Life Science Compliance Update

July 18, 2016

Bipartisan PRICED Act Introduced in Congress

Three members of Congress, a bipartisan trio that spans both the House and the Senate, have introduced the Price Relief, Innovation, and Competition for Essential Drugs (PRICED) Act in the House of Representatives and the Senate on Friday, June 24. The bill points to higher medication prices and if passed, would reduce the data exclusivity period for biologic drugs from twelve years to just seven.

The bill, clocking in at a whopping two pages, would amend the Reference Product Exclusivity (RPE) provisions in the Public Health Service Act (PHS Act), striking "12 years" and inserting "7 years" in place. Conforming amendments would be made throughout the Act where necessary. The PRICED Act would apply only with respect to a biological product for which the reference product … is licensed under [PHS Act] on or after" the date of enactment of the PRICED Act.

Such a reduction would have a large effect on the biologic industry: it would mean significantly more competition for branded biologic manufacturers from the biosimilar drug makers. The current twelve-year data exclusivity threshold for biologic products was set under a compromise in the Affordable Care Act. Since that time, President Obama has advocated for reducing that period to seven years through budget proposals, in an effort to cut healthcare spending.

According to Representative Janice Schakowsky, the sponsor of the bill in the House of Representatives, the PRICED Act would "foster competition and provide opportunity for more biologics to enter the marketplace and drive down costs."

Senator Sherrod Brown believes that, "in the same way the entrance of generics helped increase competition and boost access to more affordable prescription drugs, an increased number of biologics and equally effective 'biosimilars' will provide additional competition in the marketplace and make life-saving drugs more affordable for consumers."

Senator John McCain opined that the "PRICED Act would inject much-needed competition in the biologics market, bring down costs for live-saving drugs, and save billions in taxpayer dollars."

Industry Reaction

Industry reaction has overall been very predictable: major trade groups oppose any efforts to shorten the period while generic groups support efforts. The Biotechnology Innovation Organization (BIO) argues that anything less than twelve years of exclusivity would have repercussions, i.e., stifled innovation, reduced drug access, and increased prices of drugs in the long run. They also noted that a "majority of biotechnology companies are small, private start-ups, heavily reliant on venture capital investment" and need the full twelve-year incentive.

The Generic Pharmaceutical Association (GPhA), on the other hand, argues that the legislation would "speed patient access to more affordable versions of some of the most expensive medicines" and that "as brand and specialty drug costs rise at a concerning rate, the association looks forward to working with Congress and others to ensure timely access to biosimilar medicines."

What Does This Mean

This bill is introduced in the midst of several other important, and related, discussions and events. The Trans-Pacific Partnership (TPP) currently under negotiations, has caused high-level discussions on the exclusivity period, as some countries have pushed for a shorter exclusivity period under the deal (e.g., Australia only allows for a five-year exclusivity period), which United States negotiators have resisted.

This bill also comes shortly after the Supreme Court's invitation to the Solicitor General to file briefs regarding the Sandoz and Amgen petitions for certiorari.

The likelihood that the PRICED Act will make its way through Congress and be signed into law by the end of President Obama's term is pretty low. Even still, the bill is yet another sign of the concern on Capitol Hill over drug prices.

July 05, 2016

Bipartisan Policy Center Holds Briefing on Health Innovation

At a briefing hosted by the Bipartisan Policy Center (BPC), Senate Health, Education, Labor, and Pensions (HELP) Committee Chairman Lamar Alexander confirmed that negotiations over the Senate "Innovation Initiative" are still underway. Senator Alexander noted that the Committee is "in the midst of discussions" with House Speaker Paul Ryan and Senate Majority Leader Mitch McConnell on how to advance the package.

Specifically, Senator Alexander highlighted a conversation he had with Senator McConnell, where both senators agreed that the medical innovation package could possibly be "the most important legislation that Congress acts on this year."

In his remarks, Chairman Alexander described the Innovation Initiative as a proactive investment to help cut down healthcare spending, citing the forward-looking success of President George W. Bush's President's Emergency Plan for AIDS Relief (PEPFAR) in helping to control the HIV/AIDS epidemic abroad.

Senator Alexander highlighted the need for a smooth transition to electronic health records, as precision medicine will not work without using advanced health data. He does not believe that the federal government's investment in EHR has not been spent very well, and that Congressional leaders are working together to help ensure future efforts are more efficient.

