Legislation would establish rare disease committee

The committee would let rare disease patients connect with medical professionals at the University.

The Minnesota State Capitol as seen on May 13, 2013.

Bridget Bennett, Daily File Photo

The Minnesota State Capitol as seen on May 13, 2013.

Madeline Deninger

A legislative measure introduced at the State Capitol would give patients of rare diseases access to information about clinical research, trials and drugs.

The House and Senate bills would require the University of Minnesota’s Board of Regents to establish an advisory committee of University and Mayo Clinic medical professionals, lawmakers and patients. Authors of the measure say the committee will bridge the gap between researchers and patients and inform lawmakers about future medical policies.

Ramaiah Muthyala, research associate professor at the University’s Department of Experimental and Clinical Pharmacology, advocates for rare disease awareness and research internationally. He worked with rare disease advocates to develop the bill, which was sponsored by Rep. Matt Dean, R-Dellwood.

“There are more than 7,000 rare diseases, more than any lawmaker or policymaker could ever know [about],” Muthyala said. “Because of our professions, I thought we would be able to help them.”

The bill defines a rare disease as one that affects less than 200,000 people in the United States or one for which a drug would cost more to develop than it would produce in revenue. Patients of rare diseases, which often have no cure, would be informed of the latest clinical trials and treatments available to them.

The stigma surrounding rare diseases can sometimes prevent patients from seeking this information out, Muthyala said. One of his goals for the committee is to help patients navigate their treatment options.

“Most people don’t talk very much about health,” he said. “But unless you talk about it, you cannot get the help.”

Muthyala said the committee, which would be the second of its kind in the country after North Carolina, would let lawmakers consult doctors who specialize in rare diseases when considering legislation related to insurance policies, drug prices and screenings.

Rare disease drugs are often expensive and are not covered by insurance policies, Rep. Matt Dean said. The committee could help patients and their families voice these concerns to the Legislature.

“The idea behind the bill is to try to help families who have family members who are sick with a rare disease,” Dean said. “It’s really hard whether you have cancer or a chronic condition.”

Erica Barnes, a citizen activist for rare diseases, has testified for the bill as it has progressed through legislative committees. After her daughter passed away due to a neural disease, she began to advocate for rare disease research at the University.

She said having a child with a rare disease can be an isolating experience, and this committee could connect others going through similar struggles.

“The patient populations are very small, so it’s difficult to get information and communicate,” she said Tuesday at a House Health and Human Services Finance committee meeting.

The measure was laid over at the meeting with the intention to be included in a House omnibus bill.