Skip to content
Patient advocacy is a driving force behind some of today’s most promising breakthroughs in biotechnology—where progress is increasingly powered by strong, productive collaboration between biopharma companies and patient advocacy leaders. Together, they are advancing science— and reshaping how innovation happens.
“Patient advocates play a fundamental role in biotech innovation—elevating the patient voice to help shape better research, smarter trial designs, and more meaningful patient outcomes,” said Karin Hoelzer, Senior Director of Patient Advocacy at BIO. “In many cases, patient advocates are helping the entire innovation ecosystem work most effectively —from drug discovery through market access and beyond.”
This evolving role of patient advocacy, and the success of new partnerships such as venture philanthropy, was the focus of a recent Biotechnology Innovation Organization (BIO) Patient Advocacy Coffee Chat. The discussion highlighted that such collaboration is not just aspirational: it is happening, and it is strengthening the entire innovation ecosystem.
Patient Advocates Leveraging Venture Capital strategies
One exciting development in the biotech space is the rise of venture philanthropy. Patient advocacy organizations are increasingly stepping forward as investors, partners, and strategic guides alongside biopharma companies to help derisk early-stage assets. By deploying patient-driven capital, coupled with deep disease expertise and patient-generated data, these organizations are helping potentially transformative therapies successfully cross the Valley of Death.
Venture Funds like the National Bleeding Disorders Foundation Venture Fund, Pathway to Cures, demonstrate the power of this approach. Venture philanthropy supplies the risk‑tolerant capital needed to advance early, preclinical science, ensuring potentially transformative innovations are not abandoned before they can be sufficiently derisked with the evidence required to attract more traditional investment.
Advocacy-led venture funds are identifying promising early-stage science, investing in emerging biotech companies, and working hand-in-hand with start-up companies to ensure development aligns with the greatest patient needs. These partnerships bring together complementary strengths.
“What we’ve learned is that a lot of biotechs, and even in some cases the Big Pharma, are not looking enough at what we call Lived Experience Experts (LEEs),” explained Tim Brent, MBA, Venture Principal at Pathway to Cures. “LEEs can be patients, but they are also parents and guardians of these affected individuals.”
As Brent explained it, oftentimes the input of LEEs are not being taken into consideration early enough in the R&D or clinical trial process, sometimes not at all.
“A company may have a great discovery and want to provide assistance with the disease, which is great, but if they don’t interact with LEEs, their clinical trial process can be very challenging,” he said. “The earlier the biotechs incorporate the patient perspectives, the more effectively the therapies can meet actual needs, and at the early stages, input from these folks can also shape the study design and product development.”
Venture Philanthropy as a model is proving to be both effective and scalable. Venture philanthropy is helping to de-risk drug development by aligning scientific innovation with lived experience from the outset. Companies benefit from better trial design, more relevant endpoints, and stronger patient engagement, while patients benefit from therapies that are more likely to succeed and deliver meaningful impact.
Key to this progress is data—often generated through close partnerships with patient advocacy organizations.
Data – An Investment Opportunity for Good
Will Greene, Board Member at the Foundation for Prader-Willi Research and lead author of a new World Economic Forum white paper, Making Rare Diseases Count: How Better Data Can Unlock a Multitrillion-Dollar Opportunity, emphasized the key role of data.
“Our policy recommendations ultimately center on data capture and analysis,” he explained. While the Forum also examined priorities such as increased public R&D funding, improved reimbursement pathways for innovative medicines, and stronger advocacy networks, high-quality data emerged as a common foundation across all of them.
“We realized that nearly every strategy for investment depends on having robust, reliable data. Without it, you can’t accurately assess needs, set priorities, or measure the impact of interventions,” explained Greene. “The paper calls for greater investment in rare disease broadly, but with data as a foundational pillar. The message to government and industry leaders is straightforward: if you’re deciding where to invest, start with high-quality data infrastructure.”
The paper showed that, among other findings, an increase in rare disease investments would:
- Lower out-of-pocket costs for patients and families,
- Significantly save costs for healthcare systems,
- Lower employer insurance premiums,
- Lessen the strain on social system,
- Open up a variety of new market opportunities, and
- Reduce R&D risk, among many, many more positive outcomes.
As the white paper demonstrates, patient-centered approaches are a powerful engine for better health outcomes, stronger economies, and faster scientific progress.
