site.

* FDA recommends that a firm be transparent in disclosing its involvement on a site by clearly identifying the UGC (User-Generated Content) and communications of its employees or third parties acting on behalf of the firm.

* ... a firm generally is not responsible for UGC that is truly independent of the firm (i.e., is not produced by, or on behalf of, or prompted by the firm in any particular). FDA will not ordinarily view UGC on firm-owned or firm-controlled venues such as blogs, messgae boards, and chat rooms as promotional content on behalf of the firm as long as the user has no affiliation with the firm and the firm had no influence on the UGC.

This last item is both new and clear in it's meaning. And it's important as social media is and should be driven by independent UGC.

Will any of this “free” Pharma to pursue more aggressive social media strategies. Probably not. And that’s too bad.

Compliant social media is in the eyes of the engager -- and it's about the content not the platform.
 

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.

As any healthcare provider will tell you, the fact that actually taking a medication as prescribed is in a patient’s best interest does not lead to a patient doing what is in his best interest. And, to make matters worse, there isn’t any one single over-riding reason why patients are non-compliant.

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.)

But what about programs for medicines that are sold (because of regulatory restrictions) via specialty pharmacy? The use of specialty pharmacies to support patients with complex medical conditions is an effective, well-established practice to help ensure patients comply with their physician-directed treatment plan. One example is Exjade, a treatment option for patients with serious blood disorders who have chronic iron overload due to blood transfusions. (Chronic iron overload is potentially life threatening, and does not always have symptoms that are recognizable until serious complications occur. In order to drive compliance. Novartis (the developer and marketer of Exjade) developed a plan to drive compliance by incentivizing the specialty pharmacy BioScrip to develop and implement aggressive patient communications programs. And, yes, “incentivize” means “paying them to do it.”

Success is not always its own reward.

According to Novartis, they “worked with BioScrip to ensure it had the information needed to reach out to patients. BioScrip reached out to patients using its own protocols to provide education, counseling and information about proper administration of the medicine and to fulfill prescriptions that have been prescribed by a patient's treating physician.”

And the programs worked. Patients were more compliant and that’s a good thing, right? Not so fast.

Yesterday New York filed a joint complaint with eight other states alleging that the Novartis program amounted to “kick-backs” paid) per New York State Attorney General Eric Schneiderman, to “promote Exjade drug to treat excessive iron in the blood.

"This arrangement between Novartis and BioScrip was dangerous for patients and is against the law," Schneiderman said in a statement. "Our lawsuit against Novartis and our agreement with BioScrip send a clear message: Drug companies cannot pay pharmacies to promote drugs directly to patients."

But is working to drive patient compliance “promotion?” Is educating a patient on the urgency of compliance “dangerous?”

What message is being sent by Mr. Schneiderman?

Whether or not the Novartis program is against the law is a legal question to be argued in court – but on the face of it, the lawsuit sounds like an ill-considered shakedown with significant unintended public health consequences.

"The company disputes the allegations made by the Attorney General for the State of New York related to Novartis Pharmaceutical Company's (NPC) interactions with specialty pharmacy BioScrip and intends to defend itself in this litigation," André Wyss, NPC President, said in a statement.

This is an important issue. Hopefully Mr. Wyss’ resolve remains firm.

Dear Colleagues:

It is with regret that I inform you that Dr. Jesse Goodman has decided to leave FDA to return to academia and clinical medicine.  As FDA’s Chief Scientist, Dr. Goodman has been a strong voice for both our regulatory science enterprise, and for FDA’s role in U.S. and global public health.  Among his many accomplishments as Chief Scientist is the development of FDA’s Medical Countermeasure Initiative, which has better positioned FDA to play a strong role in building the nation’s emergency medical counter measures capacity.

Dr. Goodman first came to FDA in 1998 from the University of Minnesota.  As Senior Medical Advisor to Commissioners Friedman and Henney, he conceived of and co-chaired the first U.S. Task Force on Antimicrobial Resistance, before moving on to direct the Center for Biologics Evaluation and Research (CBER).  Under his leadership, CBER worked closely with government and industry partners to help our nation prepare for and respond to major public health threats, including bioterrorism, West Nile Virus and other threats to blood and organ safety, and both seasonal and pandemic influenza.