Senator Alexander addressed the critics of the Innovation Initiative, stating that the Senate needs to take steps to "make sure the horror stories don't derail the success stories."

Senator Alexander also used his time to highlight other issues, including combating the opioid abuse epidemic and how to improve opportunities for Americans to be treated with regenerative medicine.

New BPC Report

In connection with the briefing, the BPC published a new report, "Using Real-World Evidence to Accelerate Safe and Effective Cures." The report provides a set of recommendations on improving the use of data in data development and strengthening the Food and Drug Administration's (FDA's) ability to oversee the progress.

The recommendations focus on different ways the current drug development process could be modernized and improved through the use of real-world evidence, as well as policy action suggestions to implement those suggestions.

Among the proposals are measures to: improve the medical product development process, increase regulatory clarity, use health information technology to improve health care, and increase investment in medical products to address unmet and public health needs.


The recommendations are as follows:

Recommendation I: Improve Regulatory Clarity Regarding Use of Real-World Evidence

Current evidence requirements date back to 1962, when amendments to the Federal Food, Drug and Cosmetic Act (FDCA) included provisions requiring manufacturers of drug products to establish a drug's effectiveness by substantial evidence and adequate and well-controlled investigations before it could be approved for marketing. Since that time, several regulations and guidances have been published.

Included in the policy ideas for this recommendation were suggestions that the FDA should: develop formal guidance regarding the use of real-world evidence to inform regulatory decision-making, including the circumstances under which real-world data could be used as well as the types of real-world data, methods, and the levels of evidence that would be acceptable for use in regulatory review and decision-making. The guidance should include, but not be limited to, new drug approvals (including approvals under expedited programs), label expansions, new indications, post-market commitments, and post-market study requirements; and engage representatives of regulated industry, patient and disease research organizations, academia, experts in the use of electronic data, experts in statistical methods, and experts in privacy policy in the development of the guidance.

Recommendation II: Improve Methods and Data Quality for the Generation and Use of Real-World Evidence

The scope and amount of real-world data potentially available is rapidly expanding, as are the methods to effectively use and interpret the data for regulatory decision-making purposes. The methods and interpretations that have traditionally been used may not be the most appropriate methods to use for understanding larger data sets, or data drawn from across a network of disparate databases. It is therefore imperative that regulatory agencies and others who rely on the data educate themselves about best practices in methods of use and interpretation of real-world evidence for decision-making purposes.

This recommendation included suggestions for both the FDA and HHS, including:

  1. the FDA should establish a program to promote sharing and evaluation of methods used in the evaluation of real-world evidence for regulatory decision-making. The FDA should invite a broad spectrum of researchers who are active in the generation and use of real-world evidence and methods development, as well as leaders who rely upon such real-world evidence—including regulators and payers—to participate in this program.
  2. The U.S. Department of Health and Human Services (HHS) should support research to improve methods for the use of real-world evidence, which take into account the much larger samples of electronic data now available and enable high-throughput methods that produce accurate and well-calibrated inferences that quantify levels of uncertainty more accurately. Such research should focus on issues that include, but are not limited to, mitigating bias, obtaining solutions to better refine outcomes definitions, understanding implications to analyses for integrating observational data across a number of disparate sources, and understanding the contributions of real-world evidence to causal reasoning.

Recommendation III: Improve Policies for Information Sharing to Support Clinical Research

Under current law, in order to conduct one real-world study across multiple health care systems, multiple institutional review board (IRB) approvals are required. Given the differences in how IRBs view their remit and what constitutes a clinical trial, a unique and individualized approach is often needed to seek approval from each, which can lead to delays in trial execution and increased costs.

In light of that, two suggestions were made to Congress, including the idea that Congress should require the HHS Secretary—through the OHRP and the FDA—to issue regulations and guidance to facilitate the broader use of centralized IRBs within 36 months, by clarifying the roles of IRBs in multi-site studies and the risks and benefits to human subjects, standardizing informed consent, and incorporating community values through the use of local IRBs while continuing to use central IRBs.

Recommendation IV: Explore New Adaptive Pathways to Modernize Drug Development and Support a New Era of Personalized Medicine

As medicine continues to become more personalized and drugs become targeted for smaller populations, traditional, large-scale RCTs will become increasingly less feasible and additional approaches will be needed to assure safety and efficacy and protect the public's health. Rapid advances in technology and personalized medicine will allow for more-close monitoring to be easier, cost effective, and accurate. To advance the exploration of a new, more flexible adaptive approach to drug approval, several steps must be taken.

