Commentary|Articles|September 4, 2026

Beyond Patient Centricity: What Rare Disease Research Gets Right

Patient-centricity remains largely consultative in most clinical research, yet rare disease communities demonstrate that genuine partnership from protocol inception improves feasibility, reduces unnecessary burden, and identifies outcomes that matter to patients themselves.

The clinical research industry has made enormous progress in becoming more patient-centric. Patients are increasingly consulted during protocol development, patient advisory boards are becoming more common, and regulators continue to encourage greater patient engagement throughout drug development.

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September 23rd, 2026

Clinical Site Readiness for Cell and Gene Therapies: Operational and Supply Chain Challenges as they Scale and as they Expand into the Community Care Setting

Cell and gene therapy access expands into community care settings only when operational coordination, site readiness, and supply chain standardization become first-order priorities equivalent to manufacturing capacity, requiring standardized processes, digital integration, and distributed logistics networks.

September 14th, 2026

Why Patient Input Comes in Too Late—and What Earlier Engagement Changes

In this video interview, Amélie Lothe, global medical community head for rare epilepsies at UCB Pharma, describes how shifting patient engagement from protocol review to endpoint selection transforms both the quality of trial data and the experience of participation.

September 11th, 2026

Why Seizure Counts Alone Can't Define Success in Epilepsy Trials

In this video interview, Amélie Lothe, global medical community head for rare epilepsies at UCB Pharma, explains why the full burden of developmental and epileptic encephalopathies extends far beyond clinical symptoms—and why endpoints must reflect what families are actually hoping for.

September 10th, 2026

What It Actually Means to Embed the Patient Voice in Trial Design

In this video interview, Amélie Lothe, global medical community head for rare epilepsies at UCB Pharma, explains why genuine patient partnership begins before the protocol is written—and what it looks like when patients and caregivers tell you a trial was designed with them in mind.

September 24th, 2026

When There’s No Registry: Building Rare Disease Evidence That Supports Regulatory Scrutiny

In rare disease drug development where no registry or natural history dataset exists, real-world evidence quality depends on treating evidence engineering as a design-stage decision, defining intended regulatory use and standardizing endpoints, harmonization, and data provenance upfront rather than reconciling gaps after collection.

September 24th, 2026

Trust, Technology, and the Patient-Site Relationship: Q&A with Andrea Valente, uMotif

In this Q&A, Andrea Valente, CEO of uMotif, discusses what it takes to build genuine trust between patients and sites, why making technology easier to use isn't the same as making patient-site interaction more effective, and where the industry is still falling short in its push toward patient-centered trial design.

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September 22nd, 2026

Is Decentralization Actually Reducing Site Burden, or Just Moving It?

Decentralized trial models have demonstrated real gains in enrollment performance and patient access, but operational switching costs, fragmented technology stacks, and unresolved gaps in the patient-site relationship raise a question the industry has been slow to confront directly.

September 22nd, 2026

DPHARM 2026: The Patient Journey Gap That Study Design Still Hasn't Solved

In a video interview prior to the 2026 DPHARM conference, Andrea Valente, CEO of uMotif, explains why truly understanding the patient journey from beginning to end—and building that understanding into both study design and data collection tools—remains one of the industry's most important unfinished tasks.