Medicine

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

March 01, 2018 17:46·
Omics sweet omics – curing the incurable one disease at a time

Omics sweet omics – curing the incurable one disease at a time

The objective is to raise awareness on rare diseases and improve access to treatment and medical representation for individuals with rare diseases and their families.

The report outlines the groups target to have 3 to 5 times more rare disease therapies and medicines approved and made cheaper each year.

Rare diseases, of which there are between 6,000 and 7,000, will affect fewer than one in every 2,000 people. Events were held across the world as a fundraiser for awareness and impacted patients.

It also seeks to encourage researchers and decision makers to address the needs of those living with rare diseases. For Rare Disease Day, the FDA also announced multiple new actions to demonstrate its commitment to developing rare disease drugs and devices.

"When it comes to access, we refuse to take no for an answer".

According to global management consulting firm, McKinsey & Company to successfully launch a drug for the treatment of a rare disease companies must excel in four specific areas: The must commit to the rare disease community; identify the largest possible patient group; ensure patient access; and provide sustained support to the patients and caregivers. Growing awareness is crucial because many rare diseases do not have treatment options and if they do, the drugs are costly.

There have already been major advances in gene therapy, which hit the headlines past year. The approvals of two CAR-T cell therapies from Novartis and Kite Pharma by the US Food and Drug Administration (FDA), for example.

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"This was more than double the number of requests received in 2012". According to the National Institutes of Health (NIH), there are almost 7,000 rare diseases affecting almost 30 million Americans.

Approximately 1 in 10 Americans has a rare disease, including cancers or genetic metabolic conditions.

Many people living with a rare disease do not have access to the medicine that they need - and more often than not, a medicine does not exist to treat their illness.

Although the disease may be rare, patients and families share a common struggle.

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". One such example is that of Chapper healthcare, which is raising money through a Just Giving page for Moorfield's Eye Charity in support of Rare Disease Day and to help fund vital research needed to diagnose rare eye conditions.

Despite this, there is no cure for the majority of rare diseases and many go undiagnosed.

The agency is also planning a public meeting to help it "prepare for the changing landscape of orphan drug development posed by the growth in targeted therapies and molecularly defined diseases".

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