The 21st Century Cures Act
Implications for Research and Drug Development

Summary

THE 21st CENTURY CURES ACT: Implications for Research and Drug Development
Presented jointly with STAT

Monday, February 27, 2017

The passage of the 21st Century Cures Act has drawn both applause and criticism. A sweeping bipartisan effort with multiple components, the law dramatically boosts funding for medical research, particularly in areas such as cancer and brain disease. The law also relaxes regulatory processes for pharmaceuticals and medical devices. In doing so, the law’s supporters point to the potential for faster treatments benefiting from a streamlined approval process. Critics raise concerns that safety and efficacy might be compromised, with potentially devastating consequences. And the law also has been questioned for failing to explicitly address high drug prices, a growing public issue. These debates are unfolding as the Trump administration is expected to imminently announce its choice for a new FDA commissioner, who will head an agency directly impacted by the Cures act. (Update since this event: President Trump has nominated Scott Gottlieb for FDA commissioner). In this Forum, experts will explore the implications of the law for biomedicine, regulation, pharmaceuticals and patient advocacy.

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Presented jointly with STAT

Image Credit: iStock: 4X-image

  • Stephen Albers

    Since prescription drug anomalies are a major contributor to medical treatment deaths, how will this new law effect future treatment death?

  • The Forum Moderator

    Thank you for joining us today. Our webcast will begin at 12:30pm ET.

  • alfonso e sierra

    Part of the problem is the entities and individuals que receiving under and over the table and influencing these,, keep the prices high and non-reasonable and defeat any action to start solving the problem. Who are they?

  • alfonso e sierra

    Pubmed reports 18 – 22 articles on Alzheimer’s Disease daily, What are the real advances in treatment or even management in the last years?

  • The Forum Moderator

    Our live webcast will begin is a few minutes.

  • The Forum Moderator

    We are about to begin. Thank you for joining us today.

  • The Forum Moderator

    We are presenting this event jointly with STAT.

  • The Forum Moderator

    Information about the Cures act is here: https://www.congress.gov/bill/114th-congress/house-bill/6/text

  • The Forum Moderator

    Speaker Tenaerts is from the Clinical Trials Transformation Initiative, which was co-founded by FDA: https://www.ctti-clinicaltrials.org/

  • The Forum Moderator

    We will have a Q&A towards the end of this discussion. Post your questions for the panelists here or email them to theforum@hsph.harvard.edu.

  • The Forum Moderator

    This video is courtesy of FasterCures.

  • The Forum Moderator

    More information about the Cancer Moonshot is here: https://www.cancer.gov/research/key-initiatives/moonshot-cancer-initiative

  • David J Wierz

    Will a transcript of the presentation be available?

    • The Forum Moderator

      Yes– a transcript will be posted on this site next week.

  • The Forum Moderator

    Dr. Brawley joins us from the American Cancer Society: https://www.cancer.org/

  • The Forum Moderator

    A video of this event will be posted on-demand here later this week. The video also will be posted on YouTube and Facebook.

  • The Forum Moderator

    Have a question for the panelists? You can post it here or email to theforum@hsph.harvard.edu.

  • The Forum Moderator

    Thanks for joining us. Watch for the on-demand video here to be posted later this week. Or, look on YouTube.

  • Kristen Dweck

    QUESTION SENT BY EMAIL

    I’m very connected to the rare disease community and there’s a lot of disappointment in the Act because the OPEN clause was dropped, which would have incentivized the drug companies to invest in treatments for orphan diseases that involved costly trials. Are there any other provisions in the Act that might compensate for this, or help those with rare disorders that are not cancer related?

  • Kristen Dweck

    QUESTION SENT BY EMAIL

    How might a new FDA administration interpret and implement this Act, given Trump’s executive order directing federal agencies to cut two regulations for every new one adopted – one in two out? And how will this be managed if there is to be no increase in budget, according to that Executive Order, for implementing new regulations added or cut? How is the FDA actually going to make this work?

  • Kristen Dweck

    QUESTION SENT BY EMAIL

    The Cures Act mandates increased inclusion of underrepresented populations in clinical trials. What are some areas of research that really need more clinical trial patient diversity and could benefit from this measure?


