Life Science Compliance Update

July 25, 2017

New Report Releases Strategies to Reduce Opioid Epidemic


The National Academies of Sciences, Engineering, and Medicine recently released a report, requested by the United States Food and Drug Administration, that highlights what can be done to stop the opioid use disorder and other opioid-related harms without closing access to opioids for patients who need them.

The committee that conducted the study and wrote the report recommended actions the FDA, other federal agencies, state and local governments, and health-related organizations should take – which include promoting more cautious prescribing of opioids, expanding access to treatment for opioid use disorder, preventing more overdose deaths, weighing societal impacts in opioid-related regulatory decisions, and investing in research to better understand the nature of pain and develop non-addictive alternatives.

In more recent years, national initiatives to reduce opioid prescribing have modestly decreased the number of prescription opioids dispensed. Unfortunately, many people who otherwise would have been using prescription opioids have transitioned to heroin use. According to the report, the declining price of heroin, together with regulatory efforts designed to reduce harms associated with the use of prescription opioids – including the availability of abuse-deterrent formulations – may be contributing to increased heroin use.

One approach to addressing the opioid epidemic is to have a fundamental shift in the nation’s approach to prescribing practices and improve awareness of the risks and benefits of opioids. Therefore, the committee recommended education for both health professionals and the public. Such education should involve mandating pain-related education for all health professionals who provide care to people with pain, requiring and providing basic training in the treatment of opioid use disorder for health care providers, and training prescribers and pharmacists to recognize and counsel patients who are at risk for opioid use disorder or overdose.  In addition, the lack of attention paid to educating the public about the risks and benefits of prescription opioids needs to be addressed. The report called for an evaluation of the impact and cost of an education program that raises awareness among patients with pain and the public.

The committee stressed that restrictions on lawful access to prescription opioids could have other unintended effects, and any policy designed to curtail legal access to them will inevitably drive some people toward the illegal market. Therefore, a strategy for reducing lawful access to opioids should be coupled with an investment in treatment for the millions who have opioid use disorder. 

The committee recommended that states – with assistance from relevant federal agencies, particularly the Substance Abuse and Mental Health Services Administration – provide universal access to evidence-based treatment for opioid use disorder in a variety of settings, including hospitals, criminal justice settings, and substance-use treatment programs. The U.S. Department of Health and Human Services (HHS) and state health financing agencies should also remove impediments to full coverage of medications approved by the FDA for treatment of opioid use disorder.

Several other strategies that the committee recommended include:

  • the FDA should complete a review of the safety and effectiveness of all approved opioids;
  • states should convene a public-private partnership to implement drug take-back programs that allow drugs to be returned to any pharmacy on any day, rather than relying on occasional take-back events;
  • public and private payers, including insurance companies, should develop reimbursement models that support evidence-based and cost-effective comprehensive pain management, including both drug and non-drug treatments for pain;
  • HHS, in concert with state organizations, should conduct or sponsor research on how data from prescription drug monitoring programs can be better leveraged to track opioid prescribing and dispensing information; and
  • the National Institutes of Health, the Substance Abuse and Mental Health Services Administration, the U.S. Department of Veterans Affairs, and industry should invest in research that examines the nature of pain and opioid use disorder, as well as develop new non-addictive treatments for pain.

July 21, 2017

FDA Hearing on Innovation and Access


Earlier this week, the Food and Drug Administration (FDA) held a public hearing entitled “The Hatch-Waxman Amendments: Ensuring a Balance Between Innovation and Access,” to address the agency’s plan to increase competition on the drug market while fostering innovation. A variety of stakeholders including academics/researchers, payers/providers, pharmaceutical developers, and patient/consumer advocates provided diverse input to a panel of FDA employees. The panel included Anna Abram and Elizabeth Dickinson of the Office of the Commissioner at the FDA; Keith Flanagan, Maryll Toufanian, and Kathleen Uhl of the Office of Generic Drugs at the Center for Drug Evaluation and Research (CDER), FDA; Grail Sipes of the Office of Regulatory Policy at CDER; Peter Stein of the Office of New Drugs at CDER; and Markus Meier at the Bureau of Competition at the Federal Trade Commission.

