Transparency

Patient Organisation Support

Patients and the advocacy organisations 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 energises everything we do. Such is the foundation and premise for our interactions with patient advocacy organisations.

Annually, Amicus publishes a list of its financial support of European Patient Organisations. 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 Organisation (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.


Collaborative 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.

Collaborative 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 Collaborative working as one or more pharmaceutical companies working with other organisations to deliver initiatives which either enhance patient care or are for the benefit of patients or alternatively benefit the national healthcare system and, as a minimum, maintain patient care. Collaborative working initiatives must demonstrate a pooling of skills, experience and/or resources from all parties involved. Joint working is a form of collaborative working and is defined as one or more pharmaceutical companies working with other organisations to deliver initiatives through a pooling of resources which directly benefit the patient.

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

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.

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)