RARE Toolkits

Below is a list of all of our current RARE Toolkits.

center of excellence?
center of excellence?

A Rose by Any Other Name

This toolkit uses the term “center of excellence,” but it should be noted that the term does not have a clear definition. Many so-called centers of excellence do not use the term to describe themselves, and so it is used in this toolkit to broadly describe the concept.  In fact, none of the centers of excellence referred to in this toolkit use the term.

Some patient advocacy organizations, such as the Tuberous Sclerosis Alliance, provide designations through an application and review process to help patients understand the resources available at a clinic and provide standards to qualify for specific designations as a center for the treatment of a specific rare disease.

Centers of Excellence for Tuberous Sclerosis Complex
James Wheless, MD, of the University of Tennessee Health Science Center reminds patients with tuberous sclerosis complex (TSC) that revisiting a medical center focused on TSC can greatly improve a patient's quality of life due to the numerous advances that have been made in our understanding of this condition.

Despite the nebulous nature of the term, centers of excellence programs do share common elements. There is no minimum standard for a center of excellence, but below is a list of some common elements centers of excellence may include. A center of excellence may have some or all of these elements. Some rare disease patient advocacy organizations will look for certain elements they consider essential before considering a clinic a center of excellence.

Core Elements

The UK Plan for Rare Diseases, which recognizes the need for centers of excellence, outlined key characteristics that centers should have, according to Rare Diseases UK. These include:

• Coordinated care;
• Adequate caseload for expertise;
• Not dependent on a single clinician;
• Arrangement for transition from children’s to adults’ services;
• Engaged with people with rare conditions; and
• Research active.

The European Union Committee of Experts on Rare Diseases (EUCERD) elaborated in 2011 a set of recommendations on Quality Criteria for Centres of Expertise which helps define what is a centre of expertise for rare disease, their missions and scope. To learn more, go here.

Setting Standards

The Tuberous Sclerosis Alliance has created a set of standards for clinics wishing to be considered Tuberous Sclerosis Complex clinics. This includes standards for clinical care, education, partnerships, and research. A complete description can be found on the Tuberous Sclerosis Alliance website’s clinic guidelines, but some of the key points are highlighted here.

The organization’s guidelines call for these clinics to have board certified healthcare providers that provide multidisciplinary care as a team for children, adolescents and/or adults. The director of the clinic should be a medical doctor with medical board certification in their country of clinical practice in a specialty related to one or more aspect of tuberous sclerosis complex, such as cardiology, dermatology, nephrology, neurology, or other area.  While it requires a TSC Clinic to provide care in at least three core specialties, a TSC Clinic seeking to meet the organization’s higher Gold Standard must provide care in six or more specialties.

Beyond the medical care provided by these clinics, the organization expects clinics to play a critical role in educating patients, their families, educators, healthcare providers, and the public about the disease and to work closely with the patient advocacy group to push out information and promote awareness of the disease.

Clinics are also expected to form partnerships with their local TSC group. This would include engaging volunteers from the organization to provide resource information and support for individuals with TSC and their families, and TSC Clinic staff will refer the individuals and families to the community alliance.

Regarding research, the organization expects that one or more the clinic’s healthcare providers should either conduct investigator-initiated clinical trials in TSC, participate in a TS Alliance or industry sponsored TSC clinical trial, or conduct basic science research in TSC.