After his departure from FDA, Dr. Goodman will be serving as Professor of Medicine and Infectious Diseases and Attending Physician at Georgetown University and the D.C. Veteran’s Administration Hospital.  In addition to his teaching and patient care responsibilities, he will direct a new Center on Medical Product Access, Safety and Stewardship. 

I am pleased to note that Dr. Stephen Ostroff, Chief Medical Officer in the Center for Food Safety and Applied Nutrition and the Senior Public Health Advisor to FDA’s Office of Foods and Veterinary Medicine, has agreed to take on the role of Acting Chief Scientist. 

Dr. Ostroff has served in many distinguished roles prior to joining FDA, including at the Centers for Disease Control and Prevention (CDC), where he served as Deputy Director of National Center for Infectious Diseases and as Acting Director of CDC’s Select Agent Program as well as the Director of the Bureau of Epidemiology and Acting Physician General for the Commonwealth of Pennsylvania.  Dr. Ostroff also has consulted for the World Bank on public health projects in South Asia and Latin America. 

Dr. Ostroff graduated from the University of Pennsylvania School of Medicine in 1981 and completed residencies in internal medicine at the University of Colorado Health Sciences Center and preventive medicine at the CDC.  He is a fellow of the Infectious Disease Society of America and the American College of Physicians, and currently chairs the Public Health Committee of the American Society for Microbiology’s Public and Scientific Affairs Board. 

Please join me in thanking Dr. Goodman for his many contributions and his commitment both to FDA and to public health and in wishing him well as he engages important public health issues from his new perspective.  Please also help me welcome Dr. Ostroff to his new role.

Sincerely,

Margaret A. Hamburg, M.D.

Commissioner of Food and Drugs

Johnson & Johnson has submitted a citizen petition asking the FDA to require copies of biological products to bear names that are similar and not identical to those of their reference products.

According to J&J's Chief Biotechnology Officer Jay Siegel "Assigning names that are similar but not the same will appropriately reflect the legal and scientific reality that biosimilars are similar to but not the same as their reference products or other biosimilars.”

It’s likely that 2013 will be the year remembered as the year of the ObamaCare website fiasco. It’s a story tailor-made for the media. But the real stories lie elsewhere.

2013 should be commemorated as the year that America began to celebrate the real debut of personalized medicine. New medicines for cancers and orphan diseases were approved by the FDA using a more progressive view of the risk/benefit equation. And, for the first time, the voice of the patient really made a difference in the agency’s calculations.  2013 will be seen as the year when the FDA began to think about a greater dimensionality of benefits via functional endpoints.

2013 may also be viewed by future generations of healthcare pharmacenti as the year when “blockbuster” thinking was replaced with a focus on patient outcomes. The implications for R&D investment in new molecules as well as companion diagnostics make 2013 the year when we finally take to heart both the philosophical and business proposition that getting the right medicine to the right patient in the right dose at the right time is the best way to embrace a patient-centric care paradigm that is also cost-efficient.

But 2013 may also be viewed as the year when Uncle Sam got into the business of telling physicians (and nurse prescribers) how to practice medicine. This is, after all, the year that “government detailing” (aka, “academic detailing” or “counter-detailing”) hit the streets via our tax dollars and minus any federal oversight. It’s an issue that’s been flying under the radar screen – and that makes it more insidious and twice as dangerous.

What will define 2014? There are many possibilities. Certainly near the top of the list is the march towards American biosimilars. 2014 will be the year when the FDA decides on the INN issue. It’s the rare opportunity when we can learn from the mistakes other nations have made. I predict that the FDA will do the right thing and insist that FOBs carry a sim prefix or some such specific identifier. You cannot create comfort among prescribers and patients with therapeutic ambiguity.

