Global biotechnology company ensures excellence in life sciences compliance benchmarking

Client background

Amicus Therapeutics is a global biotechnology company focused on delivering treatments for rare and ultra-rare diseases. With two commercial products in the rare disease space, this company maintains a strong global presence and lean operational structure as well as a strong culture of patient-centricity.

The business challenge

Baker Tilly was engaged to assess Amicus Therapeutics' compliance program, ensuring alignment with industry standards, governmental guidance and best practices. Instead of conducting a traditional audit, the focus was on assessing risk and performing life sciences compliance benchmarking against peer organizations and the Office of Inspector General (OIG) seven elements of an effective compliance program, as well as leveraging Baker Tilly’s best practices for compliance program effectiveness.

Key areas of focus included evolution of foundational compliance controls, risk management governance and awareness across the organization, auditing and monitoring, training effectiveness and the usability of policies and procedures. The assessment identified strengths and opportunities for enhancement, reinforcing leadership’s confidence in the program’s overall positioning.

Strategy and solution

Baker Tilly conducted a comprehensive assessment using the OIG’s seven elements framework. The evaluation involved:

• A full day on-site working session to review the compliance program structure and clarify key areas, which was further enforced by additional interviews with leaders across the organization and a comprehensive written overview of the company’s compliance program.
• Analyzing documentation, including enterprise risk management process, third party governance process, audit methodologies, training records and policies, to assess usability and coverage.
• Benchmarking the compliance program against industry standards and peer organizations to provide a comparative performance analysis with a specific focus on Baker Tilly’s experience in working with other rare disease companies.

The findings were compiled into a structured benchmarking report, evaluating each compliance element on a scale from “Initial” to “Optimized.” A scorecard highlighted strengths and areas for enhancement, while an executive summary report supported board-level discussions.

Given both Amicus Therapeutics’ strong program foundation (including cross-functional alignment on risk) and ongoing robust execution of controls, the assessment reinforced existing processes rather than identifying deficiencies. The benchmarking analysis provided leadership with insights to validate their approach and demonstrate the compliance function’s value. With high ratings across all seven elements, the company gained a clear perspective on its industry standing and a road map for future compliance initiatives.