Opening Remarks

The meeting opened with remarks made by Dr. Scott Gottlieb, Commissioner of the FDA and Janet Woodcock, the Director of CDER. Dr. Gottlieb noted,

FDA doesn’t have a direct role in how drugs are priced. But at FDA, we do play a key, if indirect, role in the eventual cost of medicines. For one thing, our regulatory requirements impact the cost of drug development. On some level, drugs are ultimately priced to some measure of the cost of the capital needed to create them. These costs aren’t just a reflection of the direct cost of drug development, but also lots of indirect costs. They include the cost of scientific and regulatory risk. They also include the costs associated with the time it takes to develop a drug and gain its regulatory approval, and the costs associated with the research and development of experimental products that ultimately do not make it to market.

He acknowledged that the FDA does have some ability to help reduce the time and uncertainty of drug development by ensuring its regulatory requirements are efficient, predictable, and science-based.

He also pointed to another way the FDA can have an impact on drug costs:

by encouraging competition. Consumers derive greater value when they have access to more choice and competition. This is especially true when it comes to new drug categories. Novel drugs that are therapeutically similar, or can be used interchangeably, can provide price competition. In other instances, they offer important clinical differentiation for patients who might not respond to one particular drug, but benefit from a medicine that works through a slightly different mechanism.

Commissioner Gottlieb further noted that the FDA will address “gaming” of the system that keeps generics out of the market. He said that the FDA is taking steps to “improve our own regulatory framework” including a highly efficient review cycle to “increase the speed at which generic drugs can enter the market.”


Academic Perspectives

Researchers and academics focused heavily on increasing competition through: reforming REMS regulations; addressing harms from citizen petitions; and brand pharmaceutical marketing practices.

Michael Carrier, an expert in the pharmaceutical industry and antitrust laws at Rutgers Law, stated that certain actions by brand companies distort the Hatch-Waxman Act to evade the system and impede entry of generics to the market, and that these actions “are not about innovation.” FDA panelists were interested in how to prospectively address the approximately 20 percent of reformulations, or changes in brand product, that are aimed at elongating exclusivity.

Harvard Medical School researcher Ameet Sarpatwari presented research showing that violations of REMS by brand companies restricted access to drug samples and therefore delayed generic entry. He used this data to advocate for the CREATES Act and FAST Generics Act, both of which he said would close REMS loopholes and potentially save up to $5.4 billion a year in reduced drug costs. Dr. Sarpatwari noted that in 2012, brand companies spent $24 billion marketing to physicians against generics, and $3.1 billion on marketing directly to consumers.

Payer and Provider Perspectives

Payers and providers expressed frustration with brand companies using FDA regulations to inhibit generic entry, including pay-for-delay, patent evergreening, and product-hopping.

Todd Ebert and Wayne Russell of the Healthcare Supply Chain Association proposed creating a more specific timeline for fast tracked applications for generic products, lowering the FDA review time to 8 months from the current 20 months, and prioritizing generics with no suppliers on the market. REMS reform continued to be a main topic for payers and providers.

Pharmaceutical Development Perspectives

Association for Accessible Medicines (AAM) President and CEO Chip Davis said that market realities allow for “gaming” of the system, noting that “innovation is flourishing but competition is not.” Davis stated that barriers for entry stall competition, and are exacerbated in low volume markets, as markets need at least three to four generics to reduce drug costs. He proposed waiving the requirement for a single, shared REMS system, reevaluating bioequivalence (BE) standards and improving the generic drug review process through PDUFA II. He noted that “failure to act will be significant and encourage anti-competitiveness.” The panel was receptive to Mr. Davis’s proposals, asking clarifying questions about the BE standard developments and low volume markets. All manufacturers believed that REMs should not be used to restrict access of generics to samples.

David Korn, Vice President of the Pharmaceutical Research and Manufacturers of America (PhRMA) stated that the innovator industry invests billions in research and development, and that IP protections supports long term sustainability for drugs on the market. He said that uncertainties in the patent system creates challenges for innovative companies, as patent challenges have been filed more frequently. The FDA panel asked Mr. Korn if there should be a requirement for companies to demonstrate reasoning for post approval changes and if these reasoning’s should be public.

