Resources and expertise that help solve the unique challenges of rare disease research

Supporting Your Rare Disease Project

Real World Evidence

We are the industry leaders in developing and running Real World Research to support regulatory requirements, generate commercialization evidence, and engage treatment populations. Our in-depth understanding of patient populations means we are perfectly placed to meet the challenge of engaging with the very small pools of patients with a particularly rare disease.

Direct-to-Patient

Mapi provides Direct-to-Patient communications with a dedicated global team and call center, enabling patient enrollment, retention, and support throughout the study. Lengthy trials are handled consistently, and evidence can be collected thoroughly with no margin for error. Often, due to the lengthy nature of a rare disease clinical trial, a change in personnel can result and cause adverse effects. Our global team goes through a rigorous training process and provides an unrivaled consistency of communication.

Patient-Centered Outcomes

Our world-renowned Patient-Reported Outcomes leadership has access to the world’s largest exclusive COA library, as well as the most experienced custom scale development team in the industry. We also provide methodological and operational expertise in Mixed Methods Research as well as top-level endpoint strategy consulting. Our team can rapidly develop questionnaires and delve into the unmet need of a rare disease.

Language Services

Mapi is the recognized leader in the linguistic validation of COAs. We’re proven experts in supporting patient and health care provider materials, developing questionnaires in over 170 languages. As authors of the Linguistic Validation Manual for Health Outcomes Assessments, we understand the challenges of linguistic validation and will embed our expertise into the project from its inception.

Strategic Regulatory Services

We provide strategic regulatory consultation and access to experienced regulatory professionals for regulatory meetings. We’ve worked with regulatory agencies in over 60 countries and we pride ourselves on offering deep collaboration and support throughout the entire regulatory process, from rare disease classification to approval and reimbursement.

Pharmacovigilance

Mapi provides strategically placed Qualified PV contacts and International Pharmacovigilance and Safety experts. Located in Europe and North America, our specialists have experience working across a variety of regulatory landscapes and are supported by our late phase and epidemiology teams. This working structure means pharmacovigilance activities can be strengthened to meet the increasing demands of regulatory agencies.

Health Economics & Outcomes Research

Our global Health Economics team specializes in rare disease research, including Meta-Analysis and Cost Consequent Analysis to support payer, HTA, and Patient Advocacy value message development. The economics of Rare Disease research are complex and fiercely debated, but our Health Economics team has a strong knowledge of the required value messaging that will be heard by regulators and payers.

Patient and Health Care Provider Insights

Mapi has deep relationships with Patient Advocacy and Patient Communities. Sourcing and retaining patients for rare disease trials can be time-consuming and costly. Our relationships with key patient advocacy groups and communities give us a solid grounding to build reliable and long-lasting relationships with patients, while procuring valuable patient insights to rare diseases.

Pharmacovigilance & Risk Management