“The benefits extend far beyond the patients, families and communities most directly affected; they also accrue to healthcare systems, employers, payers, governments, industry and investors,” concluded Greene.
But what is “high quality data” in this context, and where is it coming from? The answer is disarmingly simple: in many cases, it comes from patients.
The Power of Patient Generate Data
At the Friedreich’s Ataxia Research Alliance (FARA), patient advocacy leaders worked closely with researchers and industry partners to help advance the first approved therapy for the condition. FARA played a key role in the first approval of a drug for Friedreich’s Ataxia by collecting the natural history study data from patients for years, while also helping to fund early-stage research and nurturing a diverse drug development pipeline.
“One of the drug approaches in our pipeline did lead to significant findings in preclinical models and labs,” said Myriam Rai, PhD, Director of Global Relations & Initiatives at FARA. “Thanks to a mother on the FARA board, who acted as a citizen scientist, we were connected to a small company in Texas who had similar molecules. It took a little while to convince them to launch the first clinical trial.”
The trial met the primary endpoint but the company struggles were far from over. Close collaboration between industry and patient advocates —anchored in patient-generated data—again came to the forefront.
“Unfortunately, the FDA raised concerns about the results at first,” Rai explained. “But the FA community believed the trial results were persuasive.” After extensive discussions, FDA eventually agreed that the natural history data FARA had meticulously collected through the years could be used, leading to the drug’s approval on Rare Disease Day February 28, three years ago.
Greene, who is the father of a son with Prader-Willi Syndrome, shared a similar experience from his community. “Over roughly a decade, our community built a registry with more than 2,000 participants and 60,000 completed surveys,” Greene explained. “That dataset delivered tremendous value for our community.”
The registry data, he explained, not only helped parents like him better understand the natural history of the condition; it also informed clinical guidelines and clarified which interventions are most effective.
“Crucially, it reached a level of rigor that regulators could rely on to evaluate therapies,” he added. “Data was one of the highest-leverage investments we made.”
Similarly, on March 30th, 2026, FDA’s Rare Disease Innovation Hub (RDIH) hosted its third RISE workshop on ‘Data Sharing across the Rare Disease Ecosystem,’ bringing together multi‑sector stakeholders to identify strategies, infrastructure needs, and collaborative models to make data sharing a consistent and reliable component of rare disease drug development. The themes from this workshop also emphasized the power of patient data, illustrating how patient‑contributed information can transform research, regulatory decision‑making, and therapeutic progress through close partnerships.
The Power of Data to Inform Good Policy
Patient-generated data is not only accelerating research—it is also exposing critical gaps in the healthcare system and opening opportunities for data-informed policy change.
“We just did a survey of 200 plus patients with chronic and rare diseases to better understand their experiences,” Sarah Jones, MPA, MS, Community Engagement at the Eosinophilic & Rare Disease Cooperative explained. “We were concerned about 340B abuses and what was happening with the 340B program at Disproportionate Share Hospitals (DSH). Their hospital systems are charging patients astronomical costs for their medications and in-hospital treatments, all while knowing that they’re getting these at deeply discounted rates without passing any of the savings on to their patients. And similarly, we’ve been absolutely concerned as we looked downstream and see what happened with these foreign price controls, and taking into consideration what happened the last time they were proposed. People with rare disease rely on a large number of medications, not just rare disease treatments, to manage their complex issues.” The data ERDC collected provide unique insights into the impact of these policies on chronic disease patients and how they experience access to the care they need.
Patrick Wildman, Senior Vice President, Advocacy & Government Relations, Lupus Foundation of America echoed this sentiment, noting “the MAPRx coalition has produced a couple of reports looking at how access barriers are undermining the affordability provisions in the Inflation Reduction Act (IRA) with the out of pocket cap, because we are seeing significant barriers that have been erected as a result of the IRA, whether it’s increasing cost shifting from co-pays to coinsurance, limited plan choice, a lot of things that we’re seeing challenges on the access side.” Having the data to truly understand the impact on patients and to guide policy solutions is key.
From early discovery to market access and policy, one message is clear: patient advocacy – including advocacy-led funds and data collection efforts – represents a powerful driving force that helps turn the hope of biotech innovation into treatments and cures for patients.
