Patient Organization Support

Patients and the advocacy organizations that represent them are at the heart of everything we do. It is the mothers, fathers, sons, and daughters – the real people who live with rare disease – who are the focus and beneficiaries of our work. To do that great and meaningful work we need to be great listeners and learners. We need to understand the true burdens of the individuals, families, and caregivers living with the rare diseases we seek to alleviate, and to grasp the medical needs of those rare disease communities. That understanding in turn informs and energizes everything we do. Such is the foundation and premise for our interactions with patient advocacy organizations.

Annually, Amicus publishes a list of its financial support of European Patient Organizations. Although we are not a member of EFPIA, we follow the standards for such reports set out in the EFPIA code, which is implemented at the national level through the country-specific codes of EFPIA’s member associations.

Healthcare Practitioner (HCP) & Healthcare Organization (HCO) Disclosures

Amicus has a singular mission. To make medicines that improve the lives of patients living with rare disease. In the pursuit of this mission, we collaborate closely with HCPs and HCOs. Such interactions occur, for example, when we conduct clinical trials, gain critical medical insights regarding how our medicines are used and how we can improve them, and when we share up-to-date information about our research and products at scientific congresses. In all such interactions, we are committed to providing science-based, truthful, and non-misleading information about our products, and to paying no more than fair market value for needed and legitimate services.

A growing number of countries around the world are adopting laws that require companies to make public the level of funding they provide to HCPs and HCOs, such as for research and consulting. Amicus is committed to complying with such transparency laws and with the transparency codes of the pharmaceutical industry associations of which it is a member.

Joint working with the NHS

At Amicus, we believe in building long-term value for our stakeholders – that includes patients with rare diseases and the NHS.

With patients at the heart of everything we do, we share a common agenda with the NHS to improve patient outcomes through high quality and cost- effective treatment and management. The common agenda to improve patient outcomes can be achieved through working together to ensure that patients get optimal care, providing support to help them maximise the benefits of treatment.

Joint working provides the formal foundation and framework for creating, developing and implementing innovative healthcare solutions, which deliver better health outcomes for patients and the NHS. The Department of Health defines Joint Working as “situations where, for the benefit of patients, one or more pharmaceutical companies and the NHS pool skills, experience and/or resources for the joint development and implementation of patient-centred projects and share a commitment to successful delivery”.

Through these projects, we are able to work with the NHS to pool skills, experience and resources to benefit patients with rare diseases.

Executive summary: United Kingdom – Royal Free NHS Foundation trust joint working project with Amicus Therapeutics (pdf)