Policy and patient advocacy: Lessons from fireside chats

Several leading politicians in the healthcare sector gathered earlier this year for a fireside chat to discuss key issues affecting 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, VP for policy and government affairs at the National Council on Health and Christine Schnemann, senior director at the Milken FasterCures Institute.

During the hour, the panel discussed a range of topics, including the implications of the Inflationary Reduction Act (IRA), the growing development of real-world records (RVE) policies, the complexities surrounding equity in care and the future of data privacy legislation.

Future impacts of the IRA

The commission spent time analyzing the next steps for the IRA and grappling with the implications of this far-reaching legislation. Several panelists acknowledged its impact on research and development (R&D) departments and that, with limited budgets, the pace of drug discovery could slow for years to come. A University of Chicago study suggests that could lead to 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 may be affected. The implications of this scenario could mean that patients would no longer be able to access treatments and, in the absence of new drugs, could face higher long-term medical costs.

Further implications include the opportunity for pharmaceutical sponsors of clinical trials to re-prioritize and refocus their research and development efforts and investments, which has a direct impact on patients and their treatment options. With fewer drugs coming to market, manufacturers and even the FDA need to hear patients’ perspectives. We need to ask critical questions such as ensuring that the drugs that come to market will actually help patients.

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

The panelists suggested that when companies partner with patient advocacy organizations to educate patients, they in turn ensure that their voice remains central to the implementation of the new law. Companies must also improve their processes for engaging the patient community in the post-market environment, particularly in the context of implementing IRAs.

The value of RVE in healthcare

The digital era has transformed healthcare. One of the key changes is the rise of RVE, which goes beyond traditional clinical trials to capture how patients experience treatments and outcomes in everyday settings. Increasing amounts of real-world data (RVD) from sources such as electronic health records (EHR) and patient-reported outcomes can support structured clinical trial data to inform product research and development decisions, with RVE providing more data on safety and effectiveness of the drug. in real situations.

However, the increasing use of RVE is not without its challenges. The vast variety of RVD available means that its interpretation requires meticulousness and expertise. Recognizing this, the FDA proactively published a series of draft guidance for the use of RVD in regulatory activities.

As the panel discussed, patient organizations play a key role in this changing landscape. Their deep connections to patient communities offer a way to directly impact patients through education. As the primary source of RVD, patients must understand the implications of RVE. They need to know how their personal health data and treatment results contribute to research and influence health care choices. By demystifying RVE, patient organizations can ensure that patients are actively contributing to the healthcare ecosystem, rather than just passively providing data. Initiatives led by patient organizations collect RVE on behalf of their communities and there are opportunities to engage materially in the process. For example, Medicare can be used to look at patient groups as sources for RVE and RVD that can then be shared to influence regulatory decisions for more effective treatments. For example, their influence is evident in Medicare’s drug price negotiation process, as it now reviews data on patients’ experiences with therapeutic alternatives.

Patient organizations also help drive regulatory decisions through RVE generation. This can be seen in the recent approval of a drug to treat Friedreich’s ataxia. The approval is based largely on the RVD on the medical history collected by the manufacturer in partnership with the Friedreich Ataxia Research Alliance. That data has been shared with numerous academic researchers and companies to aid in product research and development and is an excellent illustration of the potential of patient-driven RVE to accelerate approvals and improve care.

However, the integration of RVE into healthcare is still at an early stage. While there are success stories, the industry needs a more unified approach. This will ensure that RVE generation and interpretation are in line with regulatory expectations. The work of The Real-World Evidence Alliance, showcased during the fireside chat, is an excellent example of efforts to unite the industry around RVE. The Alliance is a coalition of RVD and analytics organizations dedicated to leveraging RVE to better inform regulatory decisions and, ultimately, benefit patients.

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

How clinical trial reforms affect patients

The panelists also confirmed the connection between diverse representation and clinical trials. IKVIA is taking the lead in driving legislative proposals to support diversity in clinical trials. Patient organizations are also playing a key role in this changing landscape with their boots on the ground. Their challenge is often in educating patients about clinical trials and the options available.

As the panelists noted, patient data and experiences contribute to robust RVD, which is invaluable for research and advocacy. While the potential for collaboration between trial sponsors and patient groups is enormous, the approach needs to be improved. In this context, the FDA’s guidance on trial diversity is a model. He emphasizes the need for comprehensive, deliberate strategies, from site selection to researcher recruitment. Urges sponsors to ensure trials accurately represent target communities.

Decentralized (DCT) and hybrid clinical trials (HCT) offer the potential to increase diversity and inclusion, driven by technological advances. However, careful design and execution are needed to ensure data security and privacy and to widen participation without introducing disparities. Patient organizations play a key role in promoting and supporting DCT and HCT 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 patient advocacy and research.

Patient organizations can partner with sponsors and other stakeholders in the development and implementation of DCT and HCT. 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 policy on patient experience

Patient organizations play a vital role in today’s evolving healthcare ecosystem. They serve as a bridge between real-world patient experiences, industry care environments and regulatory agencies, supported by the unique value they bring to the policy debate in terms of real-life experience. The strength of patient organizations stems from the deep trust they have built and the relationships they have with patients and those affected by the diseases, disorders or conditions they represent. This position, coupled with their subject matter expertise, allows them to gain invaluable insights that can shape health care policy and practice. Policy makers must understand that their work directly affects patient outcomes. The point here is that data, not just anecdotal or qualitative, but quantitative, can add weight to support legislative debates and initiate transformative changes in the health care system.

Patient data can guide clinical trial policy in several ways. For example, it can be used to identify patient groups that are underrepresented in clinical trials. This information can then be used to develop policies and programs that encourage and support the participation of these groups, as well as to inform 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 conduct clinical trials, such as monitoring safety and efficacy.

As agencies like the FDA adapt to the changing dynamics, they will continue to understand and recognize the impact and value of RVD for the patient population. Collecting data, supporting it, managing it, and ensuring that it is shared with the right players (both on the academic front and on the industry front) can lead to tangible, meaningful progress in creating RVE. This will not only improve drug development but will ultimately improve patient outcomes; a goal that everyone in the healthcare ecosystem is already striving for.

About participating organizations:

FasterCures, a center of the Milken Institute, works to build a system that is efficient, effective and driven by a clear vision: patient needs above all else. The Center believes that transformative and life-saving science should be fully realized and provide better treatments to people who need them.

Focus areas include accelerating innovation, building nonprofit capacity, improving the research and development environment, and strengthening global health.

Created by and for patient organizations more than 100 years ago, the National Health Council (NHC) brings together diverse organizations to build consensus and drive patient-centered health policy.

The Council promotes greater access to affordable, high-value, sustainable, equitable health care. Comprised of more than 160 national health organizations and businesses, the NHC’s core membership includes the nation’s leading patient organizations. Other members include health-related associations and nonprofit organizations, including the provider, research, and family care communities, as well as companies representing biopharmaceutical, device, diagnostic, generic drug, and payer organizations.

The Real-World Evidence Alliance is a coalition of RVD and analytics organizations with a shared interest in harnessing the power of RVE to inform regulatory decisions to improve patients’ lives. Its members have deep knowledge and experience working with health data across disease areas and patient populations.

The Real-World Evidence Alliance envisions a future in which data from EHRs, administrative claims and billing data, product and disease registries, personal devices, wearables, and health apps will be used to gather evidence to support regulatory decision-making regarding safety of medical products and effectiveness.

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