Editor’s note: This post is part of a Health Affairs Blog Symposium on Health Law stemming from 4th Annual Health Law Year in P/Review conference hosted by the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School. Holly Fernandez Lynch wrote an introductory post in January 2016 and you can access a full list of symposium pieces here or by clicking on the “The Health Law Year in P/Review” tag at the bottom of any symposium post.
This post is based in part on a debate which took place between Jerry Avorn and Mit Spears. After reading this piece, check out the counter argument. You can also watch a video of the debate.
The ability of pharmaceutical companies to provide information about medicines to health care professionals beyond that which is contained in the Food and Drug Administration (FDA)-approved labeling is a topic that has generated a great deal of discussion, particularly in light of the August 2015 Amarin decision rendered by a US District Court in the Southern District of New York, and the FDA’s recent settlement of the case.
In Amarin, Judge Paul Engelmayer ruled that the FDA’s regulation of information provided by pharmaceutical companies violated the First Amendment’s Commercial Speech doctrine to the extent that they prohibited the communication of truthful and non-misleading speech, even where the information provided went beyond the FDA-approved labeling. On March 8, FDA and Amarin settled the case in a way that preserves Amarin’s ability to share truthful, non-misleading information about its medicine. In fact, the FDA even agreed to provide advisory review of two such communications per year with a 60-day timeline.
This case followed on the heels of the Second Circuit’s 2013 decision in Caronia and the Supreme Court’s 2011 ruling in Sorrell v. IMS Health, Inc., in which the Courts affirmed that the First Amendment’s protections of commercial speech extended to pharmaceutical companies and their employees.
The Justice Department did not appeal the decisions in Amarin or Caronia. Consequently, for the foreseeable future, Amarin provides the most complete explanation of how the First Amendment protects communications between pharmaceutical companies and health care professionals.
While some believe that that the Amarin decision opens opportunities for irresponsible and misleading claims about medicines, in fact Amarin provides the basis for a robust and effective regulatory platform to ensure that communications between pharmaceutical companies and health care professionals are truthful and non-misleading.
The Origins And Limitations of Labeling
The FDA’s regulatory structure governing the provision of information by pharmaceutical companies can trace its origins to the Kefauver Amendments to the original Food, Drug, and Cosmetics Act. These amendments were enacted in 1962, long before many of the innovations and advances in medical knowledge we depend on today were even considered. The amendments were passed in response to the Thalidomide tragedy, where women taking the drug to counter the effects of morning sickness gave birth to children who were horribly injured.
Under the Kefauver Amendments, pharmaceutical companies were required to prove the safety and effectiveness of products before they entered the market. They were also required to provide safety and usage information about the medicine to health care professionals in the form of approved FDA-labeling.
In 1962, the information needs of the health care system about prescription medicines were relatively limited. Doctors needed to know the indications for which the medicine was approved, potential side-effects and counter-indications, prescribing and dosage information, and data about the clinical trials that had been conducted to obtain FDA approval. Beyond that, pharmaceutical companies at the time didn’t have a lot more information to offer.
While the double-blind, placebo controlled clinical trials are considered the “gold standard” for product testing, it’s important to recognize that such studies were the only source of information for much of the past half century. We didn’t have electronic medical records. Insurers did not maintain enormous searchable databases detailing symptoms, diagnoses, treatments, or outcomes. And even if they had, we didn’t yet have the technological capability to process, analyze, and understand those data sets.
The Information Revolution
Medical technology and pharmaceutical innovation today have placed an arsenal of new and effective tools in the hands of treating physicians. The amount of information available about new treatments and techniques has exploded and the need to acquire, understand, and intelligently use this information has placed incredible pressure on physicians and payers. Examples include:
- Physicians, practice groups, and payers all need information to help manage costs and improve outcomes.
- Health care providers need comparative data that permits them to compare one form of treatment with another.
- Payers with enormous electronic medical record databases need analytical support to better identify places where adjustments to practice guidelines, formularies, or reimbursement schedules can help control costs and improve outcomes.
This information is never going to be found in product labeling. Even under the FDA’s current restrictive approach, the amount of information shoe-horned into a product label has grown to the point where even the FDA is concerned that the “noise” contained in product labels threatens to overwhelm the critical information that physicians need to make informed prescribing decisions. Printed product labeling simply cannot accommodate ever-changing pharmacoeconomic and comparative effectiveness information that can change from week-to-week as new products enter the market and analytics deliver new and important insights.
