Congressman Murphy convenes “Path 2 Cures” roundtable
Dr. Pamela McInnis, a deputy director at National Institutes of Health, explains improvements, but also troubles, of clinical trials and their publication.
Donald Furko Jr., an advocate for pancreatic cancer treatment after losing his father, said detection is still a problem, so patients are left to contend with vague and embarrassing symptoms. “If you have jaundice, or, less obviously, (changes in the bathroom), you need to get it checked out. It was too late for my dad.”
Meeting in the Mt. Lebanon public safety building during a solar eclipse, patients and disease advocates told a deputy director of the National Institute of Health they feel burned on how clinical trials are often left in publishing purgatory.
“We’re increasing how we share data, but it’s still shocking the number of trials that aren’t finishing or being published … that’s dooming us to repeat trials and we don’t know what happened last time,” deputy director of translational sciences Dr. Pamela McInnes said.
A roundtable of advocates and health professionals representing everything from mitochondrial and rare genetic disorders, to common diseases like diabetes, was gathered by Congressman Tim Murphy (R-Upper St. Clair) on Oct. 23 to get feedback on how to improve the discovery of new medicines and deliver treatments out of trial stage sooner.
“The FDA approved prednisone (for pulmonary fibrosis) in 2012, two years after universal feedback that this drug was good to go and had minimal side effects. That (September 2012) meeting was useless. We found out after the fact they were already going to approve it. Why didn’t they do this in 2010?” asked James Uhrig, who has idiopathic pulmonary fibrosis.
Fast-tracking drugs could be helped, McInnes said, by having patients more directly involve themselves with clinical trials.
“Contact NIH, contact a department of Genetic and Rare Diseases Information Center … we have to start respecting patients as part of the process instead of simply the object of examination,” McInnes said, “but the problem is we need to start treating all diseases as having something in common; there are underlying factors to rare and common disease groups; let all boats rise to the top at the same time.”
Though for some patients, that analogy more than falls flat, as some have mobility issues or are diagnosed with a disease that sees little research funding and awareness. With campaigns and celebrity status attached to diseases like Parkinson’s and the recent Lou Gherig’s ALS ice bucket challenge, patient groups like those with the rare but debilitating Chronic Fatigue Syndrome, or Myalgic Encephalomyelitis, are finding being part of a clinical trial just isn’t an option.
“I understand their frustration. Chronic fatigue has such a wide range of manifestations and we’re still having trouble defining it… I was originally involved when doctors were looking for a viral biomarker, and, nothing.”
“We need more rare disease advocates. We’re not getting the funding or the attention we need, often because we’re immobilized or stuck in a wheelchair or a bed,” Chuck Mohan of the United Mitochondrial Disease Foundation said. “Remember: the ark was built by amateurs; the Titanic was built by professionals.”
Meantime, McInnes says hysteria and “the closeness” of Ebola has now made it an “economically viable” virus to treat because pharmaceutical companies are now interested.
“Despite thousands dying abroad, now that it’s hitting us at home, you’re going to see drugs start coming on the market, a vaccine perhaps, in as soon as a few years.”
Other patients at the meeting said they deal with chronic diseases that they say are difficult to deal with financially.
“Treatment for those just in remission, or relapsed multiple sclerosis, is $60,000 a year,” said Frank Austin, of the MS Society, who has stage two multiple sclerosis and is wheelchair-bound. “My hope is that Congress makes sure cost is not a barrier to treatment.”
Julie Cerrone of the Arthritis Foundation said there is a bill on Capitol Hill that could help chronically ill patients more easily afford treatment.
“Right now it’s a co-pay, and it can sometimes be hundreds each time. H.R. 460 would implement a percentage formula for cost-sharing of treatment instead of costly co-pays.”