‘Disease Adovcacy’ Has Changed How Medical Research Is Funded

October 8, 2012 by

Repost from CBS Detroit

ANN ARBOR — Patient-led advocacy has created a shift in the way the United States government prioritizes funding for medical research and has significantly changed the way policymakers think about who benefits the most from these dollars, says a public health researcher at the University of Michigan.

In “Disease Politics and Medical Research Funding: Three Ways Advocacy Shapes Policy,” a paper published in the October issue of the American Sociological Review, Rachel Kahn Best analyzed data on 53 diseases over a 19-year period from 1989 to 2007.

Best, a UM School of Public Health fellow in the Robert Wood Johnson Education Scholars in Health Policy Research Program, found that those diseases tied to strong advocacy organizations received millions of dollars more in research funding over the period than others whose advocates were not as strong. She also found an increasing number of these organizations, from about 400 large nonprofits working on disease advocacy in the early 1990s to more than 1,000 by 2003.

In addition, Best noted another fundamental shift in policy brought about by advocacy. Where policymakers once focused on providing dollars to the scientists who made the best case for funding — with the general population thought of as the beneficiaries of their research — the government began to think of patients with particular diseases as the recipients of the research funds. This resulted in funding based on “perceived moral worthiness.

(Read entire article…)

Can Our Society Afford to Provide Treatments for People with Rare Diseases?

October 3, 2012 by

REPOST:   A Medical Adviser to the National Organization for Rare Disorders (NORD) Responds to this Question

 

WASHINGTON DC, Oct. 3, 2012—-Can our society afford to provide costly medical care for patients with rare diseases, or would that money better be spent on treatments for more common conditions affecting larger numbers of people?

That was the question raised in a recent publication of the Hastings Center, a research institution dedicated to bioethics and the public interest.

Now a medical adviser to the National Organization for Rare Disorders (NORD) has responded to the report, writing that viewing rare diseases as “peripheral and unimportant” to the healthcare system would be a major mistake on several levels.

“Not only does it seem to justify abandoning millions of people but it also can undermine the integrity of the entire research enterprise,” notes Doris T. Zallen, PhD, professor of science and technology in society at Virginia Tech and a long-time adviser to NORD.  “It can reduce the chance of finding successful treatments for ALL diseases — common and rare alike.”

A rare disease is defined in the U.S. as one affecting fewer than 200,000 Americans.  There are approximately 7,000 such diseases affecting nearly 30 million Americans, two-thirds of them children.  Only a few hundred of these diseases have medical treatments, and only a small number are being studied by researchers to develop treatments.

The current investment in research on rare diseases is small, Dr. Zallen writes, and further reducing research on rare diseases by deciding that our society can’t afford the cost of providing treatment would “harm the broad scientific enterprise” by discouraging young scientists from choosing that career path.

Because rare diseases often have a singular genetic basis, she writes, “the study of rare diseases has already provided fundamental understandings of genetic systems, biochemical pathways, and DNA-repair mechanisms that have helped elucidate the basis of, and improve treatments for, common diseases.  The blockbuster drugs Botox and Viagra were originally developed to treat benign essential blepharospasm and pulmonary hypertension, two rare disorders.”

Since there is little research funding for rare diseases, many patient groups try to raise money themselves, conducting car washes, bake sales and garage sales.  Often, it takes many years for these small patient groups to raise enough money to fund a study.

At a recent meeting of NORD’s medical advisers, Dr. Zallen writes, “we struggled to decide which of two excellent research grant submissions should receive the modest available funding.  Even with all its hard work, the patient group providing the funding had raised only enough money for one seed grant.”

People with rare diseases are not asking for “preference” when it comes to allocation of resources but only a “fair shake”, Dr. Zallen adds.  “The tax dollars used for government research programs come out of their pockets too, and society has an ethical duty not to punish minorities because of their small size.  Not only they, but the wider community as well, would benefit — since such research is essential to the health of the overall medical science enterprise.”

Dr. Zallen has received numerous awards and honors for her teaching, research and outreach activities.  An expert on topics related to bioethics, she helped write the federal guidelines to protect human subjects in gene therapy experiments and is the author of the book, Does It Run in the Family? A Consumer’s Guide to DNA Testing for Genetic Disorders.

The full text of Dr. Zallen’s response and a link to the original article are available on the NORD website (www.rarediseases.org).

Established in 1983, NORD is a nonprofit organization representing all patients and families affected by rare diseases and all seeking to help them.  It provides programs of advocacy, education, research and patient services. 

—————————————————

CONTACT:
Mary Dunkle
mdunkle@rarediseases.org

 

Letting Children Share in Grief

September 20, 2012 by

Repost from the New York Times, written by Catherine Saint Louis

STRENGTH IN NUMBERS Camp Erin in Palm Desert, Calif., a grief camp, offers children a place to discuss death without feeling self-conscious or worrying about making friends cringe.

 

A FEW decades ago, children often didn’t attend funerals. The thinking was that they should be sheltered from the pain of losing a loved one. And as Americans started living longer, the need to even broach the subject of death was delayed because many grandparents survived deep into their golden years.

But recently, the opposite view — that children should be as involved in the grieving process as adults are — has been taking hold, reflecting an increasingly common belief that children are better off when their grief is acknowledged and they are allowed to mourn in the company of relatives and peers.

Grief centers for children are one example: there are now more than 300 of these nonprofit counseling centers, up from 204 in 2002. And Donna Schuurman, the executive director of the Dougy Center for Grieving Children and Families in Portland, Ore., which helped establish these centers, estimated that there are at least 150 more peer-to-peer programs nationwide that serve a similar function. The rise of hospice care, which provides bereavement services for relatives, including children, has also played a role, as have grief camps for children.

“Twenty-five years ago, children were ‘invisible grievers,’ ” said Vicky Ott, executive director of Fernside, a nonprofit center in Cincinnati that served 1,300 children and adults last year.

Continue reading…

 

Crowdfunding helps little girl get one step closer to a diagnosis

September 1, 2012 by

Reposted from Talking Points Memo.  Written by Carl Franzen.

Crowdfunding, or fundraising over the Web by soliciting a mass of donors, is good for more than just financing pet projects like music albums and iPhone accessories.

Just ask the Nieders: The New York family of three (and one dog, Parker) have been searching for answers to the cause of four-year-old daughter Maya’s global developmental delays. She hasn’t been able to speak aside from a few short words for most of her life.

Now, thanks to crowdfunding, they may have a clue: Researchers at the Rare Genomics Institute, a nonprofit founded in 2011 dedicated to helping patients with unique genetic diseases, on Tuesday reported that a genome sequencing test performed on Maya’s DNA and paid for with online donations has uncovered a previously unknown gene variant that could be responsible for Maya’s developmental challenges.

“Though we need to do further research to confirm this first gene discovery, it is a major milestone,” said Jimmy Lin, a geneticist at Washington University in St. Louis and the principal founder of the Rare Genomics Institute, in a statement.

(Read more)

«Older Posts