  • Kristen Dweck

    QUESTION SENT BY EMAIL

    I seems like the Cures Act is just a pharma give away that loosens regulations on the drug companies but does nothing to bring down drug prices. I would argue that it benefits pharma at the expense of other public health initiatives. What are your opinions on that and where do controls on drug pricing fit in?

  • Kristen Dweck

    QUESTION SENT BY EMAIL

    I’m interested in what you were saying about how prevention programs fit into all of this, particularly as related to cancer. Why wasn’t more money in the Cures Act put in that direction, especially given the rise of chronic diseases like cancer and diabetes?

  • Kristen Dweck

    QUESTION SENT BY EMAIL

    Under the Cures Act, a promising Alzheimer’s drug which recently failed in phase three clinical trials might have been approved a few years ago based on early clinical trials that were very favorable, early safety studies that were very favorable, and some trends toward impressive efficacy. This is just one example. Many scientists are not convinced the new measures are the best route to drug approvals. What safety net can be put in place to make sure all drugs that go to market are safe?


  • Kristen Dweck

    QUESTION SENT BY EMAIL

    It would be good to hear more about the advantages and shortcomings of being able to submit “data summaries” and “real world evidence” as compared to clinical trials in the FDA drug approval process. Clearly there are risks associated with these measures, but they also are going to speed up the process for drugs that have may have no reason to be held back. How do we actually measure the risks versus the benefits here?


  • Kristen Dweck

    QUESTION SENT BY EMAIL

    Under the Cures Act’s ‘Next Generation of Researchers Initiative’, new researchers will have more opportunities to coordinate, develop, modify, and prioritize policies and programs, and benefit from loan repayment programs. Looking back to the early days of your career, how game-changing could this have been for you?


  • Kristen Dweck

    QUESTION SENT BY EMAIL

    It’s very troubling that the research appropriations to the NIH are subject over the next decade to annual review by Congress. Seems to me that opens up whole slew of other issues. Would you say that this NIH funding pipeline isn’t really secure, given that reality? And if that’s the case, the real winners here are the pharma and medical testing industries, not the research community.

  • Kristen Dweck

    QUESTION SENT BY EMAIL

    Where is the place for evidence-based natural medicine in this Act?

  • Kristen Dweck

    QUESTION SENT BY EMAIL

    At least $1 billion of non-NIH money will be available for grants to states to supplement opioid abuse prevention and treatment activities. What are your recommendations for states to make the best use of this money, what programs should be prioritized, and could this mean the end of this epidemic?


  • Kristen Dweck

    QUESTION SENT BY EMAIL

    Critiques of the Cures Act have mentioned that despite its wide net, the legislation does little to alleviate the rising cost of drugs. Where exactly are the price controls? Does lowering drug prices require the 21st Century Cures Act, or would simply focusing on getting the FDA to approve generics for sale do the trick? 


  • Kristen Dweck

    QUESTION SENT BY EMAIL

    Trump has said he wants to drive the prices of drugs down. If he were to authorize the FDA to hire more people and get the generic drug backlog down a year or two, would that be enough to create a huge drop in drug prices?


  • Kristen Dweck

    QUESTION SENT BY EMAIL

    Is there any evidence to be skeptical that the $5 billion in annual funding for for genetics and brain research initiatives is leading hospitals and universities to lobby in favor of this bill, even if some are skeptical of unsafe FDA approvals? Was it wise, in your opinion, to include both provisions in the same piece of legislation?


  • Kristen Dweck

    QUESTION SENT BY EMAIL

    It appears that many other elements of the Cures Act are focused on reducing administrative burdens for researchers and NIH paperwork — how much will that impact the speed and process of research and clinical trials?


  • Kristen Dweck

    QUESTION SENT BY EMAIL

    Could one of you speak more to the “High-Risk, High- Reward” clause and share what doors that might open regarding new ground-breaking research?


  • Kristen Dweck

    QUESTION SENT BY EMAIL

    The Cures Act attempts to set up mechanisms for encouraging collaboration and data sharing between researchers and healthcare professionals. What steps have already been taken to increase the accessibility of patient data to researchers for therapeutic development, or what actions are soon to be taken?