Some acknowledge that there is a need for pharmaceutical companies to provide this kind of information beyond the FDA-approved product label, but draw a distinction between information pertaining to approved indications and information relating to a non-approved use of an approved medicine. Their view is that the pharmaceutical communications to health care providers should be confined to those uses approved in FDA labeling.
This view ignores some basic facts about the non-approved uses of medicines. Twenty-one percent of all prescriptions are prescribed for uses other than those contained in the approved product label. Fifty percent to 75 percent of oncology prescriptions are written for non-approved uses. Sixty percent of prescriptions written in out-patient pediatrics are for uses other than those listed in the product label. And, in pediatric intensive care, that percentage increases to greater than 95 percent. Even with more conventional medicines like asthma and cardiac medications, over 40 percent are written for non-approved uses.
These figures reflect the undeniable fact that non-approved uses of approved medicines constitute an integral part of modern medical care. Many of these alternative uses are listed in widely respected medical compendia. Others reflect the recognized standard of care in their field of medicine. And many of these alternative uses are readily reimbursed by both government and private payers.
With millions of patients being prescribed medicines for non-approved uses, what purpose does it serve to prevent pharmaceutical companies providing information about these uses? To prescribe intelligently, physicians need basic information like recommended dosages, titration protocols, potential drug interactions, or counter-indications. No one has a more complete understanding about these alternative uses than the pharmaceutical company who developed the drug.
Our modern health care system needs much more information about medicines than can be found in the product label and, in many cases, pharmaceutical companies are the only source of that needed information. But how can we ensure that the effective communication of this information serves the needs of health care professionals without permitting unscrupulous actors to make irresponsible, untruthful, or misleading claims?
Building A Modern Information Ecosystem In Health Care
Often overlooked in Judge Engelmayer’s opinion in Amarin is the observation that the FDA has every right to establish and enforce standards to ensure that communications by pharmaceutical companies are truthful and non-misleading. Instead of preventing the communication of all non-label information about medicines, the FDA can and should shift its focus to preventing the communication of false and misleading information about medicines.
How might the FDA accomplish this?
First, communications should be substantiated by solid science. Every claim made by a pharmaceutical company about its products should be substantiated via scientifically valid and statistically sound methodologies. Questionable science and sketchy statistical analyses can produce potentially false and misleading information which is not protected by the First Amendment and need not be permitted by agency regulation.
Second, companies should be required to be transparent about their claims and their substantiation. When statements rely upon something less than FDA approval standards, the science and statistical analyses supporting the claims should be disclosed and made available to the person receiving the communication. If the company possesses information that contradicts or questions a claim, that too should be disclosed. Clearly the company would retain the right to critique or question contrary results as part of that disclosure, but it should not be able to use such concerns to justify non-disclosure of all the data.
Moreover, the regulatory status of any use not otherwise approved by the FDA should be clearly and conspicuously disclosed. Communications relating to the non-approved use of an approved medicine should be conspicuously disclosed as such.
Third, claims and their supporting substantiation should be tailored so that the target audience understands the scope and limits of any claims being made. For example, communications that might be appropriate with a formulary manager or a specialty medicine practice standards committee may not be appropriate for general practitioners, where additional explanation and context may be required.
These three basic principles would permit us to develop comprehensive regulatory standards to help ensure that communications between pharmaceutical companies and health care providers are truthful and non-misleading. Collectively, we can develop reasonable standards and common sense rules to govern the provision of information at medical conferences, the provision of reprints from peer-reviewed journals, and the provision of information relating to unapproved medicines with formulary managers.
The needs of our health care system reach far beyond the limited information that was available about medicines 50 years ago. By shifting the focus of its regulatory efforts from blocking communications to ensuring that communications are truthful and non-misleading, the FDA can preserve its critical mission of protecting patient health and better serve the needs of patients, providers, and payers. It’s what the First Amendment commands and it’s what our health care system needs. The recent Amarin settlement demonstrates that FDA can adapt to a constitutional standard that guarantees the ability to share truthful, non-misleading information about medicines in a responsible way. PhRMA looks forward to working with the agency to meet the needs of health care professionals and payers in this data-driven health ecosystem.
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