Several of the health sector’s leading policy voices came together earlier this year for a fireside chat where they discussed key issues impacting the patient experience. The chat was moderated by two IQVIA leaders – Alexandra Weiss Roeser, Director of Nonprofit Strategy and Patient Advocacy, and Andrew Barnhill, Head of Public Policy – and featured industry experts, Wade Ackerman, Partner and head of Covington’s FDA practice group, Eric Gascho, SVP of policy and government affairs at the National Health Council, and Kristin Schneeman, Senior Director at the Milken Institute’s FasterCures.

Over the course of the hour, the panel discussed a range of topics including the implications of the Inflation Reduction Act (IRA), the growing developments in real-world evidence (RWE) policy, complexities surrounding equity in care, and the future of data privacy legislation.

Future impacts of the IRA

The panel spent time analyzing the next steps for the IRA and wrestled with the implications of this far-reaching legislation. Several panelists acknowledged its impact on research and development (R&D) departments and that, with constrained budgets, the pace of drug discoveries might slow in the years to come. A University of Chicago study suggests this could result in significantly fewer new drug approvals in the coming decades. Multiple orphan drugs, small molecule drugs, new indications for approved drugs, and cell and gene therapies could all be affected. The implications of this scenario could mean patients will no longer be able to access treatments, and, in the absence of new drugs, might incur higher long-term medical costs.

Further implications include the possibility of pharmaceutical sponsors of clinical trials reprioritizing and redirecting their R&D efforts and investments, which has a direct impact on patients and their treatment options. With fewer drugs coming to market, patient perspectives need to be heard by manufacturers and even, the FDA. We need to ask critical questions like ensuring that the drugs that are coming to market are actually going to help patients.

By educating communities about the IRA and its potential impacts, patient organizations can advocate for patient-centric drug development.

Panelists suggested that when companies partner with patient advocacy organizations to educate patients, they will in turn ensure their voices remain a central aspect in the implementation of the new act. Companies must also improve their processes for engaging patient communities in the post-market setting, particularly in the context of IRA implementation.

The value of RWE in healthcare

The digital era has transformed healthcare. One key change is the rise of RWE, which goes beyond traditional clinical trials to capture how patients experience treatments and outcomes in everyday settings. The growing amount of real-world data (RWD) from sources like electronic health records (EHR) and patient-reported outcomes can support structured clinical trial data to make research and product development decisions, with RWE providing more data around a drug's safety and effectiveness in real-world situations.

However, the growing use of RWE is not without challenges. The massive variety of RWD available means interpreting it takes meticulousness and expertise. Recognizing this, the FDA proactively released a series of draft guidance documents for the use of RWD in regulatory activity.

As the panel discussed, patient organizations play a key role in this changing landscape. Their deep ties to patient communities offer a way to directly impact patients through education. As the primary sources of RWD, patients must understand RWE's implications. They need to know how their personal health data and treatment outcomes contribute to research and influence healthcare choices. By demystifying RWE, patient organizations can ensure patients actively contribute to the healthcare ecosystem, rather than just passively providing data. Patient organization-led initiatives collect RWE on behalf of their communities, and there are opportunities for them to materially engage in the process. For example, Medicare can be utilized to look at patient groups as sources for RWE and RWD which can then be shared to influence regulatory decisions for more effective treatments. For instance, their influence is evident in Medicare's drug price negotiation process, as it now reviews data on patient experiences with therapeutic alternatives.

Patient organizations also help drive regulatory decisions through RWE generation. This can be seen in a recent drug approval for treatment of Friedreich’s Ataxia. The approval was based heavily on RWD about the disease’s history which the manufacturer collected in partner with the Friedreich's Ataxia Research Alliance. That data was shared with numerous academic researchers and companies to help drive research and product development and is a great illustration of the potential of patient-driven RWE to speed up approvals and enhance care.

However, integrating RWE into healthcare is still in the early stages. While there are success stories, the industry needs a more unified approach. This will ensure that RWE generation and interpretation align with regulatory expectations. The work of The Real-World Evidence Alliance, on display during the fireside chat, is a great example of efforts to unify the industry around RWE. The Alliance is a coalition of RWD and analytics organizations dedicated to harnessing RWE to better inform regulatory decision making and, ultimately, benefit patients.

Part of the Alliance’s focus has been supporting the advancement of the FDA’s RWE framework, increasing communication around the generation and use of RWE to better inform FDA decision making, and encouraging ways to use RWE to better understand the treatment effects of drugs and medical devices in underrepresented populations.

How clinical trial reforms affect patients

The panelists also acknowledged the relationship between diverse representation and clinical trials. IQVIA is taking the lead in guiding legislative proposals to support diversity in clinical trials. Patient organizations also play a crucial role in this changing landscape with their boots on the ground. Their challenge is often in educating patients about clinical trials and available opportunities.

As the panelists noted, patient data and experiences contribute to robust RWD, which is invaluable for research and advocacy. While collaboration potential between trial sponsors and patient groups is immense, the approach needs refining. In this context, the FDA’s trial diversity guidance is a model. It stresses the need for deliberate strategies across the board, from site selection to researcher recruitment. It calls for sponsors to ensure trials accurately represent the target communities.

Decentralized (DCTs) and hybrid clinical trials (HCTs) offer the potential to increase diversity and inclusion, driven by technological advances. However, careful design and execution are required to ensure data security and privacy and to broaden participation without introducing disparities. Patient organizations play a key role in promoting and supporting DCTs and HCTs by educating patients and the public about their benefits, which can help increase enrollment and participation. This can also raise awareness of the patient organization's mission and work, as well as amplify their voice in advocating for patients and research.

Patient organizations can partner with sponsors and other stakeholders to develop and implement DCTs and HCTs. These partnerships can help patient organizations build relationships with key stakeholders and provide opportunities to contribute their expertise and insights to the clinical research process.

Building policies on the patient experience

Patient organizations play a vital role in today’s evolving healthcare ecosystem. They serve as a bridge between patients’ real-world experiences, industry care settings, and regulatory agencies, underpinned by the unique value they bring to the policy debate in terms of real-life experience. The strength of patient organizations comes from the deep trust they have built and the relationships they hold with patients and those affected by the diseases, disorders, or conditions they represent. This position, paired with their subject matter expertise, enables them to garner invaluable insights that can shape healthcare policies and practices. Policymakers must understand their work directly impacts patient outcomes. It’s here that data, not merely anecdotal or qualitative, but quantitative, can add weight to support legislative discussions and drive transformative changes to the healthcare system.

Patient data can drive clinical trial policies in several ways. For example, it can be used to identify groups of patients who are underrepresented in clinical trials. This information can then be used to develop policies and programs that encourage and support participation from these groups as well as informing the design of future clinical trials, such as the selection of endpoints and the development of eligibility criteria. Patient data can also be used to guide how clinical trials are conducted, such as the monitoring of safety and efficacy.

As agencies like the FDA adapt to changing dynamics, they will continue to understand and acknowledge the impact and value RWD has for patient populations. Collecting data, supporting it, driving it, and making sure it's being shared with the right players (both on the academic front and on the industry front) can drive tangible, meaningful progress to generate RWE. This will not only improve drug development but, ultimately, improve patient outcomes; a goal that everyone in the healthcare ecosystem already strives for.