Catalyst Corner


The 21st Century Cures Act legislation, H.R. 6, passed the U.S. House of Representatives on July 10, 2015, with a vote of 344 to 77. The act’s intent is to remove barriers to innovation, allowing the rapid pace of scientific discovery we are experiencing to be more rapidly translated into cures for patients. In announcing the passage, the bill’s authors said, “Today, we took a big leap on the path to cures, but we still have much work left to do.” The authors include House Energy and Commerce Committee Chairman Fred Upton (RMich.), and Reps. Diana DeGette (D-Colo.), Joe Pitts (R-Pa.), Frank Pallone, Jr. (D-N.J.) and Gene Green (D-Texas)

If you have not been following the legislation, it has been under development for more than a year by the chief sponsor, Fred Upton. The first discussion draft was released in January of this year and weighed in densely at more than 400 pages after the committee held numerous roundtables and hearings and issued several white papers. While I have yet to find a concise summary of more detailed issues that may impact the pharmacy arena, the overall scope of the legislation itself, with an emphasis on speeding new medications to market, will certainly be significant to the profession and its partners and vendors.

  Documents to Know
  »The Congressional Budget Office (CBO) cost estimates.
»A section-by-section summary of the 21st Century Cures Act.

Sponsor Upton, in a two-page summary sheet framing the act, writes, “The pace of scientific advancement over the past two decades, including the mapping of the human genome, has been impressive, giving us a myriad of genetic clues about the underpinnings of disease. Translating these discoveries into new treatments for patients, however, has proven to be difficult. H.R. 6 accelerates the discovery, development and delivery of life saving and life improving therapies, and transforms the quest for faster cures . . .” According to the summary, the act addresses several key areas, including:

➤Removing barriers to increased research collaboration by breaking barriers to sharing and analyzing the growing amount of health data generated in research and clinical settings.

➤Incorporating the patient perspective into the drug development and regulatory review process by strengthening the FDA’s ability to take the direct experience of patients with particular diseases and conditions, and the effect of their current therapies, and use that data to modify and improve potential treatments.

➤Measuring success and identifying diseases earlier through personalized medicine by providing guidance for the broader, more collaborative development, understanding, and utilization of drug development tools such as biomarkers, which can be used for earlier assessment of how a particular therapy is working and on whom.

➤Modernizing clinical trials through more targeted trials based on personalized medicine that can produce results faster and cheaper. Areas include greater use of patient-generated registries to speed enrollee recruitment, screening patients in advance to determine if their genetic predisposition makes them better candidates for targeted therapies, and allowing use of new and creative adaptive trial designs that deploy the most modern statistical and data tools while significantly reducing existing, duplicative, or unnecessary paperwork requirements.

➤Removing regulatory uncertainty for the development of new medical apps that generate real-time patient data.

➤Providing new incentives for the development of drugs for rare diseases.

➤Helping the entire biomedical ecosystem coordinate more efficiently to find faster cures by creating a new coordinating mechanism to remove areas that slow the connections between scientific discovery, drug, and device development, and how these therapies are approved and made available to patients.

➤Investing in 21st century science and next-generation investigators. H.R. 6 creates the “Innovation Fund,” a dedicated and offset funding stream of $1.75 billion per year for five years for the NIH and $110 million per year for five years for the FDA that will allow congressional appropriators to invest additional resources without impacting current budget caps, and with provisions to invest more resources in the next generation of scientists for the next generation of drugs.

Of particular interest to technology vendors are sections that fund continued efforts to ensure health record and system interoperability through the Office of the National Coordinator for Health Information Technology (ONC) and supporting telehealth programs under Medicare.

Upton says that the act will help keep and create jobs here at home, and writes, “The United States has led the global medical device and biopharmaceutical industries for decades, helping us become the medical innovation capital of the world and causing China and others to try to take our innovation and jobs. Because of our leadership, U.S. medical device-related employment totals over 2 million jobs, and the U.S. biopharmaceutical industry is responsible for over 4 million U.S. jobs. NIH funding currently supports over 400,000 jobs at research institutions across the country, including jobs for young scientists. The policies in H.R. 6 will help us fight off foreign competitors so we can keep these jobs, and add more, here at home.”

In addition to targeting orphan drug development, the bill specifically targets innovation for antibiotics and vaccines. Further, it has provisions related to prescription drug abuse and diversion, to prevent unintended consequences that may threaten legitimate patient access to needed medications.

Of particular interest to technology vendors are sections that fund continued efforts to ensure health record and system interoperability through the Office of the National Coordinator for Health Information Technology (ONC) and supporting telehealth programs under Medicare.

The National Association of Chain Drug Stores (NACDS) has been actively involved in the legislation’s evolution and noted in a July 10 press release that several provisions related to AMP (average manufacturer price) calculations were impacted by its efforts. Specifically, they write, “NACDS successfully advocated to prevent a provision that would have excluded brand and authorized generic drugs from the calculation of average manufacturer price (AMP) in setting federal upper limits for generic drug matching rates under Medicaid. NACDS noted that this provision could have jeopardized the patient access that is at the heart of the legislation.” The association also notes that “House Energy and Commerce Subcommittee on Health Chairman Rep. Joe Pitts (R-Pa.) submitted a statement for the record of the proceedings of the U.S. House that clarified that a subsequent and final AMP-related provision in the legislation ‘is not intended to affect Medicaid programs’ pharmacy reimbursements.’”

The Congressional Budget Office (CBO) released its cost estimates on the legislation June 23, 2015. (See https://www.cbo.gov/sites/default/files/114th-congress-2015-2016/costestimate/hr6.pdf.) Federal spending is expected to be reduced by $470 million through 2025. Funding for the legislation will come from crude oil sales and reductions in Medicare spending, including:

➤The sale of up to 10% of the United States’ crude oil from the Strategic Petroleum Reserve between fiscal 2018 and fiscal 2025.

➤Limiting federal reimbursement to state Medicaid programs for durable medical equipment payments, which may increase states’ Medicaid costs by an additional $2.6 billion, along with provisions that would delay entry of generics.

➤Delaying payments to stand-alone prescription drug plans beginning in 2020, which effectively shifts spending from fiscal 2025 to fiscal 2026.

A section-by-section summary of the 21st Century Cures Act is available here: http://energycommerce.house.gov/sites/republicans.energycommerce.house.gov/files/114/20150702HR6SectionBySection.pdf. Given the broad support and unique way the legislation was crafted, I encourage ComputerTalk readers to delve into the bill and see how it may more specifically impact you. That said, the bill is not without controversy, as some blogs and press reports from consumer advocates and researchers reflect the view that the provisions may make the drug approval process less safe and more risky. CT


Marsha K. Millonig, R.Ph., M.B.A., is president of Catalyst Enterprises, LLC, in Eagan, Minn. The firm provides consulting, research, and writing services to help healthcare industry players provide services more efficiently and implement new services for future growth. The author can be reached at mmillonig@ catalystenterprises.net.