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Karen Telleen-Lawton: A ‘Rare’ Day to Show Your Stripes

What do zebras and Feb. 29 have in common? Zebras are rare among equines for their black and white stripe pattern. Feb. 29ths are not strictly rare, but certainly noteworthy: They come along only every 1,460 days. These two phenomena combine on the last day of February to commemorate Rare Disease Day.

You can show your support for people with rare diseases and rare disease research by “showing your stripes” and by posting to social media.

A rare disease is defined in the United States as one that afflicts fewer than 200,000 Americans at any time, according to the National Organization for Rare Disorders. In Europe, the definition is a disease affecting no more than one in 2,000 people. Incredibly, there are 7,000 such diseases, prompting a saying in the field, “While a rare disease is rare, rare diseases are common.”

Rare Disease Day began in 2008. This year, events will be held in more than 90 countries.

The history of focusing on rare diseases for recognition and research began with the infamous drug thalidomide. Prescribed in the 1950s and 1960s to alleviate pregnant women’s morning sickness, the medication caused malformation of fetus’ limbs and some organs. The drug resulted in a 40 percent mortality rate of children whose mothers had taken it.

Thalidomide’s devastation led to much stricter drug development requirements. Drug manufacturers subsequently focused on common diseases, where the large number of potential patients would increase the likelihood of a new drug’s profitability. Rare diseases were “orphaned” by this chain of events, languishing with little or no research.

In December 1981, Rep. Henry Waxman, D-CA, presented a bill in Congress called the Orphan Drug Act. The bill called for incentives for drug companies to develop drugs for rare diseases. Incentives included tax breaks, enhanced patent protection and marketing rights, clinical research subsidies, and the creation of a government-run enterprise to engage in research and development. The Orphan Drug Act became law in January 1983.

Drug companies are nearly unanimous in declaring the success of ODA. Critics argue that some of the drugs developed under the act were in the queue anyway. Nevertheless, statistics show an amazing difference. In the decade previous to 1983, fewer than 10 such drugs came on the market in the United States. In the two decades after, more than 1,100 orphan drug designations were granted and about 250 orphan drugs received marketing authorization.

This research has resulted in treatment for 200 rare diseases thus far. In just the past few years, new medicines help patients with ailments such as cystic fibrosis, several enzyme deficiency disorders and more than a dozen new cancer therapies. Furthermore, research on complicated diseases often provides insights into other rare or common diseases, leading to other advancements.

Ninety percent of rare diseases still lack FDA-approved treatments, including our family’s disease of “choice,” PSC (primary sclerosing cholangitis). You likely know someone who suffers from a rare disease, whether or not they have revealed that to you. If you have no connection to a rare disease, celebrate your health and the knowledge that progress made here aids other conditions.

You can support Rare Disease Day by wearing zebra stripes (or fish stripes — I recently spied some tropical fish bearing zebra coloration while snorkeling in Hawaii). Use the social media hashtags #PSCPartners, #showyourstripes and #rarediseaseday. Or tag PSC Partners and NORD in your social media posts:

» Facebook: @PSCPartnersSeekingACure @rarediseases

» Twitter: @PSCPartners @RareDiseases

» Instagram: @psc.partners @rarediseasedayus

— Karen Telleen-Lawton serves seniors and pre-seniors as the principal of Decisive Path Fee-Only Financial Advisory in Santa Barbara. You can reach her with your financial planning questions at [email protected]. Click here to read previous columns. The opinions expressed are her own.

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