Life Science Compliance Update

December 08, 2016

21st Century Bill Headed to President Obama’s Desk

President-obama-desk

Nearly three years after its initial introduction, the 21st Century Cures Act has finally passed both houses of the Congress, and is expected to be signed by President Obama relatively quickly. This bill represents one of the few truly bipartisan efforts undertaken by this Congress.

We have previously written about some of the items included (and excluded) in the bill. The bill gives the health institutes the authority to finance high-risk, high-reward research using special procurement procedures, as opposed to more conventional grants and contracts. It also requires the agency’s director to establish “Eureka prize” competitions to advance biomedical research and improve treatments for serious illnesses. The bill also creates a new assistant secretary for mental health and substance use, to be appointed by the president, thereby raising the status of mental health issues throughout the country.

Further, the bill directs federal agencies to step up their enforcement of laws that require equal insurance coverage for mental and physical illnesses. Federal laws and rules requiring mental health parity have been adopted with bipartisan support over the last twenty years, but a White House task force recently found that compliance with those laws has been lagging.

Political Reactions

The bill includes $500 million a year to help states prevent opioid misuse and get better treatment for addicts. "These additional resources are particularly critical in rural areas, where rates of opioid misuse and overdose are high, access to treatment is limited, and patients who seek treatment are often met with waitlists that can mean the difference between life and death," said Agriculture Secretary Tom Vilsack.

In a statement made by President Obama shortly after passage of the bill, “We are now one step closer to ending cancer as we know it, unlocking cures for diseases like Alzheimer’s, and helping people seeking treatment for opioid addiction finally get the help they need.”

Obama also noted, “This is a reminder of what we can do when we look out for one another. Like Joe Biden and so many other Americans, I’ve lost people I love deeply to cancer. I’ve heard often from those whose loved ones are suffering from Alzheimer’s, addiction and other debilitating diseases. Their heartbreak is real, and so we have a responsibility to respond with real solutions. This bill will make a big difference, and I look forward to signing it as soon as it reaches my desk.”

Senator Susan Collins (R-ME) stated, “I doubt that there is a family in America who will not be touched by this important legislation.” Representative Steve Cohen (D-TN) was also pleased with the passage, saying, “I don’t think there is enough money that we can put into the N.I.H., because it is important and affects all Americans independent of political party, race, sexual orientation – you name it.”

Medical and Industry Reactions

"The remarkable bipartisan, bicameral support for the 21st Century Cures Act proves that congressional lawmakers are serious about the need for scientific research, effective care-delivery, and the removal of barriers to scientific progress," said Dr. Daniel Hayes, president of the American Society of Clinical Oncology.

"This legislation will improve the lives and health of countless Americans," said American Psychological Association President Susan McDaniel. "It will increase access to effective, evidence-based care, particularly for those with serious mental illness."

In a Statement from Sharad Lakhanpal, MBBS, MD; President of the American College of Rheumatology, he noted,  "While we are pleased that the 21st Century Cures Act will infuse additional, much-needed funding to the NIH to support medical research across the healthcare spectrum, we also know that more can and should be done to help Americans who are disproportionately impacted by arthritis and other rheumatic diseases. Today, arthritis is the leading cause of disability among U.S. veterans and the second most common reason for medical discharge from the U.S. Army. One in three veterans is diagnosed with arthritis, compared with one in five members of the general U.S. population.

As Congressional leaders turn their attention to budget appropriations for the upcoming fiscal year, we urge them to prioritize the creation of a dedicated $20 million arthritis research program at the Department of Defense so that we can better meet the care needs of the thousands of active duty and veteran members of our Armed Forces who live with arthritis.”

Conclusion

Once the bill is signed by President Obama, it will be up to the Food and Drug Administration (FDA) to implement many portions of the law, which could take years. Funding may be immediate, but the actions the FDA and other federal agencies are responsible for may still be quite a bit away. We will all have to stay tuned as this bill, and its effects, continue to progress.

November 10, 2016

FDA Off-Label Hearing: Day One

Prescription

Yesterday, the Food and Drug Administration (FDA) kicked off a two-day public meeting to review the extent to which off-label information about medicines may be disseminated to physicians. The meeting opened with Dr. Robert Califf, Commissioner of the FDA, making brief opening remarks, followed by a slew of individual presentations.

