EURORDIS Group call for more access to rare disease therapies in Europe

As a Patient with a Rare Disease Here’s What Rare Disease Day Means to Me

As a Patient with a Rare Disease Here’s What Rare Disease Day Means to Me

"We have already seen huge breakthroughs in the a year ago alone: early clinical trials in gene therapy have given hope to haemophilia patients, scientists have found a way to supress the build-up of harmful proteins in patients suffering from Huntington's disease and we have seen a CAR-T therapy launched to use a patient's own immune system to fight rare types of cancer", noted Thompson when speaking to Health awareness.

The FDA is also entering into a memorandum of understanding with the National Organization for Rare Disorders to conduct outreach with the FDA's new patient affairs staff on "ways to enhance the incorporation of patient experience into regulatory discussions".

We, rare disease patients, are not the problem: we are part of the solution. These conditions largely affect pediatric patients.

The FDA has instituted a number of programs to bring the patient perspective to the product review process, consistent with legislation enacted by Congress and advanced by NORD.

"These listening sessions will provide FDA review division staff with better insight into what is important to patients in managing their diseases and improving their quality of life".

"It is important for FDA to understand, from the patient perspective, disease burden, management of symptoms, daily impact on quality of life, and patients' risk tolerance". "Patients and caregivers bring a pragmatic, realistic perspective about what they are willing to deal with in terms of potential risks and benefits for new therapies". With this pilot patient engagement activity, the FDA and NORD will partner to identify patient community representatives and coordinating listening sessions where patients can share their experiences with the FDA review division staff. "FDA is committed to do what we can to stimulate the development of more products by improving the consistency and efficiency of our reviews, streamlining our processes and supporting rare disease research". "The need is most significant when it comes to rare diseases".

The FDA Commissioner Scott Gottlieb has also pledged to re-examine what represents an "appropriate application" of orphan incentives in this changing landscape. The Orphan Drug Act was enacted in 1983 as an effort to spark interest in rare disease drug development.

The Vatican on Wednesday expressed its solidarity with those suffering from rare diseases and urged public authorities to contribute to scientific and medical research, involving all available agencies and companies, and making available the knowledge, funding and best medical practices. We need to close the gap between innovation and access.

New draft legislation would give access to experimental drugs that have completed phase 1 studies for patients with terminal conditions. In observance of Rare Disease Day, the FDA reviews the progress it has made thus far and determines what more can be done in the future to facilitate accelerating drug approvals, according to the agency.

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