Integral Solutions for Successful Orphan Drug Commercialization
Support for the Complex Landscape of Rare Diseases
Mapi offers the support you need to navigate the research, regulatory, evidence, payer, and scientific challenges that drain your resources and stand in the way of your passion and success.
Our experience in rare disease research gives us unrivaled expertise in working within this complex landscape. Thanks to our unique resources and team of orphan drug commercialization specialists, we can help your project easily overcome the common barriers in orphan drug evaluation / assessment in a real world setting.
Unparalleled Contribution to the Scientific Community
Mapi supports rare disease programs from clinical development onwards, including the full breadth of payer, patient insights, advocacy engagement, and regulatory services. Our teams of experts are experienced in working within in the entire drug life-cycle. Our wide-ranging expertise allows us to conduct Mixed Methods Research (MMR) that combines quantitative and qualitative research methodologies and provides unique opportunities to document the patient experience of a new drug before market authorization.
Our knowledge on orphan status and rare disease prevalence is second-to-none, and having a clear understanding of the market is key to strategically planning for commercialization.
Mapi is also the world leader in localization and language services, enabling unprecedented evidence generation and value synthesis resources.
Finally, we benefit from the work of the Mapi Research Trust, a not-for-profit organization that facilitates access to information in the fields of Patient-Centered Outcomes. Through the non-profit Mapi Research Trust we have access to world-leading experts as well as Clinical Outcome Assessment (COA) developers and authors with unique insights in overcoming scientific barriers.
We are the only entity in the industry with a not-for-profit research trust of this type, and this positions Mapi as one of the top providers of patient-centered outcomes research.
Better Access to Patients than Any Other Company in the Industry
Patients are at the heart of our research, and with over four decades of real-world patient recruitment expertise, Mapi is the industry leader able to rapidly deploy internal expertise and decades of experience to overcome any patient engagement and retention challenge. Our integral Patient & Health Care Provider Insights combined with our unique Direct-To-Patient resources and technology partners to collect patient-reported data, with proactive patient follow-ups provides your study with accelerated recruitment, increased
patient engagement, improved patient-reported data quality, and minimized study burden and attrition.
Our approach is a perfect fit for rare disease retention programs, where patient populations are small, retention can be an issue, and study burdens may prove challenging. We maintain communication with caregivers and patients every step of the way with our integrated direct telephone contact, standard mail, email, and SMS. We become a trusted partner to patient populations and directly support patients and caregivers in overcoming many logistical compliance, data collection, and program adherence challenges.
Holistic Solutions in a Dynamic and Uncertain Study Environment
Clinical trial data that can demonstrate efficacy and safety of a molecule as early as possible is one of the ultimate product development goals.
Mapi’s integrated solutions give us a unique vantage point on study challenges and allows us to be dynamic and agile throughout the entire project. For example, Mapi partners with orphan drug developers to design the endpoint strategy and ensure the patient’s voice will be considered in value communication based on the best scientific standards. When adequate measures are not available for specific populations, Mapi is the industry leader with the expertise to concurrently develop Clinical Outcome Assessments in multiple languages. Accelerating your program start-up and patient recruitment. Mapi is also the industry leader in the field of Mixed Methods Research, an ideal approach to address the challenges of drug development in rare diseases.
And our unparalleled pharmacovigilance and risk management team works alongside our real world evidence researchers, and their collective expertise is integrated into each phase of the study rather than into service silos. This means we can map out a sound study design that is executed properly right from the very beginning.
Navigating You through the Regulatory Maze
The Strategic Regulatory Group at Mapi works with companies of all sizes to manage the regulatory aspects of rare disease projects around the world, assisting with orphan drug designation followed by the application for marketing approval. We’ve worked with regulatory agencies in over 60 countries, allowing us to offer a diversity of regulatory experience that’s unparalleled in the industry. Our seasoned regulatory group has assisted over 20 companies with gaining orphan drug designation and market commercialization.
Our team of experts pride themselves on offering the deep collaboration and cooperation that only a truly global team can offer, working closely with regulators, clients, and other Mapi units to achieve success. With Mapi’s Strategic Regulatory Services, we don’t just provide comprehensive regulatory expertise…we deliver cohesive teamwork that gets results.
Priority Review Voucher Strategy
Mapi can help you access the resources needed for valuable benefits such as priority review vouchers from regulatory agencies. These vouchers are transferable and able to be either monetized to continue previous research or leveraged to expand your own portfolio and compete across broader markets. Mapi helps you gain access to these programs, consult on your strategy, and even support your decision through negotiation support to assure you gain the greatest value from these opportunities.
The regulatory landscape for reimbursement, much like the guidelines for classification, is varied and continually changing. Mapi experts can help you navigate the regulator / payer labyrinth and facilitate scientific advice meetings.
When assessing global reimbursement challenges posed by orphan drugs, we know it’s not always just cost-effectiveness taken into consideration. Factors such as the seriousness of the health condition, the availability of other therapies, and the cost to the patient if the drug is not listed for reimbursement are also important. Finally, our experience in rare disease research, coupled with our close relationships with global regulatory agencies, allows us to offer an unparalleled range of orphan drug regulatory support.