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Letters to the Editor

Readers: Training Patients To Advocate For Their Illnesses Is ‘A Virtue Not A Sin’

Letters to the Editor is a periodic KHN feature. We welcome all comments and will publish a selection. We edit for length and clarity and require full names.

The recent story Drugmakers Help Turn Patients With Rare Diseases Into D.C. Lobbyists (Tribble, 4/10) drew a strong reader response. Here’s a sampling:

Encouraging patient advocacy is a virtue not a sin. Your article fails to put the gap in treating rare diseases in context. There are tens of millions of patients with rare diseases but about 95 percent of them have no effective treatment. Pushing for more research and cures is not venal or symbiotic with the pharmaceutical industry policy agenda. Rather, patient advocates — often parents of very sick children — need the resources necessary to push the government toward helping those patients.

— David Beier, San Francisco

I was extremely distraught by your recent story. … You have grossly misrepresented the outstanding work advocates Shira Strongin and Emily Muller do. … You have painted them, the advocacy groups they work with and the legislation they have worked on as corrupt and solely fueled by the pharmaceutical industry. [But] they selectively choose advocacy groups to work with, such as EveryLife Foundation, and give all of themselves to bettering the world, creating a reality where people with diseases … can live to see another day and can build happy and fruitful futures. I have seen the efforts of their labor firsthand, and they were born not out of the influence of the pharma industry but out of grass-roots labor.

— Shannon Linford, Leesburg, Va.

I have a rare disease and am the president of a patient foundation. I have also participated in Rare Disease Week on Capitol Hill two times. I could not disagree more strongly with this article. I have never received any message that I am supposed to push when meeting with my representatives. Nor am I asked to share a sob story. What I am given [is] information on how our government works. … We might have a better functioning process if all individuals were given the opportunity to learn hands-on. … If anyone is using patients to their gain, it was the author. Disgusting.

— Andra Stratton, Los Lunas, N.M.

When you or a loved one gets sick, you assume there will be a treatment. However, if you are one of the 30 million Americans diagnosed with a rare disease, there is only a 5 percent chance that your disease will have an FDA-approved treatment.  What’s worse is that treatments that have already been developed for more common diseases could help millions of rare disease patients, but our health system does not provide sufficient incentives to bring these treatments to those who need them the most.

Rare-disease patients are underserved by virtually every aspect of the health system from diagnosis to availability of treatments to reimbursement because they have not been heard by policymakers and regulators. Our community deserves better, which is why the EveryLife Foundation for Rare Diseases works to ensure rare-disease patients and their families have the opportunity to impact these life-and-death policy decisions. There is real hope in bipartisan legislation such as the OPEN ACT (HR 1223), which would bring lifesaving treatments to rare-disease patients quickly, safely and affordably.

Kaiser Health News’ recent article undermines the value of the patient voice in the legislative process, demeans patients and damages the democratic process. We believe that participation in our democracy should not be available only to people who have the financial means to travel to D.C. We submit grant applications to both foundations and industry to cover our program costs so we can make our events free and provide some assistance to patients and caregivers to offset travel costs. We received 397 applications for travel assistance, but due to limits on funding we were able to provide assistance only to 95 patients. Without support from our industry partners, patients would continue to be denied a voice.

As an organization that seeks to enhance innovation so that all rare-disease patients can access FDA-approved treatments, we value our collaborations with patients, advocacy organizations, researchers, public officials and industry — which all play a critical role in the discovery, development and delivery of lifesaving medicines to patients.

— Julia Jenkins, executive director and board member, EveryLife Foundation for Rare Diseases; Max Bronstein, EveryLife chief advocacy and science policy officer; and Mark Dant, EveryLife board member and president and CEO of the National MPS Society

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