Top Rare Disease Expertise at QualityMetric | We Measure Health.

Rare Disease Expertise

Understanding how rare diseases and new treatments affect patients

QualityMetric is an expert in rare disease evidence generation. We take an end-to-end approach to build novel instruments and support new label claims for therapies.
Contact us for a review of our capabilities by phone at (800) 572-9394 or by filling out the form below. For Academic organizations click here.

QUALITYMETRIC HAS CREATED MORE THAN 25 PROPRIETARY
INSTRUMENTS AND SUPPORTED 50 LABEL CLAIMS

The Challenges in Rare Disease

Often there is scant evidence and low understanding of rare disease populations. Disease onset, presentation and the patient experience are not well understood. The natural history of the disease and treatment journey with associated short- and long-term outcomes may not be well documented.

Our end-to-end approach to
PRO instrument development / determining fit
of existing PRO Instruments

Our end-to-end approach to
PRO instrument development / determining fit
of existing PRO Instruments

QualityMetric takes an end-to-end approach to helping clients put together a robust evidence generation plan with patient reported outcomes and clinical outcomes assessments that illuminate how diseases impact patients and support new label indications that reflect therapeutic value to patients’ lives. QualityMetric’s approaches include validation of the SF‑36v2, SF‑12v2 and other instruments in a new rare disease, validation of non-QM instruments and design and validation of new PRO instruments including submission of FDA and EMA dossiers to provide evidence of fit for purpose in pursuit of PRO label claims.

An Expert in Rare Disease

QualityMetric has projects in the following rare diseases:

Bespoke Rare Disease Services

QualityMetric also offers bespoke research services at any phase of research.

TARGETED LITERATURE REVIEWS, SYSTEMATIC LITERATURE REVIEWS
& META-ANALYSES

Provide information related to a rare disease (symptoms, impacts, etc.) and measures used in clinical studies of those diseases.

QUALITATIVE INTERVIEWS

Work alongside patient advocacy groups and clinical professionals to conduct qualitative research studies with clinicians, patients and caregivers.

COA MEASUREMENT STRATEGY

Recommend and develop COAs that are most appropriate for your research purpose.

STUDY DESIGN & INTERPRETATION

Burden of Disease: Compare functional health status against conditions with a significant impact on quality of life to contextualize patient scores.

Treatment Efficacy: Assist your team in determining study endpoints, trend analyses, categories of change and clinical markers to establish benefits of different treatments.

PSYCHOMETRIC VALIDATION

Evaluate psychometric properties of COAs in a particular disease.

REGULATORY SUPPORT

Ensure your COA tools adhere to global regulatory standards, including those of the FDA, EMA and other regulatory agencies worldwide.

PATIENT ADVOCACY

We partner with patient advocacy groups to assist in patient recruitment and sustained engagement.

Rare Disease Infographic

Learn how to generate evidence for new therapies with COA measures.

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