People with rare diseases wait five to six years and see seven to eight doctors before an accurate diagnosis. Their clock starts when the disease takes hold — not when they receive a diagnosis or when a treatment is approved.

In AL amyloidosis, a rare, deadly disease in which the immune system turns against patients, the median delay to diagnosis is about two years. By then, the heart and kidney may already be damaged beyond repair. The emotional toll is harder to measure, but no less real.

Patients may spend weeks between appointments, unsure what to ask or whether the next visit will help. Regulation cannot ease their fear or untangle complex diagnostic pathways. But lawmakers and regulators can recognize that time matters in rare, rapidly progressing diseases. Every month spent navigating an unclear pathway or waiting for the next step can carry real consequences for patients whose disease is already advancing.

For decades, single-arm trials — those without a randomized control group — were a standard route to approval for rare, rapidly progressing diseases. When no FDA-approved alternative exists, assigning patients to a placebo or an inferior treatment may be impractical or unethical.

That approach was questioned last year after comments attributed to the FDA’s Center for Biologics Evaluation and Research raised doubts about whether single-arm data would still meet the bar for rare-disease approvals. FDA leadership has since backtracked, saying the comments do not reflect official policy. Developers still lack clarity as they design trials.

Rare-disease treatments may need different routes to the same evidentiary standard. With small patient populations and no clear approved comparator, traditional randomized trials can be difficult to design, enroll, and complete.

The goal is not to lower standards. A single-arm trial is no shortcut around rigorous science; in many cases, it offers the most ethical way to generate meaningful evidence. Clearer pathways should account for small patient populations and rapidly progressing disease. Patients and developers need to know what evidence is required and how long it may take to produce.

Drug development exists to serve patients, so their experiences and needs belong in regulatory discussions. Trial delays, enrollment challenges, or prolonged reviews can mean months or years of uncertainty for people whose diseases are progressing. They may not live to see a lifesaving solution reach the market.

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Patient stories cannot replace clinical evidence, but they can help regulators understand its consequences and context. For families, quality of life, hospitalization, and mortality affect whether someone can work, care for children, remain independent, or spend meaningful time with family. Lawmakers and regulators should recognize and weigh these outcomes.

New FDA leadership should put rare-disease patients at the center of its next chapter while upholding rigorous safety and efficacy standards. It can do so by supporting trial designs and regulatory pathways suited to small patient populations and rapidly progressing diseases, and by creating more ways for patients, physicians, and researchers to inform decisions. Like everyone, rare-disease families deserve hope and confidence that the FDA can protect and support them through life’s most difficult decisions.

David Gusick received his BA from Cornell University and his MA from NYU’s Interactive Telecommunications Program. His goal is to remove barriers — including cost, scheduling, and stigma — to professional emotional support and make free, high-quality support accessible to every rare-disease family, regardless of location, education, income, or access to medical professionals.