Cost concerns over rare disease treatment

Photo courtesy: MGNonline

BOSTON (SHNS) – As drug companies pioneer new methods of treating an array of diseases, some lawmakers want to limit the out-of-pocket cost to health plan members purchasing medications while others worry that tack would cause costs to soar.

Ilene Sussman, executive director of the VHL Alliance, which advocates for people with Von Hippel Landau, a genetic condition that involves “the abnormal growth of blood vessels,” said co-payments can run upwards of $1,000 per month.

Boston Biomedical Associates Vice President of Operations Ken Dhimitri said while about 30 percent of all new drug applications are for rare diseases and approval rates are “about even” between rare and common diseases, 95 percent of rare diseases lack a single treatment approved by the Food and Drug Administration.

Sarah MacDonald, executive vice president of the Massachusetts Biotechnology Council, said costs increase for patients who are on multiple medications because there is not a single specific treatment.

Legislation filed by Sen. Anthony Petruccelli, an East Boston Democrat who chaired the Financial Services Committee last session, would limit to $100 per month the amount of co-payments due for a covered prescription drug under an insurance plan.

Jon Hurst, president of the Massachusetts Retailers Association, said the proposed legislation would disproportionately raise costs for small businesses because larger, self-insured employers are regulated by the federal government and are independent of state health insurance mandates. Medicaid, a state and federal taxpayer-funded insurance program, is also subject to the state mandates, said Hurst, who said the state has passed 24 mandates and assessments since requiring health insurance in the 2006 effort to expand coverage.

“I’ve frankly been shocked that we continue to pass three new mandates each year,” Hurst told the News Service. He said he doesn’t “put anything past big pharma and the big providers” who stand to gain if consumers are unburdened by out-of-pocket drug costs. He said, “There is no bigger more powerful lobbying group in Massachusetts than big health care.”

Senate Ways and Means Chairwoman Karen Spilka, an Ashland Democrat, encouraged those gathered at the State House Monday to tell personal stories to their local senators and representatives.

“You are what will make a difference on whether or not we get these bills passed,” said Spilka. She said constituents’ stories give lawmakers “added weight” in making their case and said, “You are giving these rare diseases a face.”

Asked what could be done for people with abnormally expensive conditions, Hurst suggested insurers offer options for people who want additional coverage without lawmakers mandating the coverage be included in every state-regulated plan. He said Maine has a similar system.

According to a November brief by the Massachusetts Association of Health Plans, specialty drugs made up 1 percent of prescriptions written in the U.S. and 25 percent of prescription drug spending in 2012. The report highlighted the new hepatitis medication Sovaldi, which it acknowledged is a “breakthrough drug,” while noting its price tag of $1,000 per pill.

Gov. Charlie Baker signed a proclamation stating that there are 7,000 rare diseases or conditions, so defined because they each affect fewer than 200,000 Americans, and rare diseases collectively affect almost 30 million Americans. The governor dubbed this Saturday Rare Disease Day.

For people with rare diseases and their loved ones, convincing researchers to study treatments and cures can be a challenge.

Karen Peluso said her adopted daughter had neurofibromatosis (NF), a genetic disorder that can cause tumors and other maladies, and her doctor gave her the name of other families coping with NF. Peluso said that occurred in the era before a federal health care privacy law.

“In the beginning we worked out of my kitchen,” Peluso said. She said the emerging NF group tried to encourage research with “seed grants” and lobbied Congress with the help of a lobbyist. Former U.S. Sen. Tom Harkin directed medical research funding toward NF, starting with $6 million, said Peluso, who said, “We helped generate over $500 million for NF research.”

Dr. Norman Barton, vice president of clinical development at Shire, said pharmaceutical researchers have moved beyond the “low-hanging fruit” of figuring out how to reduce metabolites, which are produced by the body’s metabolism.

“The challenge now is to go in and edit the genes or to put a structural protein into a place where it has to hang out with other structural proteins and work. That’s a huge challenge. It’s much, much more complicated than trying to change the level of a metabolite or a compound that has accumulated in the body,” Barton said. He said, “It’s overcoming the barrier of launching the rocket for the first time, sequencing the first protein, etc., etc., etc. It’s that set of challenges that is in front of us right now.”

Megan Duff told the crowd she was diagnosed with NF as a young child and has faced a range of challenges, including a recent double-mastectomy, chemotherapy and radiation treatment to stop “an aggressive form” of breast cancer.

“There is no way to predict the severity of one’s NF,” said Duff, 31, who said some people with NF merely have a few “café-au-lait” spots.

Lisa Cimino described her daughter’s affliction with focal segmental glomerulosclerosis, which caused swelling as her kidneys failed to filter her blood, causing fluids to build up. Cimino said she had to limit her daughter’s water intake, giving her only a small Dixie cup when she was thirsty on a hot day, and she said her daughter learned how to “take 17 pills with one large tablespoon of applesauce.”

Cimino said her daughter is now 18, still beset by symptoms such as fatigue and thinking about studying cosmetology and taking the high school equivalency exam.

National Organization for Rare Disorders President Peter Saltonstall, a cousin of the late U.S. Sen. Leverett Saltonstall, said that while there are disparate diseases those afflicted can have a common purpose.

“Alone we’re rare. Together we’re strong,” said Saltonstall.

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