• <div class="header-image" style="background-image: url(/live/image/gid/4/2635_V6N3_Header.rev.1522162449.jpg);">​</div><div class="header-background-color"/>

21st Century Cures Act: Advancement or Detriment to U.S.’s Drug and Medical Device Approval Process

August 11, 2015
On July 10th, the 21st Century Cures Act (H.R. 6) passed a vote in the U.S. House of Representatives and now heads to the Senate. The bill hopes to encourage more innovation in drug development in addition to speedier approval processes for new drugs and medical devices.  However, some of the bill’s provisions could have a profound effect on what is known about the safety and efficacy of medical products.

By Alyssa Kennedy, C’17

As a result, this could dramatically alter the regulation of ways pharmaceutical and medical device products are regulated in the U.S. As this bill moves forward in Congress it is important to understand how the provisions of the bill could affect patients’ healthcare.

One very promising aspect of the bill calls for annual increases to the National Institute of Health’s (NIH) budget to about 3% per year for 3 years (when adjusted for inflation)[1]. The bill would also appropriate $2 billion a year in fiscal 2016 through 2020 for a new “Cures Innovation Fund” to supplement research funding for the NIH and Food and Drug Administration (FDA) [2]. However, this figure falls short of the $10 billion that the House Committee on Energy and Commerce approved in May[3]. Yet this overall increased support will help counteract the effects of lowered funding and budget cuts the NIH has been experiencing over the years[4]. Furthermore, given the crucial role that NIH-funded research plays in generating the findings on which so many new drugs are based, this boost is a much-appreciated development[5].  

However, providing increased funds to research and development is not always the best way to improve patients’ access to cures. This has led to concerns among members within the healthcare industry regarding the bill’s ability to truly improve patient outcomes. Although its stated purpose is to modernize medicine, it is mostly focused on streamlining the drug and medical device approval process. Over the past 80 years, the U.S. has worked on improving its regulatory approach criteria to better assess drugs’ and medical devices’ therapeutic efficacy and risk. The U.S. also has a comparatively fast rate of drug and medical device approval as compared to other developed countries[6]. For example, the FDA typically evaluates new drug and device applications within 6 to 10 months, but already more than two thirds of drugs and medical devices are approved on the basis of studies lasting 6 months or less[7]. This approval process for drugs and devices appears to have an already impressively quick turnaround for such complex assessments. This short approval window already poses a potential risk for patients taking medications that are designed for treatments lasting longer than 6 months, yet the Act calls for even faster approval[8].

To speed up the approval process the Act undermines a fundamental principle of medical and scientific experimentation: the importance of randomized clinical trials (RCTs). RCTs are considered the “gold standard” for clinical trials because its design allows researchers to properly test the efficacy of different types of medical interventions. These types of clinical trials provide researchers with a “risk-benefit profile” on certain medications or devices, which are used: 1) to prevent pharmaceutical and medical device companies from selling products that are potentially life-threatening and help protect their reputation, and 2) to protect patients from unwanted harm or risk[9].  However, to speed up the process for deciding which drugs or medical devices to approve the bill encourages the FDA to consider “nontraditional study designs.” This would mean that the “regulatory-approval criteria” could be based off of “evidence from clinical experience, which includes observational studies, registries, and therapeutic use” instead of other methods of data analysis that are often generated through RCTs[10]. The new law would also have a similar effect on how high-risk medical devices can be approved. Instead of having medical device companies submit an application to the FDA, it allows these manufacturers to pay third-party organizations to determine whether these device makers can be relied on to assess the safety and effectiveness of its own products. As a result, these companies have complete authorization to continue to assess its own products on an ongoing basis (a clear conflict of interest)[11]. The pharmaceutical and device industries might benefit in the short-term by the more rapid approval of experimental medicines and medical devices. However, the downstream consequences of putting patients at risk from side-effects that went unnoticed from shoddy clinical trials and poor approval-reviews may outweigh any immediate benefits. Furthermore, these changes in the approval process could lead to overall less salutary outcomes for patients and the U.S. healthcare system.

The 21st Century Cures Act offers some good ideas on how to improve medical innovation and drug development so that more patients and families can get the medicines and cures they desperately need. However, this should not come at the cost of undoing important safety and regulatory measures. Doctors understand that there exist many different cures to remedy a single illness, and often try different treatments before settling on one. Congress could benefit from thinking like a physician so that they can find the best course of treatment to cure the U.S.’s current ills when it comes to drug development and approval before accepting the 21st Century Cures Act as a cure-all.