Will 2014 be the year when healthcare communicators and pharmacovigilance trackers decide to embrace social media? Will 2014 be the year of more creative and aggressive use of adaptive clinical trials? Will 2014 be a year of developmental and regulatory success for new antibiotics? Only time will tell.

Let’s hope that 2014 builds upon the success of 2013, learns from its failures and surprises us in exciting ways that we didn’t even expect.

When it comes to medication adherence, is knowledge power? Or is that even the right question. Perhaps patients, and healthcare professionals (and payers and regulators) also need to learn how to share knowledge. When it comes to medication adherence in the 21^st century, the medium is the medicine.

Are package inserts, hard copy med guides, brochures and “starter packages” still the best way to make important healthcare information “sticky?” Were they ever? Will the tried-and-true ways enhance safe use or drive positive therapeutic outcomes? Or do today’s patients (also known as consumers) want their healthcare intelligence the same way they’re getting enlightenment and orientation on all the other things they want and need to know about the daily details of their lives? In short, on tablets and smart phones.

Is there an app for that? 

OH, East is East and West is West and never the twain shall meet.

Nice verse, but unacceptable R&D policy.

This morning the NEJM published "Asia's Ascent: Global Trends in Biomedical R&D Expenditures."

Co-authored by Steve Sammut, Senior Fellow, Health Care Management Lecturer, Entrepreneurial Programs Wharton School, University of Pennsylvania, and board member at the Center for Medicine in the Public Interest, the article provides data to support the positions of Francis Collins and others that the US contribution to research is rapidly eroding relative to other countries, particularly in East Asia.

In recent years, industry has reduced its investment in U.S. biomedical research and development by billions of dollars, while increasing investment in Asia–Oceania. Thus, boosting U.S. government funding alone may be inadequate for retaining long-term R&D leadership.

Francis Collins is sending the article to Congress

Attention must be paid.

India’s Efforts to Aid Poor Worry Drug Makers (New York Times, December 30, 2013) points to the price of cancer medications as the sole impediment to access. Not so.

Price is one variable, but it is not the only one.

Less than 10% of India's 45+ million citizens with cardiovascular disease get even the most common medicines like diuretics and statins. Yet there are 10,500 licensed Indian drug manufacturers producing hundreds of generic anti-hypertensives and other CVD products on the market in India.

While the Times’ story shares a sad tale, the plural of “anecdote” isn’t “data.” The truth is much more complicated and the issue of local production vs. patent protection is a red herring. Almost every drug on the WHO’s list of essential drugs is off-patent – and yet patients in India (and almost every nation in the developing world) lacks access due to poorly run government programs, failing domestic infrastructure, dearth of healthcare providers and, worst of all, lack of diagnosis.

The FTC and biosimilars

Instead of playing name game, it should choose clarity

By Peter Pitts

Bottom of Form

The Federal Trade Commission (FTC) has started to meddle on a health issue that probably isn’t on the radar of more than a couple of health-policy wonks, but it should be of interest to anyone who treats patients or cares about patient safety.

It’s about a class of medicines called “biosimilars,” copycat versions of innovator biologics — or medicines made from living cells. Most people aren’t even familiar with biologics, let alone biosimilars. Biologics are the fastest-growing segment of the pharmaceutical industry and are primarily used to treat life-threatening or difficult-to-target diseases like cancer, diabetes, multiple sclerosis, lupus, Crohn’s disease, rheumatoid arthritis and epilepsy. Many people aren’t familiar with them yet and only know about traditional chemical compounds, which are vastly different.

Why is the FTC involved? Good question. The FTC’s interest here would be to ensure that “anti-competitive” or “deceptive practices” don’t compromise consumers’ ability to know which product he or she is prescribed, and to ensure it’s the one he or she actually receives. That’s because that’s what “informed consumer choice” is all about — right?

Not in this case. The issue the FTC is actually focusing on is whether any and all biosimilars that follow a particular “reference product” (the innovator biologic) ought to have the exact same name as that reference product. And all current indications point to the FTC is about to concur that they should.

