Rare Focused
PUBLICATIONS
RAM is a network of community-based activists and vetted community-focused advocates that have made a public vow to always place the best interests of the naturally diverse rare disease patient community first. Assumptions and biases have historically led to misinformed insights, which are extremely damaging to the people of the rare disease community.
RAM's network is dedicated to correcting misinformation, biased "insights" and to providing real world guidance to community-focused stakeholders interested in acquiring a comprehensive understanding of a disease-specific landscape or the general rare disease ecosystem.
The following is a comprehensive list of reports, white papers, guidances and more from various agencies located throughout the rare disease ecosystem that RAM's network has found to be helpful and informative.
Reports, White Papers & More from sources not affiliated with the Rare Advocacy Movement.
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Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database - Orphanet *Note: This analysis is of rare genetic diseases found in populations that racially identify predominantly as white and is therefore conservative as it does not include the naturally diverse population, rare cancers, nor rare diseases caused by rare bacterial or viral infectious diseases or poisonings.
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Patient partner compensation in research and health care: the patient perspective on why and how - Patient Experience Journal *Note: This publication details the argument as to why community-based stakeholders should be compensated for their contributions within research and healthcare in great detail, most of which is agreeable to the professionals of the Rare Advocacy Movement. The compensation rates, however, are outdated. For up-to-date fair market value for community-based insights please Click Here.
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Caregiving in the US 2015 Report - National Alliance for Caregiving *Note: This Report does not differentiate between the "caregiver" role and the "care partner" role. Caregivers serve a parental guardian role to the recipient. Care partners do not serve as parental guardians, but rather as committed life partners to the recipient. It is important to recognize and acknowledge the difference between the two types of relationships.
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Defining patient centricity with patients for patients and caregivers: a collaborative endeavour - an Open Access article attempting to develop a consistent definition of patient centricity and its associated principles in order to provide the biopharmaceutical industry with a clearer understanding as to how to adopt and use appropriate reference points for consistent patient engagement throughout the product life cycle. *Note: This article focuses more on establishing that patient-centric relationships must be developed rather than establishing how to establish appropriate patient-centric partnerships. This article attempts to place patient-centric programs into a neat box with predetermined algorithms misleading the biopharmaceutical industry into thinking that patient-centric relationships don't require the implementation of a value system in exchange for community-based insights. The Rare Advocacy Movement's Community-based Stakeholder (CBS) Collaboration model is a real-world model that proves its value-based system works and simultaneously corrects misinterpretations and misinformation regarding patient-centric protocols, campaigns and programs from the patient and carer perspectives.
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FDA PFDD Guidance for Enhancing the Incorporation of the Patient's Voice in Medical Product Development and Regulatory Decision Making - U.S. FDA has made available a site that contains information and documents related to FDA's development of the methodological PFDD guidances, including public workshops, draft guidances, and hypothetical scenarios, which are all intended to serve as a basis for dialogue.