  [1] LaMattina, John.  “Is the 21st Century Cures Act Good or Bad for the Biopharmaceutical Industry?” Forbes Magazine.  June 8, 2015.  http://www.forbes.com/sites/johnlamattina/2015/06/08/is-the-21st-century-cures-act-good-or-bad-for-the-biopharmaceutical-industry/

  [2] Goldstein, Beth and Pari Mody.  “21st Century Cures Act on House Floor; CMS Proposes Modification to Two Midnight Rule, Publishes End-Stage Renal Disease PPS Proposed Rule; Supreme Court Upholds ACA Subsidies.”  The National Law Review.  July 6, 2015.  http://www.natlawreview.com/article/21st-century-cures-act-house-floor-cms-proposes-modification-to-two-midnight-rule-pu

  [3] ibid

  [4] Avorn, Jerry and Aaron S. Kesselheim.  “The 21st Century Cures Act – Will It Take Us Back in Time?” The New England Journal of Medicine.  June 25, 2015.  http://www.nejm.org/doi/full/10.1056/NEJMp1506964

  [5] Kesselheim, Aaron, Yongtian Tina Tan, and Jerry Avron.  “The Roles of Academia, Rare Diseases, and Repurposing In The Development of the Most Transformative Drugs.” Health Affairs.  February 2015.  http://content.healthaffairs.org/content/34/2/286

  [vi] Kane, Jason.  “Health Costs: How the U.S. Compares with Other Countries.”  PBS News Hour.  October 22, 2012. http://www.pbs.org/newshour/rundown/health-costs-how-the-us-compares-with-other-countries/

  [7] Avorn, Jerry and Aaron S. Kesselheim.  “The 21st Century Cures Act – Will It Take Us Back in Time?” The New England Journal of Medicine.  June 25, 2015.  http://www.nejm.org/doi/full/10.1056/NEJMp1506964

  [8] ibid

  [9] ibid

  [10] Schank, Adam and Jim Meyers.  “What You Need to Know About the ’21st Century Cures’ Bill.”  Bloomberg Government.  June 8, 2015.  https://www.bgov.com/news_item/xgm5qfsDeve5ZPYyNu8tyQ

  [11] ibid

Student Blog Disclaimer
  • The views expressed on the Student Blog are the author’s opinions and don’t necessarily represent the Penn Wharton Public Policy Initiative’s strategies, recommendations, or opinions.

PENN WHARTON PPI
RESOURCE SPOTLIGHT:

  • <h3>The World Bank Data (U.S.)</h3><p><img width="130" height="118" alt="" src="/live/image/gid/4/width/130/height/118/484_world-bank-logo.rev.1407788945.jpg" class="lw_image lw_image484 lw_align_left" srcset="/live/image/scale/2x/gid/4/width/130/height/118/484_world-bank-logo.rev.1407788945.jpg 2x, /live/image/scale/3x/gid/4/width/130/height/118/484_world-bank-logo.rev.1407788945.jpg 3x" data-max-w="1406" data-max-h="1275"/>The <strong>World Bank</strong> provides World Development Indicators, Surveys, and data on Finances and Climate Change.</p><p> Quick link: <a href="http://data.worldbank.org/country/united-states" target="_blank">http://data.worldbank.org/country/united-states</a></p><p>See all <a href="/data-resources/">data and resources</a> »</p>
  • <h3>Federal Reserve Economic Data (FRED®)</h3><p><strong><img width="180" height="79" alt="" src="/live/image/gid/4/width/180/height/79/481_fred-logo.rev.1407788243.jpg" class="lw_image lw_image481 lw_align_right" data-max-w="222" data-max-h="97"/>An online database consisting of more than 72,000 economic data time series from 54 national, international, public, and private sources.</strong> FRED®, created and maintained by Research Department at the Federal Reserve Bank of St. Louis, goes far beyond simply providing data: It combines data with a powerful mix of tools that help the user understand, interact with, display, and disseminate the data.</p><p> Quick link to data page: <a href="http://research.stlouisfed.org/fred2/tags/series" target="_blank">http://research.stlouisfed.org/fred2/tags/series</a></p><p>See all <a href="/data-resources/">data and resources</a> »</p>
  • <h3>Congressional Budget Office</h3><p><img width="180" height="180" alt="" src="/live/image/gid/4/width/180/height/180/380_cbo-logo.rev.1406822035.jpg" class="lw_image lw_image380 lw_align_right" data-max-w="180" data-max-h="180"/>Since its founding in 1974, the Congressional Budget Office (CBO) has produced independent analyses of budgetary and economic issues to support the Congressional budget process.</p><p> The agency is strictly nonpartisan and conducts objective, impartial analysis, which is evident in each of the dozens of reports and hundreds of cost estimates that its economists and policy analysts produce each year. CBO does not make policy recommendations, and each report and cost estimate discloses the agency’s assumptions and methodologies. <strong>CBO provides budgetary and economic information in a variety of ways and at various points in the legislative process.</strong> Products include baseline budget projections and economic forecasts, analysis of the President’s budget, cost estimates, analysis of federal mandates, working papers, and more.</p><p> Quick link to Products page: <a href="http://www.cbo.gov/about/our-products" target="_blank">http://www.cbo.gov/about/our-products</a></p><p> Quick link to Topics: <a href="http://www.cbo.gov/topics" target="_blank">http://www.cbo.gov/topics</a></p><p>See all <a href="/data-resources/">data and resources</a> »</p>
  • <h3>HUD State of the Cities Data Systems</h3><p><strong><img width="200" height="200" alt="" src="/live/image/gid/4/width/200/height/200/482_hud_logo.rev.1407788472.jpg" class="lw_image lw_image482 lw_align_left" srcset="/live/image/scale/2x/gid/4/width/200/height/200/482_hud_logo.rev.1407788472.jpg 2x, /live/image/scale/3x/gid/4/width/200/height/200/482_hud_logo.rev.1407788472.jpg 3x" data-max-w="612" data-max-h="613"/>The SOCDS provides data for individual Metropolitan Areas, Central Cities, and Suburbs.</strong> It is a portal for non-national data made available through a number of outside institutions (e.g. Census, BLS, FBI and others).</p><p> Quick link: <a href="http://www.huduser.org/portal/datasets/socds.html" target="_blank">http://www.huduser.org/portal/datasets/socds.html</a></p><p>See all <a href="/data-resources/">data and resources</a> »</p>
  • <h3>NOAA National Climatic Data Center</h3><p><img width="200" height="198" alt="" src="/live/image/gid/4/width/200/height/198/483_noaa_logo.rev.1407788692.jpg" class="lw_image lw_image483 lw_align_left" srcset="/live/image/scale/2x/gid/4/width/200/height/198/483_noaa_logo.rev.1407788692.jpg 2x, /live/image/scale/3x/gid/4/width/200/height/198/483_noaa_logo.rev.1407788692.jpg 3x" data-max-w="954" data-max-h="945"/>NOAA’s National Climatic Data Center (NCDC) is responsible for preserving, monitoring, assessing, and providing public access to the Nation’s treasure of <strong>climate and historical weather data and information</strong>.</p><p> Quick link to home page: <a href="http://www.ncdc.noaa.gov/" target="_blank">http://www.ncdc.noaa.gov/</a></p><p> Quick link to NCDC’s climate and weather datasets, products, and various web pages and resources: <a href="http://www.ncdc.noaa.gov/data-access/quick-links" target="_blank">http://www.ncdc.noaa.gov/data-access/quick-links</a></p><p> Quick link to Text & Map Search: <a href="http://www.ncdc.noaa.gov/cdo-web/" target="_blank">http://www.ncdc.noaa.gov/cdo-web/</a></p><p>See all <a href="/data-resources/">data and resources</a> »</p>