This is no trivial issue. It is a fact that no two biologic products produced by different manufacturers will be the same. A biosimilar can only resemble its reference product. Therefore, how biologics are named will directly impact clarity of information around which products a patient has been using. Greater clarity will obviously occur if biologics and biosimilars have distinguishable names, and that clarity will enable better safety monitoring, “adverse event” reporting and timeliness in managing adverse events if they occur, and can even help us better understand which products work better for certain patients and specific subpopulations.

Let’s employ some common sense here. Suppose parents were to give birth to a set of fraternal twins: would it make sense to name them both “Tim,” on the grounds that it would “level the competitive playing field” as they grow and master their fate? Or do the boys have the right — and the rest of world an interest — in being able to tell them apart? And while we’re on the subject of names, let’s remember that the name “biosimilar” was coined for a reason — as with fraternal twins, who are not identical. (Even if they were — and you wanted to marry one of them — you’d probably want your “choice” to be “informed” by their having distinguishable names.)

In the FTC’s eyes, it seems to come down to clarity of information versus eking out every possible cent of cost savings. It’s not worth the trade-off. Biosimilars have the potential to provide quality alternative medicines and to improve prices in the biologics space. Because of the complexities associated with all biologics (including biosimilars), however, cost savings from biosimilars are not expected to exceed 10 percent to 20 percent over branded products. Chemical compound generics can realize savings of up to 80 percent over brands.

If we go in the direction of non-unique names, and issues arise, we might not have the information we need to quickly understand which among similar products is causing the issue. That can unnecessarily affect trust across a class of drugs and biosimilars as a whole, and that could significantly affect uptake.

Biosimilars are already available in other parts of the world. This gives us a unique opportunity to learn from the experiences of those markets. In Europe, where biosimilars share the same names as the originator product, they’re experiencing an increased number of adverse events, and it can take months for manufacturers to determine if their product is causing the problem.

Thailand also uses nondistinguishable names and rapidly approved biosimilars to treat certain diseases, which has led to both a dramatic increase in the number of cases of life-threatening blood-related adverse events and near futility in efforts to track back to which products are causing the problems. Australia opted for distinguishable codes for all biologics, and they appear to be experiencing successful rollout and uptake of biosimilars.

It’s a universal reality: What’s in a name is a fundamental ability to tell things apart. Nothing more informs American competitiveness and informed consumer choice. No one more than the FTC should recognize that fact — and be its champion.

Peter J. Pitts, a former FDA associate commissioner, is president and co-founder of the Center for Medicine in the Public Interest.

According to the FDA Law Blog:

There’s a hot debate brewing over whether or not a biosimilar biological product licensed under Section 351(k) of the Public Health Service Act (“PHS Act”), as added by the Biologics Price Competition and Innovation Act of 2009 (“BPCIA”), should share the same non-proprietary name – or International Nonproprietary Name (“INN”) – as its brand-name reference product counterpart.  Last Friday, Amgen Inc. (“Amgen”) further turned up the heat when the company announced the submission of extensive comments (89 pages in length) to two citizen petitions submitted to FDA earlier this year requesting that the Agency require that a biosimilar be identified by the same INN as the reference product it relies on for approval.

The first petition (Docket No. FDA-2013-P-1153) was submitted by the Generic Pharmaceutical Association (“GPhA”) in September and requests that FDA “implement its INN naming policy equally to all biologics” such that all biosimilars “share the same INN name as the” reference product (see our previous post here), The second petition (Docket No. FDA-2013-P-1398) was submitted in October by the Novartis Group of companies (“Novartis”) and requests that FDA “require that a biosimilar[] be identified by the same [INN] . . . as the reference product.” (see our previous post here).

According to Amgen:

Contrary to the position of GPhA and Novartis . . . , we believe that a policy of identical non-proprietary names would pose significant public health risks, and is inconsistent with applicable legal requirements.  Petitioners fail to take into account fundamental scientific principles of biological products as well as their distinctive posology and methods of administration in clinical practice.  We believe that, both as a matter of public health and as a matter of law, biological products licensed under the BPCIA should have distinguishable non-proprietary names—that is, non-proprietary names comprised of common roots and distinguishable prefixes or suffixes, as deemed appropriate by FDA, to achieve the goals of patient safety through effective post-market surveillance, and widespread physician and patient acceptance and appropriate use of this important new class of medicines.

