False promises rebuked by FDA, no tea or vitamin can cure cancer

Bogus cancer “treatments” being marketing and sold without FDA approval were the target of 14 warning letters and four online advisory letters, according to a press release and consumer update from the agency. The 65-plus products listed by the agency include pills, tablets, creams, syrups, sprays, oils, salves, teas, and medical devices, claiming to cure cancer in humans and pets, and have been found illegally for sale online, in retail stores, at flea markets and swap meets, and even at trade shows.

The FDA called these illegal products a “cruel deception,” and urged consumers to stay away from products that have not passed the agency’s review process, designed to ensure the safety and effectiveness of treatments. It listed the following phases or concepts as warning signs that the advertised product was unlikely to be approved by the agency:

  • treats all forms of cancer;
  • miraculously kills cancer cells and tumors;
  • shrinks malignant tumors;
  • selectively kills cancer cells;
  • more effective than chemotherapy;
  • attacks cancer cells, leaving healthy cells intact; or
  • cures cancer.

Additionally, many of the products that were the subject of the warnings were advertised as “natural” or “non-toxic.”

The warning and advisory letters ask the recipient companies to provide written responses to the violations covered in the letters; if the companies fail to respond and make adequate corrections, they could be subject to further actions including criminal prosecution. According to the FDA, the best scenario for consumers who have purchased or used these products is ineffectiveness. It is possible, however, that these products could interfere with proven, beneficial treatments, or even cause direct harm.

PDUFA VI reauthorization would aid 21st Century Cures Act implementation

Since 1992, the Prescription Drug User Fee Act (PDUFA) has authorized the FDA to collect user fees from biopharmaceutical manufacturers to supplement Congressional appropriations. Revenues from these fees are used on activities related to the review and regulation of new drug products. In exchange for these fees, the FDA commits to meeting certain performance goals, such as reviewing applications within specified timeframes. The FDA’s ability to collect these fees must be reauthorized every five years. Each five-year reauthorization sets a total amount of fee revenue for the first year and provides a formula for annual adjustments to that total based on inflation and workload changes.

On March 22, 2017, the House Energy and Commerce Committee’s Subcommittee on Health held a hearing to examining the PDUFA program. PDUFA, as reauthorized by the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA) (P.L. 112-144), expires in September 2017, and must be reauthorized for the fiscal years 2018 to 2022.

This will be the sixth reauthorization of PDUFA. The proposed agreement (PDUFA VI), builds upon process improvements enacted pursuant to FDASIA, including enhanced support for the Breakthrough Therapy Program. Further, it would aid in the implementation of several key provisions in the 21st Century Cures Act and further streamline the development and review of innovative new drugs for patients. The FDA estimates that the fees negotiated in PDUFA VI will average approximately $1 billion per year.

At the hearing, the following individuals testified on how the program has been implemented to date and presented recommendations pertaining to its reauthorization:

Allen

In his testimony, Allen pointed out that: “Prior to the initial user-fee authorizations, patients in other parts of the world were gaining access to new medicines faster than Americans, with only about 10 percent of new treatments reaching U.S. patients first.” That paradigm has largely been reversed, according to Allen. “Between 2003 and 2016, 73 new cancer drugs were approved by both the FDA and EMA [European Medicines Agency]. Of those drugs, 97 percent (71 of 73) were available in the U.S. before Europe. Furthermore, the FDA approved new cancer drugs on average nearly 6 months faster than the EMA.”

Allen also stated that PDUFA VI:

  • Advances the role of patients and their experiences;
  • supports the continued success of the Breakthrough Therapy Designation, a designation that may be given to a drug intended to treat a serious illness for which preliminary clinical evidence indicates a substantial improvement over any existing interventions. To date, 170 Breakthrough Therapy Designations have been granted, leading to 79 indications approved by the FDA using this process;
  • promotes qualifications and the use of drug development tools;
  • enhances the use of real-world evidence in regulatory decision-making; and
  • effectively communicated scientific advances.

Allen cautioned, however, that “proposed cuts to biomedical research will put the brakes on the engine of discovery, abandon progress on new tools to enhance product evaluation, impede opportunities for new businesses in the biotech sector, and most perilously, jeopardize the development of new medicines for patients desperate for progress.”