John Murphy, Deputy General Counsel for Biotechnology Innovation Organization, noted that FDA efforts to publish a list of products where there is no competition “should go a long way to relieve concerns of competition for generics,” but also said that supplying unlimited samples for testing can be challenging for innovative companies.

Patient and Consumer Perspectives

Patient and consumer stakeholders echoed concerns about REMS and abuses of citizens petitions, though the bulk of the presentations centered around the patient experience. Speakers encouraged “regulatory humility” since abuse of government procedures presents a threat to competition.

July 18, 2017

United States House of Representatives Passes PDUFA


On Wednesday, July 12, 2017, the United States House of Representatives passed a bill via voice vote to reauthorize the prescription drug, generic drug, medical device and biosimilar user fee programs through 2022.

The current user fee programs expire September 30th, though the United States Food and Drug Administration (FDA) has said that if the reauthorization bill is not signed by President Donald Trump before August 1st, the FDA will be forced to send out layoff notices to about 5,000 employees.

“This legislation will save lives,” Representative Greg Walden said on the House floor, noting he thinks the Senate will adopt the bill, as well.

In addition to reauthorizing the user fee programs, the bill contains several new directives for the FDA, including provisions requiring quicker reviews of some generic drugs and changes in how the agency will conduct medical device inspections. The FDA will also be instructed to work on expanding clinical trial criteria so more patients can be eligible for experimental medicines. The bill is expected to cut the time it takes the agency to approve new treatments.

The House added more than a dozen new sections to the bill ahead of the vote, including sections aimed at curbing abuse of the orphan drug designation and policies designed to speed generic drug approvals. Most of the new elements of the House bill were added to align the chamber's legislation with the version passed out of the Senate HELP Committee. The House bill added a new title focused on pediatric drugs and devices that would allow the FDA to require adult cancer drugs that share a common target with a pediatric cancer to be studied in children starting in 2020. Other new elements of the bill clarify when pediatric information can be left off a drug's label to speed access to generic medicines, and new sections mandating that FDA review generic drug applications within eight months if the brand drug patents have expired and the treatment has three or fewer approved competitors on the market.

Several sections of the bill were removed ahead of the vote, including provisions altering the notification requirements for FDA related to laboratory developed tests, requiring FDA to issue guidance on how to demonstrate bioequivalence to a reference drug to facilitate generic development, a section on information technology contracting, and legislation designed by industry and the FDA to create an over-the-counter monograph.

Senate Reaction

The Senate has yet to schedule a floor debate and vote on its version of the user fee bill. Senate Lamar Alexander said Wednesday that it's now time for the full Senate to consider these bipartisan user fee agreements.

Senate Majority Leader Mitch McConnell (R-KY) has stated, “It’s an extremely important bill that I think we’ll be able to move on a bipartisan basis.” And ranking member Patty Murray (D-WA) pledged a “bipartisan and non-controversial” user fee bill, highlighting the necessity of similar legislation.

However, Senator Ron Johnson also said Wednesday that he will try to delay the bill unless it includes language related to the “Right-to-Try” legislation, which undercuts FDA and has been sweeping across states. A nonpartisan report issued Tuesday notes that issues with expanded access are not linked to FDA regulations.

The Congressional Budget Office says it expects the FDA to collect roughly $9 billion in fees—$8 billion for drugs and $1 billion for devices—between 2018 and 2022, based on the fee level set in the Senate bill.

Industry Reaction

FDA Commissioner Scott Gottlieb, MD, praised the passage of the bill in the House on Twitter:


Pharmaceutical Research and Manufacturers of America (PhRMA) president and CEO Stephen J. Ubl issued the following statement,

Timely reauthorization of the Prescription Drug, Biosimilar and Generic Drug User Fee Acts is crucial to patients in need of life-saving treatments and enhancing the competitive market in biopharmaceutical innovation. The House took an important step in reauthorizing the user fee programs and we look forward to swift action in the Senate on behalf of America’s patients.


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