Amgen lays out its case for distinguishable non-proprietary names – and opposition to the GPhA and Novartis petitions – in seven parts, arguing along the way that:

(1)  “Biosimilars licensed under the BPCIA are appropriately neither required nor expected to be structurally identical either to their reference counterparts or to each other. Indeed, the product quality attributes for biosimilars may fall outside even the range of variability that is acceptable for the reference product, and differences in both structure and function will become increasingly prevalent as the complexity of the reference products increases.  Because biosimilars cannot be—and are not—brought to market through the same abbreviated pathway as generic drugs, the legal requirements applicable to generic drugs (including the requirement that generics bear the same labeling and established names as their reference counterparts) do not apply to biosimilars.  This difference in regulatory treatment is both appropriate and critically important to development of biosimilar medicines using state of the art technology.”

(2) “Distinct safety and immunogenicity profiles are found among structurally related biological products.  Similar biological products cannot be fully characterized in premarket clinical trials, but instead must be subject to effective post-market surveillance that distinguishes accurately among related products. . . .  [I]t is especially important that pharmacovigilance measures account for the possibility of differences in the safety profiles of related biological products.  These differences could be intrinsic to products as a result of their distinct methods of manufacture, or they could be emergent as a result of post-approval manufacturing changes.”

(3) “Because FDA’s spontaneous reporting/post-market surveillance system does not encompass the separate tracking of [products] sharing the same non-proprietary name to be separately tracked, biosimilars with the same non-proprietary names but potentially different immunogenic profiles will be difficult to distinguish, hampering immunogenicity tracking and optimal pharmacovigilance.”

(4) GPhA and Novartis include in their petitions “numerous unsupported and erroneous objections to the use of distinguishable names for biological products.”

(5) A biosimilars naming policy that would mirror the naming rules for generic drugs “would create a significant risk of confusion regarding a biosimilar product’s bioequivalence or identity to its reference product.  Moreover, given FDA’s obligations under the Administrative Procedure Act to treat like cases alike and to explain any departures from precedent, FDA would need to reconcile any naming policy with its past statements recognizing the potential safety risks of using identical established names for similar biological products, as well as instances in which FDA has required, for safety reasons, the use of distinguishable names for similar products.”

(6) “[T]the need for better pharmacovigilance measures for all drug products does not obviate the need for distinguishable names to facilitate post-market risk management in the context of biosimilars.”

A short missive from White Oak that may portend broader consequences:

 “Rachel Sherman has decided to retire after many years of service to both FDA and CDER. We will miss her leadership and guidance as the OMP Office Director and wish her well in future endeavors!”

Wow.

 Very few words to announce a very big loss.

While Medicare as a whole is a fiscal basket case -- due to run out of money in 2024 -- Part D has been the very model of a well functioning federal program since its implementation in 2006.

The Congressional Budget Office (CBO) found that, between 2004 and 2013, Part D will cost an extraordinary 45 percent below what was initially estimated. Premiums for the program, meanwhile, are roughly half of the government’s original projections. Part D enrollees pay, on average, $30 a month -- a rate that has remained essentially unchanged for years. It’s no wonder that beneficiaries are so pleased with the program. In fact, 96 percent of those enrolled in Part D say that their coverage works well.

These unprecedented results are largely due to Part D’s market-based structure. Beneficiaries are free to choose from a slate of private drug coverage plans, forcing insurers to compete to offer the best options to American seniors. It’s hardly surprising that the program has led to low prices and satisfied customers.

Through their own negotiations with drugmakers, private insurance plans that operate under Part D have already had great success in keeping pharmaceutical prices down. In fact, the CBO has observed that Part D plans have “secured rebates somewhat larger than the average rebates observed in commercial health plans.”

