Posted in Communicating for Social Change, Compassion, Conflict, Political communication

50 Years Ago: MLK Riverside Speech

Fifty years ago today, April 4, 1967, Dr. Martin Luther King delivered a powerful speech in which he connected the U.S. civil rights movement to opposition to the Vietnam War.

MLK-quote-riverside 779x400

His words about nonviolent action, compassion, and social change remain meaningful today – especially today.

Here is the true meaning and value of compassion and nonviolence, when it helps us to see the enemy’s point of view, to hear his questions, to know his assessment of ourselves. For from his view we may indeed see the basic weaknesses of our own condition, and if we are mature, we may learn and grow and profit from the wisdom of the brothers who are called the opposition.




Posted in Advocacy, Health Communication, Political communication

More on a Health Care Solution in Search of a Problem

Yesterday, I posted a response to the portion of the President’s February 28 address to Congress where he used the example of a young woman suffering from Pompe Disease, and her father’s heroic efforts to develop a treatment for the disease, as fodder for an attack on the FDA. I was motivated by the striking disconnect between the problem – developing effective treatments for rare diseases and making them accessible to patients – and the President’s proposed solution (gut the FDA). It was as if he had a solution in mind and then went looking for a dramatic problem that could justify his solution. The pieces just did not fit.

Since the President’s speech, others have dug deeper into this dubious connection between problem and solution, questioning the truth of the President’s claim that government bureaucracy was responsible for keeping new treatments from reaching patients who needed them. Their reports demonstrate that, contrary to what the President stated, the FDA process is not the problem in getting accessible and effective treatments to patients with rare diseases:

Herper, M. (2017, March 1). Would Trump’s FDA deregulation create an age of miracles? Don’t bet on it. Forbes. Retrieved from

Huron, J. (2017, March 1). NORD issues statement on President Trump’s Address to Congress. National Organization for Rare Disorders. Retrieved from

Johnson, C. (2017, Feb. 28). The backstory behind the rare disease patient Trump highlighted to Congress. The Washington Post. Retrieved from

Raymond, L. (2017, March 1). Trump’s disingenuous attack on the FDA highlights the precarious future of medical research funds. ThinkProgress. Retrieved from

What most of these authors did conclude, however, as I concluded yesterday, is that various other features of the President’s agenda may pose more of a danger to patients with rare diseases than FDA procedures. Health care policy is complicated. People’s health, welfare, and lives are at stake. We should be wary of the oversimplification, linear thinking, and easy scapegoating that serve a purely political agenda.

Posted in Advocacy, Opinion, Political communication

Rare Diseases: The Day After

The buzz went around the Facebook pages of the Pompe patients groups yesterday that Megan Crowley, a 20-year old Pompe patient, would be sitting beside the First Lady during the President’s address to Congress. Megan is not just any Pompe patient. Her father is John Crowley, who spearheaded the research into a treatment for Pompe disease and thereby saved Megan’s life and countless others, including mine. (For a dramatic retelling of his story, see the film Extraordinary Measures.) While there is still no cure for Pompe, it is no longer a death sentence, thanks to the enzyme replacement treatment John Crowley helped develop. We are all deeply indebted to the Crowley family.

During last night’s address to Congress, the President called attention to Megan, her father, and their story. I felt a sense of both gratitude and inspiration as the camera moved to Megan and her father in the gallery. I was also grateful to the President for mentioning Rare Disease Day. Sadly, my warm response ended there. Within less than a minute, the President’s true agenda emerged. He used the Crowley’s story as a prop for his mission to destroy federal agencies and eliminate federal regulations:

An incredible young woman is with us this evening who should serve as an inspiration to us all.

Today is Rare Disease day, and joining us in the gallery is a Rare Disease Survivor, Megan Crowley. Megan was diagnosed with Pompe Disease, a rare and serious illness, when she was 15 months old. She was not expected to live past 5.

On receiving this news, Megan’s dad, John, fought with everything he had to save the life of his precious child. He founded a company to look for a cure, and helped develop the drug that saved Megan’s life. Today she is 20 years old — and a sophomore at Notre Dame.

Megan’s story is about the unbounded power of a father’s love for a daughter.

But our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need.

If we slash the restraints, not just at the FDA but across our Government, then we will be blessed with far more miracles like Megan.