The presentations were limited to eight minutes (joint presenters were given 16 minutes) and included speakers such as Mr. Michael Labson of Covington & Burling LLP, on behalf of Pharmaceutical Research and Manufacturers of America (PhRMA); Ms. E. Cartier Esham with the Biotechnology Innovation Organization (BIO); Ms. Khatereh Calleja of the Advanced Medical Technology Association (AdvaMed); Dr. Andrew Koenig of Pfizer Inc.; Dr. Edith Perez with Genentech; Dr. John Kamp with the Coalition for Healthcare Communication; and Ms. Jennifer Graff of the National Pharmaceutical Council.

The public hearing comes amongst a growing tide of court decisions and settlements that affirm the First Amendment right of pharmaceutical and medical device companies to engage in truthful, non-misleading speech about approved products, including off-label conditions of use.

Concerns of Both “Sides”

One of the FDA’s chief concern is that public health may be compromised if a company communicates an unapproved use that has not been confirmed to be safe or effective. According to Michael Carome, director of Public Citizen’s Health Research Group, “It is tremendously important that the FDA strongly regulate manufacturer communications regarding unapproved uses of approved or cleared medical products to protect public health. Without strong limits on such industry communications, our long-standing regulatory framework for ensuring that drugs and medical devices are safe and effective for their intended uses would be undermined, threatening patient health.”

Industry, on the other hand, is concerned that public health may be compromised if a company is not permitted to communicate relevant, truthful, non-misleading and clinically-proven information – even if the use is unapproved.

Deputy general counsel of BIO, Deborah M. Shelton, believes, “Removing current regulatory barriers, and clarifying the ability of companies to share truthful and non-misleading information about medicines, is essential to our collective ability to realize the full potential of 21st century medicines and helping to ensure that patients are able to get the right medicines at the right time for them.”

 

What is the FDA Looking For?

The FDA sought responses to a variety of questions, including: (1) how increased communications from firms about unapproved uses impact the public health and how it might differ across different categories of medical products and categories of communications; (2) what kind of information or systems currently exist to help the FDA determine how firms’ increased communication of information about unapproved uses could affect prescribing and medical product development and research; (3) whether or not there should be different approaches for different categories of medical products (i.e., human drugs and biologics, medical devices, animal drugs), or for different disease areas or patient populations; (4) what standards should apply to unapproved use communications to minimize the potential of these communications to be misleading or otherwise cause harm; and (5) what standards should the FDA otherwise use to determine whether or not the communications are truthful and non-misleading, including disclosures.

Consumer Reports

A Consumer Reports survey showed that a majority of Americans do not like the idea of allowing pharmaceutical companies to advertise a drug for a use that has not been approved by the FDA. Consumer Reports spoke at the meeting, calling on the FDA to maintain its ban on the “off-label” promotion of drugs and medical devices.

PhRMA Statement Released

In addition to Mr. Labson’s comments at the hearing, PhRMA president and CEO Stephen Ubl released a statement, noting that,

As the U.S. health care system evolves to become increasingly focused on value, patients, payers and providers seek more science-based safety, effectiveness and economic information about existing and emerging treatments for patients. The market for medicines is changing rapidly as alternative payment models proliferate and novel decision tools like value frameworks are being applied. Increasingly, prescribing decisions are made not by independent physicians, but influenced by the integrated delivery systems in which they practice or by financial incentives created by powerful health plans. In this changing environment, it is important that biopharmaceutical companies be able to share appropriate science-based information with population health decision makers, yet the U.S. Food and Drug Administration’s (FDA) current federal regulations have not kept up with this new reality.

Ubl continued, noting,  

Given our increasingly data-driven, value-based health care system, we hope the FDA will adopt a stepwise approach in defining new and clearer regulatory standards to permit responsible sharing of information and data about medicines with other parties in the health care system. The FDA should start by clarifying rules around information sharing with insurance companies and other population health decision makers by the end of the year. Such clarification – also requested by stakeholders in the insurance industry – will help improve the efficiency of our health care system and facilitate increased value-based care.