  • <h3>Federal Aviation Administration: Accident & Incident Data</h3><p><img width="100" height="100" alt="" src="/live/image/gid/4/width/100/height/100/80_faa-logo.rev.1402681347.jpg" class="lw_image lw_image80 lw_align_left" srcset="/live/image/scale/2x/gid/4/width/100/height/100/80_faa-logo.rev.1402681347.jpg 2x, /live/image/scale/3x/gid/4/width/100/height/100/80_faa-logo.rev.1402681347.jpg 3x" data-max-w="550" data-max-h="550"/>The NTSB issues an accident report following each investigation. These reports are available online for reports issued since 1996, with older reports coming online soon. The reports listing is sortable by the event date, report date, city, and state.</p><p> Quick link: <a href="http://www.faa.gov/data_research/accident_incident/" target="_blank">http://www.faa.gov/data_research/accident_incident/</a></p><p>See all <a href="/data-resources/">data and resources</a> »</p>
  • <h3>Internal Revenue Service: Tax Statistics</h3><p><img width="155" height="200" alt="" src="/live/image/gid/4/width/155/height/200/486_irs_logo.rev.1407789424.jpg" class="lw_image lw_image486 lw_align_left" srcset="/live/image/scale/2x/gid/4/width/155/height/200/486_irs_logo.rev.1407789424.jpg 2x" data-max-w="463" data-max-h="596"/>Find statistics on business tax, individual tax, charitable and exempt organizations, IRS operations and budget, and income (SOI), as well as statistics by form, products, publications, papers, and other IRS data.</p><p> Quick link to <strong>Tax Statistics, where you will find a wide range of tables, articles, and data</strong> that describe and measure elements of the U.S. tax system: <a href="http://www.irs.gov/uac/Tax-Stats-2" target="_blank">http://www.irs.gov/uac/Tax-Stats-2</a></p><p>See all <a href="/data-resources/">data and resources</a> »</p>
  • <h3>National Center for Education Statistics</h3><p><strong><img width="400" height="80" alt="" src="/live/image/gid/4/width/400/height/80/479_nces.rev.1407787656.jpg" class="lw_image lw_image479 lw_align_right" data-max-w="400" data-max-h="80"/>The National Center for Education Statistics (NCES) is the primary federal entity for collecting and analyzing data related to education in the U.S. and other nations.</strong> NCES is located within the U.S. Department of Education and the Institute of Education Sciences. NCES has an extensive Statistical Standards Program that consults and advises on methodological and statistical aspects involved in the design, collection, and analysis of data collections in the Center. To learn more about the NCES, <a href="http://nces.ed.gov/about/" target="_blank">click here</a>.</p><p> Quick link to NCES Data Tools: <a href="http://nces.ed.gov/datatools/index.asp?DataToolSectionID=4" target="_blank">http://nces.ed.gov/datatools/index.asp?DataToolSectionID=4</a></p><p> Quick link to Quick Tables and Figures: <a href="http://nces.ed.gov/quicktables/" target="_blank">http://nces.ed.gov/quicktables/</a></p><p> Quick link to NCES Fast Facts (Note: The primary purpose of the Fast Facts website is to provide users with concise information on a range of educational issues, from early childhood to adult learning.): <a href="http://nces.ed.gov/fastfacts/" target="_blank">http://nces.ed.gov/fastfacts/#</a></p><p>See all <a href="/data-resources/">data and resources</a> »</p>
  • <h3>National Bureau of Economic Research (Public Use Data Archive)</h3><p><img width="180" height="43" alt="" src="/live/image/gid/4/width/180/height/43/478_nber.rev.1407530465.jpg" class="lw_image lw_image478 lw_align_right" data-max-w="329" data-max-h="79"/>Founded in 1920, the <strong>National Bureau of Economic Research</strong> is a private, nonprofit, nonpartisan research organization dedicated to promoting a greater understanding of how the economy works. The NBER is committed to undertaking and disseminating unbiased economic research among public policymakers, business professionals, and the academic community.</p><p> Quick Link to <strong>Public Use Data Archive</strong>: <a href="http://www.nber.org/data/" target="_blank">http://www.nber.org/data/</a></p><p>See all <a href="/data-resources/">data and resources</a> »</p>
  • <h3>MapStats</h3><p> A feature of FedStats, MapStats allows users to search for <strong>state, county, city, congressional district, or Federal judicial district data</strong> (demographic, economic, and geographic).</p><p> Quick link: <a href="http://www.fedstats.gov/mapstats/" target="_blank">http://www.fedstats.gov/mapstats/</a></p><p>See all <a href="/data-resources/">data and resources</a> »</p>
  • <h3>USDA Nutrition Assistance Data</h3><p><img width="180" height="124" alt="" src="/live/image/gid/4/width/180/height/124/485_usda_logo.rev.1407789238.jpg" class="lw_image lw_image485 lw_align_right" srcset="/live/image/scale/2x/gid/4/width/180/height/124/485_usda_logo.rev.1407789238.jpg 2x, /live/image/scale/3x/gid/4/width/180/height/124/485_usda_logo.rev.1407789238.jpg 3x" data-max-w="1233" data-max-h="850"/>Data and research regarding the following <strong>USDA Nutrition Assistance</strong> programs are available through this site:</p><ul><li>Supplemental Nutrition Assistance Program (SNAP) </li><li>Food Distribution Programs </li><li>School Meals </li><li>Women, Infants and Children </li></ul><p> Quick link: <a href="http://www.fns.usda.gov/data-and-statistics" target="_blank">http://www.fns.usda.gov/data-and-statistics</a></p><p>See all <a href="/data-resources/">data and resources</a> »</p>
  • <h3>The Penn World Table</h3><p> The Penn World Table provides purchasing power parity and national income accounts converted to international prices for 189 countries/territories for some or all of the years 1950-2010.</p><p><a href="https://pwt.sas.upenn.edu/php_site/pwt71/pwt71_form.php" target="_blank">Quick link.</a> </p><p>See all <a href="/data-resources/">data and resources</a> »</p>