RARE Toolkits

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


Undiagnosed and rare disease patients often face endless healthcare questions with few to no answers. Doctors frequently know little to nothing about their patients’ rare disease, and with little treatment options available (if any), patients are often the ones taking the lead in driving and managing their own diagnosis and treatment.

Proactive patients and family members reach out to pharmaceutical and biotechnology companies on many important issues, including:

• The status of research
• Gaining access to investigational or approved medications
• Help with patient and prescription assistance programs
• Financial assistance to offset the burden of disease
• Reward program services
• Disease education
• Product information
• The drug development process
• Fundraising, grant, and sponsorship opportunities

Evolution of the Patient Advocate
Jayne Gershkowitz, Vice President of Patient and Professional Advocacy and Public Policy at Amicus Therapeutics, spoke with Rare Disease Report on the evolution of patient advocates in the rare disease community.

Alison Rockett Frase, the Founder and Vice President of the Joshua Frase Foundation, says that as someone working to propel research for almost two decades, a patient advocate, and a mother of an affected child, she sees the importance of improving communication between rare disease patients and their families with biopharmaceutical companies.

“Increasing awareness and education for underrepresented populations will ensure a more open communication between all, and a more equal and balanced enrollment of the population in a clinical trial,” Alison Rockett Frase says. “This must be a team effort for all parties involved in order for success to be achieved on all levels.”

This toolkit breaks down the process of reaching out to the biotech and pharmaceutical industry effectively and the steps you can take to get answers.