As new therapies continue to revolutionize the treatment of many diseases, it is positive news that policymakers in Washington are discussing ways to ensure patient access to new treatments and potential cures. As we close out 2019, PhRMA recently submitted comments to the Call to Action on Cures 2.0 being discussed in the House of Representatives by Congresswoman Diana DeGette and Congressman Fred Upton. The comments address opportunities to advance the use of digital health tools and real-world evidence (RWE) in promoting adherence and improving health outcomes as well as increasing efficiencies in the R&D process, promoting interoperability and evolving coverage and payment policies for breakthrough therapies, including potentially curative therapies.
In case you missed it, here are a few key excerpts:
- Enhancing the use of digital health technologies to improve adherence and patient outcomes, as well as to expedite drug development and access: “While progress has been made in adopting digital health technologies, more can be done to harness the full power of these platforms. Congress should direct HHS to identify potential coverage and access barriers related to the adoption and use of digital health tools and to identify potential changes in coverage and payment policies to facilitate their use. Similarly, Congress should urge the FDA to prioritize evolving the regulatory framework to allow biopharmaceutical companies to fully leverage digital R&D to improve drug development and speed access to those products.”
- Improving interoperability: “Biopharmaceutical manufacturers are committed to contributing to the meaningful advancement of health care data flow…improved interoperability can play a key role in fostering coordination of care and improving patient adherence to treatment regimens. A more interoperable system could result in increased efficiencies and advance patient engagement. Congress should encourage HHS to work with the biopharmaceutical industry, academia and other relevant stakeholders to support interoperability to facilitate the sharing of information that can lead to improved patient outcomes.”
- Modernizing the FDA’s data/information technology (IT) infrastructure: “Modernizing and enhancing the FDA’s on-going technological advances will be needed to help FDA scale its data/IT infrastructure…Congress should encourage FDA to work with the biopharmaceutical industry and other stakeholders to develop and finalize a formal and holistic modernization framework that includes provisions on how data requirements will be deployed and managed throughout the regulatory lifecycle. Such work could build upon FDA’s recently introduced Technology Modernization Action Plan.”
- Advancing the use of RWE to support regulatory decisions: “To fulfill the promise of the 21st Century Cures Act, Congress should encourage FDA to provide additional guidance on how studies using RWE—in the appropriate regulatory context-of-use—can be “adequate and well-controlled” and be sufficient to meet the existing “substantial evidence” standard, including addressing the need to consider RWE in the context of the totality of evidence.”
- Advancing coverage and payment policy reforms to support emerging technologies: “PhRMA appreciates that the initial vision for Cures 2.0 aims to recognize not just the incredible promise of emerging biopharmaceutical advances to save and improve the lives of patients, but the importance of payment policy reforms to unleash this innovation and ensure that new therapies benefit patients as quickly as possible. Because cell and gene and other breakthrough therapies hold great potential to transform care, payment policies should reflect the potential added value of these therapies to the health care system and to patients…. Specifically, Congress should encourage the [CMS] to build on the steps it has taken to date to recognize the value of these [emerging] through further payment reform. CMS, along with the HHS Office of Inspector General (OIG), should also be directed to revisit outdated regulations and guidance developed for a fee-for-service world. In particular, the federal Anti-Kickback Statute and government price reporting rules should be examined to determine how they are limiting the number, scale and types of these arrangements, specifically as related to value-based contracts for medicines.”
As we head into 2020, PhRMA looks forward to working with all stakeholders to support policies that benefit patient access to new treatments and potential cures.
To read the full comments, click here.
Anne McDonald Pritchett, PhD Anne McDonald Pritchett, Ph.D., is Senior Vice President, Policy and Research, at the Pharmaceutical Research and Manufacturers of America (PhRMA). Her primary focus is overseeing the development of legislative and policy analysis and research studies on a range of issues impacting innovative biopharmaceutical companies including intellectual property issues, FDA policy issues, and other issue areas impacting the environment for innovation. In addition to her public policy work, she and her team lead the development of a range of educational and other materials focused on explaining the R&D process, the value of innovation, and the role and contributions of the innovative biopharmaceutical industry.