In January 2025, the International Council for Harmonisation (ICH) formally adopted the updated E6(R3) Good Clinical Practice (GCP) guidelines. Annex 1 and the revised 12 principles are already in effect. Annex 2 arrives in July. This is not just regulatory housekeeping. It is a clear message to the research world: Meaningful patient involvement is no longer aspirational. It is expected.
For decades, clinical research positioned patients as participants, not partners. They were expected to show up, sign forms, follow protocols, and stay quiet. Their insights were rarely sought, and their lived experience was often dismissed as anecdotal or irrelevant.
That model no longer holds.
The ICH E6(R3) guidelines now state: “Engaging patients in the design and conduct of clinical trials can help ensure that a trial is relevant and not unduly burdensome to participants.” (Section 3.1.3)
This is not a polite suggestion. It is a fundamental shift. Patient input is now a quality factor. Ignoring it puts the relevance, rigor, and compliance of a study at risk.
As the founder of a rare disease charity and someone actively involved in clinical research design, I have seen the cost of failing to involve patients. Studies often struggle with recruitment and retention, or miss outcomes that truly matter. This is not just inefficient. It is short-sighted.
Patient collaborators help research teams ask better questions, select outcomes that reflect lived priorities, and identify design flaws that would make participation difficult. In rare disease research, this becomes even more important. In areas like multiple birth complications, where data are limited and guidance is still evolving, patient organizations can provide insights that would otherwise be missed.
The updated GCP framework reflects years of global consultation. The 2020 Public Engagement Summary shows that stakeholders asked for more flexible, inclusive research practices and better integration of patient perspectives. The final guideline delivers on that request. But now, the responsibility shifts back to researchers and sponsors to put those expectations into practice.
There is also a regulatory reality to address. By July 2025, studies are expected to be fully compliant with E6(R3), including demonstrating how patient input informed the development process. That means the rationale for design choices must now include lived experience, not just operational convenience.
Engagement is no longer a communications strategy. It is part of study quality.
So how does patient involvement improve research?
When done well, it starts early. Patient organizations can:
- Refine inclusion criteria to ensure broader relevance.
- Flag unnecessary burden in visit schedules or assessments.
- Simplify consent materials so they are truly understood.
- Identify outcomes that matter beyond traditional clinical endpoints.
This leads to stronger trials, better data, and more ethical study conduct. It also builds trust between communities and researchers.
If you are unsure where to start, you are not alone. Many researchers are still learning how to meaningfully collaborate with patients and advocacy groups. Fortunately, many patient organizations are already embedded in research spaces through forums, support groups, and contributions to peer-reviewed literature. They are not hard to find if you look.
In 2024, I co-authored a study published in Midwifery examining how people with experience of high-risk monochorionic twin pregnancies used social media during their care. Over 600 participants shared how these platforms influenced decision-making, provider communication, and treatment plans. More than half reported sharing information from online support groups with their health care team, and 70 percent said their providers considered it. These findings support what the ICH guidelines now reflect. Informed, engaged patients are shaping research and care in real time.
Clinical research is evolving. E6(R3) codifies what many of us have long advocated. Research is stronger, safer, and more relevant when it is done with patients, not just for them. As the July deadline approaches, research teams have a decision to make. This is the moment to review current protocols, assess where patient input is missing, and begin building relationships that will define the next generation of good clinical practice.
Because involving patients is not just the right thing to do. It is now the standard.
Stephanie Ernst is a patient advocate.