NORD recently posted the following question on its Facebook page: How long did it take you or a loved one to get an accurate diagnosis?
We were stunned at the response. Within the first few hours, we got 200 replies and 20% of those who responded had waited 10 years or longer to get an accurate diagnosis. read more >
The rare community has made huge strides in the 30 years that have elapsed since the Orphan Drug Act was passed. There are more new treatments and there is more investment in research and more awareness of rare diseases than ever before. But what about diagnosis? Are we getting better at diagnosing rare diseases? read more >
When given the opportunity to dispense certain products, services, privileges, or other benefits that can be spread around, many of us feel an impulse to do the most good possible for the most people possible. This seems only natural, maybe even right, until you consider the adverse effects this approach can impose on people who are not among “the most”—people with rare diseases, for example. Acting on this impulse in biomedical research and health care services, then, would result in allocating resources to diseases that affect much larger numbers of people in preference to rare diseases. read more >
September is Newborn Screening Awareness Month and this year marks 50 years since the beginning of this life-changing public health program. Wilson and Jungner developed what is considered to be the gold standard for selection of conditions suitable for screening, which includes the ability to detect the condition at an early stage and the availability of an effective treatment. However, the ethical principles that have guided newborn screening for the past 50 years have been increasingly challenged as new technologies have emerged to screen for more conditions. read more >
Government agencies, industry, and patient advocacy groups all agree that a patient-centered perspective is vital during the processes of drug development, clinical trials, benefit/risk assessment, and regulatory review. However, legal barriers and proprietary constraints complicate when patients can be brought to the table and integrate their input.
At the FDA’s annual Patient Network meeting, “Demystifying FDA: An Exploration in Drug Development”, there was a concerted effort among all stakeholders to increase collaboration. read more >
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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