The year 2025 has been a year of jarring paradoxes in policy engagement that specifically affected the Rare Diseases space.
We saw the passage of the One Big Beautiful Bill Act (OBBBA) in the US and the simultaneous contraction of global health funding. On the other hand we also witnessed the Sunshine Genetics Act in Florida that piloted a state funded genetic screening for all newborns.
These are just a couple of examples. But the trend is clear - we are heading into 2026 where rare disease patients are scientifically eligible for a cure, but are structurally blocked from receiving it due to systemic changes or funding issues.
What does this mean for you as a patient engagement professional?
In a landscape where patients and caregivers are facing new barriers to access, trial participation, and HTA involvement, your role requires you to show up informed and with empathy - so you can help them navigate unfamiliar and increasingly complex territory.
That’s where we can help. We did a deep dive into the legislative and regulatory events of 2025. Here are the five definitive policy trends shaping our landscape and the strategic engagement playbook you need to navigate them.
The most seismic shift of 2025 occurred on July 4th, with the signing of the One Big Beautiful Bill Act (OBBBA). While the headlines focused on tax cuts, the fine print fundamentally restructured the US healthcare safety net in ways that disproportionately impacted the rare disease community.
Key Highlights:
In this new environment, a patient group’s ability to host a fundraising gala matters less than its ability to keep its community insured. If the patient loses coverage due to administrative churn, your innovative therapy stays on the shelf. So your funding and support could potentially shift towards “defending access”.
Your engagement strategy would be about protecting the baseline of care.
In May 2025, the World Health Assembly adopted the resolution "Rare diseases: a global health priority for equity and inclusion" mandating a 10-year Global Action Plan.
This document, co-sponsored by Egypt, Spain, and Brazil, instructs the WHO to develop a 10-year Global Action Plan. It moves rare diseases from a "niche charity" issue to a "UN compliance" issue, urging member states to build national plans and strengthen patient registries.
It was a massive diplomatic victory. This win, however, collided with a harsh fiscal reality: the dissolution of USAID and a ~$9 billion cut to US global health funding in 2025.
The paradox creates a fiscal vacuum. The infrastructure often used for rare disease diagnostics in low-and-middle-income countries (LMICs), such as HIV and TB labs, is now at risk of collapse.
With US funding retreating, Patient Groups in other regions are vulnerable. You could help them frame their national plan requests not as charity, but as a roadmap for their government to fulfill its binding international commitment to the WHO.
The primary strategy would be to help Patient Groups practically leverage the WHO resolution. Here are some examples:
This is also where your knowledge of the Patient Groups’ capability is important. If your Patient Group has ample experience with lobbying, then capacity building would not be your focus.
Instead, you would check in to see what kind of assistance they would need.
For decades, newborn screening (NBS) advocacy in the US has been a federal game, centered on adding conditions to the Recommended Uniform Screening Panel (RUSP). It is a slow, deliberative process.
In 2025, Florida broke the mold. The Sunshine Genetics Act (HB 907) bypassed the federal bottleneck by establishing a state-funded pilot program to provide Whole Genome Sequencing (WGS) to 10,000 newborns . This shifts the paradigm from "screening for what we can treat today" to "screening for everything to enable research for tomorrow."
The Act also established the Sunshine Genetics Consortium. This move calls for universities and hospitals to analyze the data, positioning Florida as a hub for genomic innovation. We are seeing early signs of this trend replicating, with Virginia and other states observing closely.
Decentralizing advocacy - You no longer need to focus all your advocacy efforts in Washington D.C or Brussels. You could equip patient groups at the national level to fight and win in state capitols and regional health authorities.
You could help position the patient groups as co-architects or co-builders of the future diagnostic system (and not be mere observers)
In 2025 a critical struggle unfolded in Europe regarding the revision of the EU's pharmaceutical legislation, specifically concerning the role of patients in the European Medicines Agency (EMA).
The proposal sought to reduce patients from "members" to "observers," arguing that scientific rigor required purely clinical input. This triggered a massive backlash from the patient community to protect their status as decision-makers.
The patient community, led by EURORDIS and the European Patients’ Forum launched the #KeepPatientsVoting campaign. They proved that patients bring unique, evidence-based insights to risk-benefit analysis that clinicians simply cannot .
While the final outcome of the campaign is a compromise, the message was clear: patient engagement is under scrutiny. Patients can no longer assume their seat at the table is guaranteed; but must continually prove its value. As a patient engagement professional, you can make that a reality.
You could invest in “technical literacy” or technical capacity building.
This will make them undeniable partners in HTA regulations, and importantly, build trust between your company and the patient group.
If there is a "feel good" story of 2025, it is the Mikaela Naylon Give Kids a Chance Act. Passed by the US House in December 2025, this legislation forces a paradigm shift in how pediatric cancer drugs are developed.
It authorizes the FDA to direct companies to study combinations of cancer drugs in pediatric trials (rather than just single agents) and reauthorizes the Rare Pediatric Disease Priority Review Voucher (PRV) program through 2029.
The PRV program is arguably the most successful incentive for rare pediatric drug development, having spurred dozens of approvals by making rare disease research financially viable for biotech investors.
The engagement becomes more education-centered. You will be asking families to participate in more complex, multi-agent clinical trials. You must be able to explain the rationale of combination therapies and help design trial protocols that minimize the burden on families already managing intensive care regimens.
Patient engagement is no longer a “nice-to-have”. There’s increasing evidence to show that patient input is highly valuable. At the same time, patient groups are no longer content with a transactional relationship with the industry.
They want to partner with a company that has shared values, trust in their vision and make sure patient perspectives are used to co-create and co-build solutions.
They expect patient engagement professionals to show up knowing their challenges, and what their objective is. Looking at how policy changes influence patient groups is just the start.
You would also need to monitor the patient group activity, look at the major events across the industry to see how you could be a more supportive partner to the patient groups you engage with.
What’s your patient engagement strategy for 2026 like? Drop a note and let us know!