Pritchett

In supporting PDUFA VI reauthorization, Pritchett stated: “For nearly twenty-five years, PDUFA has provided much needed resources to the FDA’s human drug review program that has resulted in greater certainty and predictability for patients who depend on safe and effective innovative medicines.” Pritchett also noted the following benefits under PDUFA:

  • The FDA has approved over 1,500 new drugs and biologics since 1992, including treatments for cancer, cardiovascular, neurological, infectious and rare diseases.
  • The number of new medicines being approved on their first review cycle is at a historic high, including approvals for new medicines to treat rare diseases.
  • Review times for drug applications have dropped by nearly 55 percent.
  • The median approval time for standard applications has decreased from 22.1 months in 1993 to an estimated 10 months in 2015.
  • The median approval time for priority applications has similarly decreased from 13.2 months in 1993 to an estimated 7.9 months in 2014.

Pritchett concluded: “At a time when the U.S medical innovation ecosystem is facing severe strains and increased global competition, it is imperative that the FDA is equipped to help us deliver the next generation of new treatments and cures to meet patients’ unmet medical needs. PDUFA VI will help the FDA ensure that patients receive effective and lifesaving drugs, while maintaining the United States’ global leadership in biomedical innovation.”

Holcombe

Holcombe’s testimony cautioned Congress on the cost of the program: “Since 2002, the PDUFA program has grown at an average of 11 percent per year; this is unsustainable moving into the future. Changes are needed that address the fee collection structure to increase efficiency and reduce administrative burdens for both FDA and companies.”

Holcombe believes that the proposed PDUFA VI agreement would address these concerns by:

  • limiting the carryover balance levels, thus reducing possible over-collection of fees and the need for complicated administrative mechanisms to deal with such over-collections;
  • eliminating supplement fees, which will further simplify fee collections;
  • replacing the current product and manufacturing fees with a new program fee that will constitute 80 percent of the annual fee collections; and
  • reducing the percentage that application fees contribute to the total from the current 33 percent to 20 percent, thus mitigating the overall impact of this difficult-to-predict revenue source.

Holcombe also pointed out the benefits of important overlaps between provisions in the 21st Century Cures Act and the proposed PDUFA VI agreement. She offered the following examples of overlap:

  • The 21st Century Cures Act and PDUFA VI are complementary, in terms of ensuring that FDA (1) has and uses effectively an efficient process for qualifying biomarkers; (2) publishes guidance to help applicants for biomarker qualification understand the taxonomy and data standards; (3) makes public a list of qualified biomarkers and pending applications; and (4) engages external experts in biomarker qualification.
  • Patient-focused drug development. Guidance development, public meetings, development of methods and standards for collecting information and data, and use of patient perception and experience information in the FDA regulatory decision about the benefits and risks of a drug are all elements of both 21st Century Cures and the PDUFA VI agreement.
  • Real-world evidence. The 21st Century Cures Act provides helpful context for the work under PDUFA VI, and provisions of the two that differ are easily harmonized.
  • Innovative trial design. While the 21st Century Cures Act focuses on adaptive trials and Bayesian approaches, PDUFA VI takes a broader approach, opening its pilot program to other trial designs while also highlighting adaptive trials and Bayesian approaches.

Holcombe concluded by indicating the Biotechnology Innovation Organization strongly supports and applauds the enactment of 21st Century Cures, and it strongly supports the PDUFA VI proposed agreement.

Woodcock

At the hearing, Subcommittee Vice Chairman Brett Guthrie (R-Ky) asked Woodcock for an update on the FDA’s Oncology Center of Excellence, a key component of 21st Century Cures and a committee-supported initiative. Woodcock elaborated on the center’s structure and the important work it will be doing.

With regards to PDUFA VI, Woodcock noted: “The PDUFA VI reauthorization proposal . . . was submitted to Congress in December under the previous Administration, and reflects a different approach to the federal budget.” She also stated: “Center to PDUFA VI, and its largest single investment component, are plans to elevate patient voices in developing new drugs to treat their diseases. The agreement shares the committee’s goals reflected in the 21st Century Cures Act – and the highest priority of our stakeholders – to leverage essential patient input and insights to fight disease.”

