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The #21stCentury Cures Act, Big #Pharma and the #FDA

The #21stCentury Cures Act, Big #Pharma and the #FDA

For more information, please contact The Ahearne Law Firm, PLLC for a free initial consultation and case evaluation. Call nationwide at (845) 986-2777.

Late last year, Congress passed the 21st Century Cures Act to accelerate “discovery, development and delivery” of medical therapies by encouraging biomedical research investment and facilitating innovative review and approval processes.

The provisions of the Act relating to pharmaceuticals are favorable to sponsors and manufacturers, and impose additional requirements on the United States Food & Drug Administration (FDA). The Act seeks to:

  • Expedite the review process for certain drugs
  • Facilitate the recognition of drug outcome measures
  • Encourage the consideration of data beyond that produced in randomized clinical trials to support approval
  • Create a new priority review voucher (PRV) for material threat medical countermeasures
  • Extend the current rare pediatric disease PRV program
  • Clarify the scope of permissible dissemination of health care economic information (HCEI) by manufacturers
  • Require manufacturer publication of expanded access policies.

The Act does not modify the statutory standard for the approval of a new drug or biologic. However, the Act includes several provisions that may impact the types of evidence the FDA will consider when deciding whether individual products meet the statutory standard. Further, the review and approval process for certain types of drugs has also changed:

  • For a sponsor’s drug to be designated by the FDA as a regenerative advanced therapies (RAT) — e.g., cell therapies, therapeutic tissue engineering products, human cell and tissue products — the drug must be intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence must indicate that the drug has the potential to address unmet medical needs for such disease or condition. The FDA may also issue updated guidance and regulations within one year of developing standards to support the development, evaluation and review of regenerative medicine therapies and RATs.
  • A new pathway is designed to expedite approval of antibacterial and antifungal drugs intended to treat serious or life-threatening infections in a limited population of patients with unmet needs, without requiring large-scale clinical trials or testing in specific populations. The labeling and advertisements for such drugs must contain a statement that the drug’s safety and effectiveness has only been demonstrated with respect to a “limited population,” and the promotional materials for such drugs must be submitted to FDA prior to dissemination.
  • For genetically targeted or variant protein targeted drugs — drugs for the treatment of rare diseases or serious or life-threatening conditions which, respectively, may modulate the function of a gene or modulate the function of a product of a mutated gene — the FDA may permit a sponsor to rely on data previously developed and submitted by the sponsor (or another sponsor, with the appropriate right of reference). The law is intended to address the challenge of conducting clinical trials in small populations of patients, especially subgroups of patients with the same disease or condition but different genetic mutations. To be eligible under this provision, drugs must incorporate or use the same or similar technology as the drug in the previously approved application.
  • A new PRV program entitles the holder of a PRV to expedited FDA review of a subsequent drug product application within six months (four months faster than the standard review process) for sponsors of material threat medical countermeasures. PRVs issued under this program are also transferable, which may lead to further development of “material threat” medicines.
  • Manufacturers who disseminate health care economic information (HCEI) related to drugs and medical devices will not be subject to claims for false or misleading labeling if it (1) is disseminated to persons to whom such information may be communicated, (2) relates to approved indications and is based on competent and reliable scientific evidence, and (3) includes a “conspicuous and prominent statement describing any material differences” between HCEI and FDA-approved labeling. The Act expands the population to whom HCEI may be communicated, and the analysis types that may be communicated, and lessens the prior requirement that HCEI “directly relate” to approved indications with simply a “relate” standard.
  • The Act prohibits the FDA from determining that a combination product’s primary mode of action is that of a drug or biologic solely because the combination product has any chemical action within or on the human body. Chemical action in the body is a statutory concept that distinguishes a drug or biologic from a medical device. The Act requires FDA to determine how a combination product will be regulated based on a less-restrictive statutory definition of “primary mode of action,” which focuses on the single mode of action expected to make the greatest contribution to the overall intended therapeutic effects of the combination product.
  • Manufacturers or distributors of investigational drugs for serious diseases or conditions must make their expanded access policies on requests for such drugs publicly available within 60 days of the later of the law’s enactment or the initiation of a phase 2 or phase 3 study of an investigational drug.

The Act may be welcome relief to pharmaceutical manufacturers as the FDA approved 22 new pharmaceuticals medicines for sale in 2016 — the lowest number since 2010. In contrast, the European Medicines Agency recommended 81 new prescription products (including generics). Reasons for the decline included less drugs being filed for approval, and the FDA rejecting or delaying more applications in 2016 than in the previous two years.

Given resistance to the rising cost of medical treatment from healthcare insurers, and increasing political pressure over the high prices of pharmaceuticals, it is difficult to get new drugs approved and to obtain significant financial return once launched. Returns on research and development investment at the top 12 pharmaceutical companies fell to just 3.7% in 2016. As a result, biotech and pharmaceutical companies are developing more drugs targeted at niche patient populations.

If you or a family member have suffered injury after taking prescription or over-the-counter pharmaceuticals or drugs, you and/or your family member may be entitled to money damages.

For more information, please contact The Ahearne Law Firm, PLLC for a free initial consultation and case evaluation. Call nationwide at (845) 986-2777.

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