Conclusion

While any policy changes will not be immediate, the public meeting shows that the FDA is interested in listening to stakeholders, allowing them face time to speak and meet with FDA officials. Any change – even a small change – has the power to influence what kind of information patients receive on drugs and devices, and industry and the FDA have a responsibility to ensure public health remains a priority.

November 04, 2016

FDA Open to Biomarker Comments

FDA has announced the opening of a docket for interested parties to submit suggestions, recommendations, and comments on a proposed list of biomarkers that were used as outcomes to develop FDA-approved new molecular entities and new biological therapeutics from October 2007 to December 2015. The submissions from interested parties will help FDA determine the utility of the list and may assist FDA in developing databases on biomarkers for drug development. The 60-day window for submitting comments will close on November 18, 2016. The submissions can be either public or confidential.

Specifically, FDA welcomes comments regarding the following two areas:

  • Areas of improvement for communicating and disseminating information about biomarkers and their utility as drug development tools.
  • The best approach for updating the biomarkers outcomes list, including any modifications of the list, in the future.

FDA Perspective of Biomarkers

The agency has consistently described the importance of biomarkers. A biomarker is “a characteristic that is objectively measured and evaluated as an indicator of normal biological processes, pathogenic processes, or biological responses to a therapeutic intervention”. Illustrating their importance, the FDA’s Center for Drug Evaluation and Research (CDER) established the Biomarker Qualification Program to support its work with external stakeholders to develop biomarkers that aid in the drug development process. Through the FDA’s Biomarker Qualification Program, a stakeholder may request regulatory qualification of a biomarker for a particular context of use in drug development.

Biomarkers can be used in a variety of settings, including basic research, drug development, and clinical practice. The Biomarker Qualification Program focuses on biomarkers used in drug development. Once a biomarker is qualified, it can be used in any drug development program under the context for which it obtained qualification. The Biomarker Qualification Program is one of the Drug Development Tools (DDT) Qualification Programs created by CDER to provide a framework for development and regulatory acceptance of scientific tools for use in drug development programs. 

Biomarker Challenges in Biosimilar Drug Development

Biologics have transformed the way several diseases have been traditionally treated and managed over the past several years. As patents and exclusivity on several originator biologics are expiring, the industry has been moving in to generate biosimilars. Biosimilars are products that are highly similar to the originator molecules with no clinically meaningful differences in safety, purity and potency.

Owing to the complexity of biological molecules and the variability of sophisticated modern processes by which they are generated, it is difficult to generate fully identical biosimilars. Small structural and functional differences can lead to potentially serious consequences when administered into the diseased host. Regulatory agencies require the generation of physicochemical, structural and functional data on candidate biosimilars prior to approval for use in human subjects. Thus, successful development of biosimilars requires the demonstration of pharmacological similarity with the originator biologics.

MedPAC Supportive of Biosimilar Development

Of particular note, during the last Medicare Payment Advisory Commission meeting, Commissioner Jack Hoadley, a research professor at Georgetown stated, “Unlike the situation where this was discussed in previous years, biosimilars [now] have been approved by the Food and Drug Administration. They're out there. And our goal is to see how to make it easier for consumers to access biosimilars without all the hoops that have currently to be gone through today.”

As reported by Bloomberg BNA, FDA has approved four biosimilars under a 2010 law that created a pathway for such approvals. The agency defines a biosimilar product is a biological product that is approved based on a showing that it is highly similar to an already approved biological product, known as a reference product. MedPAC staffers further noted in a presentation for the commissioners that biologics are large-molecule therapies synthesized from living cells or organisms, in contrast to chemically derived drugs. Biologics are used to treat diseases such as diabetes, rheumatoid arthritis and multiple sclerosis. The staffers stated that biosimilars are expected to reduce costs by 20 to 40 percent.

On further review by the FDA, the biosimilar can be designated as interchangeable with the reference product (RP), which means that a pharmacist could substitute the biosimilar with the RP without a physician's approval. To date, the FDA has not approved an interchangeable biologic drug, only biosimilar drugs.

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