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

Pharmacovigilance & Risk Management

The safety regulatory environment has seen significant changes over the past few years, with an increasing emphasis on proactive management of risk throughout the lifecycle of a product. Regulatory agencies are looking for continuously updated evidence that the risk-benefit balance is positive for the target population. This needs to be considered in many contexts: multiple risks being associated with each product, each varying in terms of severity; the effect on individual patients; and impact on public health. Understanding these risks will change and evolve with increasing use of a product, with potential identification of new risks and additional characterization of known risks.

Managing pharmacovigilance and risk minimization activities for products intended for rare disease populations presents unique challenges when interpreting pharmacovigilance legislation designed with a different patient population in mind. Mapi offers in-depth knowledge that can assist with:

  • Negotiating with agencies regarding required pharmacovigilance and risk minimization activities considering the challenges of small, niche patient populations
  • Preparing risk management strategies and maintaining risk management plans
  • Implementation of pharmacovigilance and risk minimization activities, including post-authorization safety studies, measuring effectiveness of risk minimization activities, preparation of educational materials, managing controlled distribution programs, targeted follow-up of adverse events of special interest, and more
  • Ensuring routine pharmacovigilance activities are adapted to take into account the needs of a rare disease product
  • Providing routine pharmacovigilance services

Mapi’s pharmacovigilance function is closely aligned with Mapi’s expertise in managing rare disease programs, allowing a seamless transition from risk management program design through to implementation.

Health Economics, Outcomes Research, & Market Access