Science Policy For All

Because science policy affects everyone.

21st Century Cures Initiative

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By: Danielle Friend, PhD

One year in the making, with several hearings, roundtable discussions, and white papers released, the 21st Century Cures Initiative is nearing completion. The bill has been spearheaded by the conservative Representative, Fred Upton (R-Mich.) and the more liberal Representative Diana DeGette (D-Colo.), both of whom are members of the Energy and Commerce Committee. While biomedical research has advanced at what seems like lightning speed, unfortunately the translation of research into treatments and cures has been slow and costly. This revolutionary new bill aims to update the regulatory processes and to keep legislation readily informed of new developments in the biomedical sciences so that patients see treatments faster.

During the early phases of the 21st Century Cures initiative, the Health Subcommittee Chairman Joe Pitts (R-P.) organized hearings, roundtable discussions, and white papers to collect feedback on the bill from health care stakeholders, patients, manufacturers, medical associations and research institutions. “While increasing accountability, this legislation would invest in the basic research so critical to equipping our nation’s best and brightest with the tools they need to discover the underpinnings of disease”, he stated. The most updated version of the bill was released on April 28, 2015 and following the release, the Heath Subcommittee held a legislative hearing to receive testimony regarding the new bill. Dr. Kathy Hudson, Deputy Director for Science, Outreach, and Policy at the National Institutes of Health (NIH), Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research at the U.S. Food and Drug Administration (FDA), and Dr. Jeff Shuren, Director of the FDA Center for Devices and Radiological Health all testified in support of the bill. On May 6, 2015 the committee also hosted a meeting of biomedical leaders, including NIH Director Francis Collins, in the first public discussion regarding the state of biomedical innovation in the United States.

What should you know about the bill?

The new draft of the bill comes in at approximately 200 pages (which is, surprisingly, about half the size of the previous draft!). The current draft is divided into three sections: Discovery, Development, and Delivery. Discovery generally focuses on the budget and organization of the NIH. Development focuses on the FDA’s regulation of clinical trials, drug development and precision medicine. Currently, Delivery focuses on changes to the Social Security Act and Medicare, however much of this section is still being drafted. Below are some of the most important highlights and take-home points from the current draft of the bill.

Title 1: Discovery

Title I of the new bill primarily concerns the organization and budget of the NIH. In fact, through the “NIH Innovation Fund”, the new draft increases the NIH budget by $2 billion per year for five years beginning in fiscal year 2016. The current NIH budget is approximately $30 billion a year, and an additional $2 billion each year is a small but significant increase. Although the programs that the “NIH Innovation Fund” will support are not yet defined, they may include precision medicine and programs for young emerging scientists, like those included in the Capstone Award (Section 1061). More details and definitions of the “NIH innovation fund” are surely to come.

The remainder of Title 1 focuses on the organization and administration of the NIH and the ways in which biomedical innovation can be better translated to the clinic. For example:

  • Subtitle E, “Promoting Pediatric Research Through the National Institutes of Health” states that the “National Institutes of Health should encourage a global pediatric clinical trial network through the allocation of grants, contracts, or cooperative agreements to supplement the salaries of new and early investigators who participate in the global pediatric clinical trial network,” with the goal of combining resources to combat pediatric diseases and birth defects.
  • Subtitle F, “Advancement of National Institutes of Health Research and Data Access” calls for data “generated through NIH-funded research” to be standardized and made available to other researchers.
  • Subtitle G, “Facilitating Collaborative Research” calls for the establishment and release of de-identified clinical trial data from “qualified clinical trials,” including trials on drugs and medical devices so that this data can be used for future discovery. Additionally, this section establishes a national neurological diseases surveillance system.
  • Subtitle H, “Council for 21st Century Cures” establishes a “nonprofit corporation to be known as Council that consists of a public-private partnership” with “the purpose … to accelerate the discovery, development, and delivery in the United States of innovative cures, treatments, and preventive measures for. ”

Title II: Development

As mentioned above, the overall purpose of the bill is to accelerate the speed with which discovered treatments reach the patient in the clinic. Title II of the new bill focuses on ways in which this may occur through the help of the FDA.

  • Subtitle B, “Qualification and Use of Drug Development Tools” aims to establish new biomarkers and other endpoints to accelerate the approval of new treatments. The bill states “The development of biomarkers and other drug development tools can benefit the availability of new medical therapies by helping translate scientific discoveries into clinical applications.” The bill also establishes that the FDA will develop guidelines regarding the use of these biomarkers. The legislation would require FDA and a sponsor to enter into an “accelerated approval development plan” to support the approval of a drug using surrogate endpoints.
  • Subtitle C, “Advancement of Precision Medicine” establishes that the FDA will produce guidelines regarding the definition of a “precision drug.” This part of the bill also establishes that the FDA will release guidelines on investigations can be designed to answer specific questions about narrow sub-populations of patients.
  • Subtitle D, “Modern Trial Design and Evidence Development” aims to accelerate clinical trials. One way this will occur is through the development of FDA guidelines for clinical data collected outside clinical trials, which may include observational trials, product registries and therapeutic use, according to the bill.
  • Section E, “Expediting Patient Access” overhauls “compassionate use”, allowing patients’ easier access to experimental drugs. This policy designates that drug companies company have a point of contact who will process patients’ requests, details of the procedures for making an expanded access request, the criteria for enrolling in a trial and the amount of time the company expects to take to process a request.
  • Section G, “Antibiotic Drug Development” creates a “limited population pathway” for antibacterial and antifungal drugs. This will allow sponsors to seek approval for a product intended to treat “a serious or life-threatening disease, condition or indication” that is currently not adequately served by existing therapies. FDA is also required to set up a website to provide recommendations on which bacteria/fungi are susceptible to specific drugs.
  • Subtitle K, “Priority Review for Breakthrough Devices”, establishes that medical devices are eligible to receive “breakthrough” designation by FDA and provides priority review for these devices. “Breakthrough devices” are those which represent “breakthrough technologies,” are intended to treat conditions “for which no approved alternative exist,” offer “significant advantages over existing approved or cleared alternative,” and are “otherwise in the best interest of patients.”
  • Subtitle L, “Medical Device Regulatory Process Improvements” aims to make the approval process for medical devices faster and easier. For example, in this section, companies would be allowed to make recommendations for the types of expertise FDA should include on an advisory committee panel.

Lastly, much of Title III, Delivery, is currently still being drafted and the Energy and Commerce Committee will be adding sections covering interoperability and telemedicine soon. We’ll look forward to reading the completion of these sections in the near future.

Developing effective treatments and cures for disease is a bipartisan, national priority. However, in order for biomedical discoveries to be translated to the clinic in an efficient and cost effective manner, regulatory processes to facilitate that must keep up to speed. The new 21st Century Cures Initiative aims to do just that. In the developing phases of the bill, Representatives Upton and DeGette explored issues that occur during the discovery of clues to treatments in basic science, during the development of these new treatments, and during the delivery of the identified cures. The current draft of the bill has developed policy to accelerate the regulatory processes involved and to facilitate each of these steps on the road to clinical treatments and cures.


Written by sciencepolicyforall

May 13, 2015 at 9:00 am

Posted in Essays

Tagged with ,

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  1. […] yesterday to approve the bills. The bills form a companion to the House of Representative’s 21st Century Cures Initiative, which aims to encourage medical breakthroughs by reforming the National Institutes of Health (NIH) […]

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