Let’s think about this for a minute. Is the FDA approval process really the most critical issue facing people with rare diseases? In taking this particular turn from Megan’s story, from all the other possible lessons to be learned from her life, her father’s work, and the daily lives of other patients with rare diseases, the President revealed his underlying beliefs. In his view, the government is an impediment to progress; laws and regulations are restraints that must be slashed indiscriminately; and society will be better off if we allow unrestrained corporations to do business whatever way they want to do business.

I am not the first to observe that, in a capitalist system, government regulation is required to protect citizens and the common good, because corporations operate on one motive only: making a profit. Still, let me offer a few observations about the role of government regulation in the lives of people with rare diseases:

  • Drug Trials. The FDA does not “keep advances from reaching those in need.” The FDA is charged with protecting the public health. Drug companies must seek FDA approval when they want to bring a new drug to market, and they must demonstrate the safety and effectiveness of the drug through clinical trials. This takes time. Vulnerable, sick people participate in those clinical trials, and they need protection from the pressures of the profit motive. Let’s not forget that people sometimes die in clinical trials. Likewise, the people who will eventually acquire the drugs on the open market need protection, too. I shudder to think of the potential for abuse in a profit-based system for drug development that does not offer a counterbalance of consumer protections. Those protections come from government regulations.
  • Orphan Drug Act. Drug companies that want to develop treatments for rare diseases can take advantage of the Orphan Drug Act, which falls under the purview of the FDA. The sole purpose of this act is to give financial and other incentives to drug companies so that they will take an interest in developing treatments for rare diseases. Before the Orphan Drug Act, drug companies showed little interest in rare diseases – the population of patients was too small to be profitable. Now, orphan drug development is a booming industry. I wonder if the President is aware of this, and that it was government regulation that made these advances possible when the profit motive itself is what stood in the way.
  • Health Care Costs. What can keep advances in medicine from reaching patients with rare diseases is, quite simply, the cost. Advances in medicine mean very little to patients who cannot afford to access medical care. Patients with rare diseases face significant, sometimes staggering, health care costs. Rare diseases are often multiple-system diseases, which means patients must consult on a regular basis with a team of specialists and they sometimes must take multiple medications. Orphan drugs are among the most expensive on the market. In addition to their medication, some patients need wheelchairs, walking frames, respiratory therapy and support devices, in-home assistance, special diets, physical therapy, orthotics, and more. Obviously, affordable health insurance with realistic coverage terms is essential. Patients with rare diseases need insurance with affordable premiums and co-payments, affordable annual deductibles, no exclusions for pre-existing conditions, no annual or lifetime caps on coverage, all-tier drug coverage, and broad physician networks that include a wide variety of specialists. Moreover, many will have to be able to purchase this insurance individually because they do not have employer-sponsored health insurance. In the President’s zeal to “repeal and replace” the Affordable Care Act, I would like to hear more about how he will address these issues.
  • Quality of Life. There is one more area where protective government regulations matter for patients with rare diseases: the quality of life issues. Children with rare diseases need access to education, which means they need the protection of the Individuals with Disabilities Education Act (IDEA). People of working age may need the protections of the Americans with Disabilities Act (ADA), so that they can continue working for as long as they are able. People who can no longer work need access to a livable disability income, or Social Security retirement benefits, as well as Medicare or Medicaid to cover their medical expenses. I once read that Americans with disabilities are “the poorest of the poor.” I do not see how slashing government regulation will improve this condition. If anything, it will exacerbate it.

So, if the President truly wants to show support for people with rare diseases, he can do a lot better than promising to slash FDA regulations. He could pursue an entire agenda that would reflect a real understanding of the complex and difficult situations of people with rare diseases as well as genuine caring for them and their families.




Posted in Communicating for Social Change, Media Communication, Political communication

TED-Ed: What Orwellian Means

In my last post, I captioned an image with the word “Orwellian,” intending a reference to the works of George Orwell, such as 1984 and Politics and the English Language.  Now, The New York Times reports that 1984, published in 1949, is surging to the top of the Amazon best-seller list.

What does “Orwellian” mean? This short video by Noah Tavlin for TED-Ed lays it out clearly and beautifully. While authoritarian governments are known to employ Orwellian tactics, Orwellian means more than just authoritarianism. The term refers to how people in power manipulate language to control the thoughts, opinions, and actions of others, and thus, their very reality. Deception is a key linguistic strategy for manipulation, and a constant barrage of deception diminishes the individual’s sense of reality. This makes the individual dependent on those in power for “truth,” which serves the goal of promoting unquestioning adherence to the ideology of the powerful.