FDA considers establishing a new ‘Office of Patient Affairs’

The FDA announced that it is establishing a public docket to solicit public input on ongoing efforts to enhance mechanisms for patient engagement at the agency. In addition, to achieve a more transparent, accessible, and robust experience for patient communities, the FDA is considering establishing a new Office of Patient Affairs.

On November 4, 2014, the FDA established a docket (FDA-2014-N-1698) for the public to submit information related to the FDA’s implementation of the Food and Drug Administration Safety and Innovation Act (FDASIA) (P.L. 112-144), Patient Participation in Medical Product Discussions under FDASIA section 1137.

Based on the comments received, the FDA identified objectives for its patient engagement activities. First, to develop a nuanced understanding of the patient experience of disease by: (1) gathering patient perspective on what is clinically meaningful; (2) assessing attitudes towards benefit-risk and tolerance of uncertainty; and (3) enhancing the science of eliciting and integrating patient input.

Second, to support patients and their advocates in understanding regulatory processes and navigating the FDA by: (1) communicating relevant FDA positions, procedures, and activities; (2) connecting patients and their advocates with the appropriate resources; and (3) resolving discrete challenges and needs.

To achieve these objectives, the FDA is considering establishing a central “Office of Patient Affairs.” The responsibilities of this central office would include:

  • offering a single, central entry point to the FDA for the patient community;
  • providing triage and navigation services for inbound inquiries from patient stakeholders;
  • hosting and maintaining robust data management systems that would incorporate and formalize knowledge shared with the FDA by patient stakeholders and the FDA’s relationships with patient communities; and
  • developing a scalable and forward-looking platform for communicating with patient stakeholders, particularly online channels.

The Office of Patient Affairs would be directly accountable to the medical product Centers. A regular evaluation of this central office and of FDA’s overall patient engagement efforts are also proposed.

 

 

Trump nominates Gottlieb for FDA Commissioner

President Trump intends to nominate Scott Gottlieb, M.D., a resident fellow at the conservative American Enterprise Institute (AEI), clinical assistant professor at New York University School of Medicine, and a member of the HHS Federal Health IT Policy Committee, to the post of FDA Commissioner. The White House announced the nomination, which brought varied reactions from opposite sides of the aisle and a general positive response from the pharmaceutical industry, via a tweet from Press Secretary Sean Spicer.

Gottlieb served as the Deputy FDA Commissioner from 2005 to 2007 and previously served as a senior official at CMS. He has testified before Congress on numerous occasions as an AEI felllow, most recently with respect to EpiPen® price increases and “How Regulatory Barriers Inhibit Pharmaceutical Competition.” Gottlieb noted that FDA regulatory policy has made developing less expensive copies of complex drugs after patent expiration difficult, discussed how the 340B program has put “upward pressure on drug prices,” while noting other change in drug insurance coverage structure, and described obstacles to competitive single source drug pricing.

Various sources report that Gottlieb has close ties to the pharmaceutical industry. Scientific American noted that Gottlieb believes in a quicker approval process for new drugs, but has focused on shortening waiting times for large, clinical trials rather than doing away with efficacy considerations. He commented on this, to an extent, in remarks he made at the 21st Annual International Meeting of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) in May 2016.

Gottlieb has also issued commentary about the Patient Protection and Affordable Care Ac (ACA) (P.L. 111-148). In May 2016, he testified before Congress that the law’s tiered marketplace approach has aided consumers with plan selection, but has forced insurers into narrow design corridors. His testimony regarding the ACA also included a suggestion that CMS move away from mandates and towards incentives to encourage individuals to enter into the insurance market (see Is there a better way than the ACA? Hearing asks experts, Health Law Daily, May 12, 2016). More recently, he coauthored a piece with another AEI fellow, opining that President Trump’s election provided, “a generational opportunity to pursue a new direction for American health care” and making suggestions about how a new health care system should operate. The authors suggested that the system should provide a path to catastrophic health insurance for all, accommodate individuals with pre-existing health conditions, allow access to health savings accounts, and deregulate the medical services market.

Senator Lamar Alexander (R-Tenn), Chairman of the Committee on Health, Education, Labor & Pensions, touted Gottlieb’s “impressive qualifications” in a released statement. His colleague, Ranking Member Patty Murray (D-Wash), expressed “initial concerns” about the nomination, including Gottlieb’s “work with multiple pharmaceutical companies, medical device companies, and investment firms.”