Included in those steps are the following suggestions:

  1. The FDA should develop a new program to develop and test a new adaptive pathway approach to expand the capacity for drug development that has the following key attributes: iterative phases of development, beginning with initial marketing authorization to a restricted patient population, then expanding to wider populations based on risk-benefit ratios; gathering evidence through close-monitoring and other real-world evidence, to supplement RCTs; and early involvement of stakeholders who have a role in determining patient access to the drug, including industry, payers, regulators, clinicians, and patients.
  2. The FDA's new program to develop and test a new adaptive pathway approach for drug development should include the following elements: qualifying criteria for the program, which will determine which types of drugs at what stages could be considered for the adaptive pathway approach; types and levels of evidence required for initial approval and expansion, including evidence generated from close-monitoring, other real-world evidence, and randomized controlled trials, as appropriate; methods for early involvement of patients, clinicians, payers, industry, and regulators; and methods for assuring market removal or label modification of products when follow-up studies and monitoring are not completed or when an unfavorable risk-benefit ratio for certain populations is demonstrated.


June 29, 2016

Markey Calls for Investigation into Purdue

United States Senator Edward J. Markey has called for a federal investigation of OxyContin's manufacturers in response to a Los Angeles Times report. The report found that the drug tends to wear off early in many patients, which can expose them to an increased risk of addiction.

According to the LA Times, OxyContin's main selling point is that it lasts for twelve hours. The investigation found that "when the effects don't last, patients can suffer symptoms of a narcotic withdrawal, including intense craving for the drug, and experience a cycle of agony and relief that experts say promotes addiction."

The report states that "Purdue has known about the problems for decades. Even before OxyContin went on the market, clinical trials showed many patients weren't getting 12 hours of relief. Since the drug's debut in 1996, the company has been confronted with additional evidence, including complaints from doctors, reports from its own sales reps and independent research."

Allegations included in the report include the idea that Purdue has held fast to the claim of twelve-hour relief to protect its revenue. They allege that OxyContin's market dominance and high price rely on the twelve-hour duration and without such a claim, the drug offers little advantage over less expensive painkillers. The report also alleged that Purdue told doctors to prescribe stronger doses, not more frequent ones, if patients complain that OxyContin doesn't last twelve hours.

In two letters, Senator Markey urged the Department of Justice (DOJ), and the Food and Drug Administration (FDA) and the Federal Trade Commission (FTC) to launch probes into Purdue Pharma. As a result of the LA Times report, Senator Markey believes that Purdue made false claims about the longevity of OxyContin's pain relieving properties.

Senator Markey posited that after investigation, if the allegations included in the LA Times report are found to be substantiated, the DOJ should take legal action against the Purdue Frederick Company, Inc., as well as its pharmaceutical subsidiary Purdue Pharma, L.P., including recouping damages that were sustained by federal healthcare programs that paid for excess OxyContin prescriptions that should have been unnecessary if the drug lasted for the full twelve hours, as Purdue claimed.

The letter to the DOJ also cites to a 2007 criminal proceeding against Purdue, in which Purdue "pled guilty to a felony charge of illegally misbranding OxyContin in an effort to mislead and defraud physicians and consumers." In that proceeding, Purdue admitted to illegally marketing and promoting OxyContin by falsely claiming that it was less addictive, less subject to abuse and diversion, and less likely to cause withdrawal symptoms than other pain medications. Purdue agreed to pay over $600 million in civil fines, penalties, and forfeitures in connection with that case. Separately, three Purdue executives pled guilty to misdemeanor charges of misbranding OxyContin and collectively agreed to pay $34.5 million in penalties.

The letter to the FDA and FTC urged the groups to immediately take action that would prevent the dissemination of misleading or false information about OxyContin's duration to those making prescribing decisions, and to patients. Senator Markey also asked the organizations to "warn prescribers, patients, and the general public about the dangerous impact of using higher or more frequent dosing to compensate for OxyContin's failed claims."

Senator Markey's claims and call for an investigation are just the latest in the onslaught on opioid drug makers and opioid abuse. Both the FDA and CDC have changed their prescribing outlines for the drugs; and there is evidence that those changes and the backlash over their widespread use is beginning to cut into sales of the drugs.


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