What’s more, the CBO has said time and again that doing away with the non-interference clause “would have a negligible effect on federal spending.” In a report from 2009, they reiterated this view, explaining that such a reform would “have little, if any, effect on [drug] prices.”

Smart partnerships between government and the free market work. They work at keeping costs low and – most importantly – improving care. As JAMA reported,  “Implementation of Medicare Part D was followed by increased use of prescription medications, reduced out-of-pocket costs, and improved medication adherence.” And this, in no small measure, significantly reduces more drastic medical interventions -- which in turn reduces our overall national health care spending.

A new report from the Manhattan Institute, “A Decade of Success: How Competition Drives Savings in Medicare Part D,” details many of the reasons why a free-market approach to healthcare access is not only working – but a model for how to design future healthcare partnerships between Uncle Sam and the private sector.

Here is the report’s executive summary:

National trends are not a sufficient explanation for Part D’s success.

While patent expirations are part of the story—national drug spending as a whole slowed during the period we examine—they are far from the full explanation for large overestimates in Part D spending (indeed, patent expirations were likely captured in the original projections). Instead, the available evidence indicates that private-sector firm-level innovations, including preferred pharmacy networks and aggressive negotiations with drug manufacturers, have played a significant role in keeping the program’s costs below projections. We find that broader market trends (e.g., patent expirations and other changes) account for only about half (56 percent) of the program’s performance. The remainder—44 percent—of Part D’s lower-than-estimated cost savings is attributable to factors not captured in national prescrip­tion drug trends, which should include competition between Prescription Drug Plans (PDPs). This is strong evidence indicating that consumer-driven competition in Part D has been critical to the program’s financial success.

Consumer-driven competition is a relatively new tool in the government’s effort to control health-care costs.

In hindsight, government overestimates of Part D’s costs are not surprising, since the program utilizes a model of consumer choice (robust competition among dozens of regional drug plans and Medicare Advantage plans) that has no perfect analogue in other government health plans, such as Medicaid.

Part D is an excellent model for future health-care and entitlement reforms.

Arguably, Part D and Medi­care Advantage plans represent the first national health-care exchange (the Federal Employees’ Health Benefits Program [FEHBP] is a close cousin). While the Affordable Care Act (ACA) operates a similar exchange concept, there are important differences. First, Part D plans compete in large regional areas, not states (this creates much bigger risk pools because even large states are incorporated into larger regions), as the ACA exchanges do. Even the federal exchange is layered on top of a state-regulated insurance market. This potentially limits the ability of plans to create economies of scale to bargain with providers and to utilize innovative tools to arbitrage cost and quality differences across state markets (preferred pharmacy and mail-order networks in Part D; telemedicine and medical tourism to “centers of excellence” for health-insurance plans). Notably, while Part D includes higher subsidies for sicker seniors (typically, the low-income subsidy population), it does not penalize healthier seniors through higher premiums, as the ACA’s community rating provisions do. Arguably, a better approach would be to rely more on backdoor (non-cross-subsidized) risk adjustment of plans and larger subsidies for sicker or older patients, while allowing plans to charge actuarially fair premiums to younger enrollees. The cross-subsidies in the Part D approach are more transparent in that sense, since they come from tax revenues rather than from private premiums.

The complete MI report can be found here.

Per a new draft guidance:

This is important and even more significant considering the agency’s recent proposed rule that would permit generic drug makers to update their labels if they received information about potential safety problems.

(For more on this, see here.)

In Friday’s Washington Post, the editorial page opines that, “… not every problem can or should be solved by federal regulation, and there is still a decent chance that the balance between regulation and liberty that the Affordable Care Act struck will work. If it does, the more rational health-care system that results will have been well worth the price in expanded government intervention in the health-care market.”

That’s the conclusion, but the body of the piece reads like an introduction to a manifesto about why what we really need is a single-payer system. A "rational health-care system" is the the global code phrase for "one-size-fits-all" care. Have a look here and judge for yourself.

Here’s my view on some of the same problems from a different angle (as seen in yesterday’s Des Moines Register).

The devil is in the details.