After watching Tavlin’s video, I was reminded of how deeply communication is entwined with projects of social change. I was also reminded that, while “communication for social change” sounds like a positive endeavor, the term itself has no inherent morality: communication can be used to foster both constructive and destructive social change. Communication is simply a tool, one that immoral actors can wield as easily and well as moral actors.

I am heartened to see the renewed interest in 1984. In a period where political leaders routinely and unabashedly lie in the face of concrete and easily retrievable contrary evidence, where a President can tell citizens not to believe photos of inauguration crowds that they can see with their own eyes, where newspaper editors debate whether it is proper to call a lie a lie, where an advisor to the President tries to silence the media by making it “the opposition party,” where another advisor to the President suggests that there are such things as “alternative facts,” we have never needed Orwell’s insights more.



Posted in Communicating for Social Change, Inspiration, Political communication

Inauguration Day: On a Moving Train

Howard Zinn, the late historian and activist, once said, “You can’t be neutral on a moving train.” The train is moving. Here are some principles on which I cannot and will not be neutral:

  • All people deserve to be treated with dignity, respect, and compassion. This includes especially those people who seem to be easy for others to marginalize, demonize, and victimize: women, people of color, the disabled, immigrants, members of the LGBTQ community, the elderly, the poor, the sick, prisoners, and people who choose religions other than Christianity as well as those who choose not to affiliate with any religion at all.
  • We must uphold and defend our Constitution, including our First Amendment protections for freedom of speech, freedom of the press, freedom of assembly, freedom of religion, and freedom to petition our government for redress of grievances.
  • We need to maintain the separation between church and state.
  • A vibrant democracy requires a free and independent press.
  • Education is a public good that creates an informed citizenry. We must support access to free, high quality public education for all.
  • Our public officials should be accountable, ethical, transparent, and truthful.
  • Service to citizens is the essence of government. Our government officials should not enrich themselves, their families, friends, or donors, at the expense of the citizens they serve.
  • Health care is a human right.
  • We need to protect the environment and the world’s natural resources from greed and undue reliance on fossil fuels.
  • We have a right to live our lives in privacy, free of government and corporate surveillance.
  • Severe economic inequality destroys the fabric of society. We must recognize that laws that unjustly enrich the few at the expense of the many, rather than any individual’s lack of effort or merit, are often at the heart of economic inequality. When we see laws that foster inequality, we must fight to change them.
  • People should come before profits.
  • Corporations are not people.

These principles are not radical. They have been expressed, to greater or lesser extent, in the Universal Declaration of Human Rights and also The Constitution of the United States. On these principles, I am not neutral and will not be silent.

Posted in Communicating for Social Change, Health Communication, Political communication

Rare Diseases: When Health Care Policy Kills

People with rare diseases are among the most vulnerable to changes in health care policy. Treatments for rare diseases, especially rare genetic diseases, tend to be expensive. The people who suffer from each disease do not comprise a large enough population to be heard in policy discussions. Thus, it is easy for governments to ignore the needs of these patients and make policy decisions that negatively impact them.

The incoming Republican administration seems determined to do all it can, as quickly as it can, to destroy the Affordable Care Act. Yet, despite the years of chest thumping about “repeal and replace,” Republicans have offered remarkably little discussion of any coherent health care policy. The reason for repeal seems to be no more complex than blind hatred of President Obama. The replacement that is supposed to be so much better remains unarticulated, both in vision and details. This slipshod approach to policy-making already has produced jitters in the health care industry, in the insurance industry, and among economists, as uncertainty and destabilization loom. But those who will suffer most from the effects of the incoming administration’s poor policy planning are those who also seem to be mentioned the least – the patients. For their sakes, it is worth remembering that health care policy can kill.

Laurence “Laurie” Hill died in New Zealand on December 30, 2016, at age 54, of respiratory insufficiency due to late-onset Pompe Disease (Harvey, 2017). Pompe Disease is a rare genetic disorder in which an enzyme deficiency causes glycogen to accumulate in the muscle cells. Patients typically experience the resulting muscle damage as progressive weakness in the skeletal and respiratory muscles. Laurie’s death at age 54 was not the inevitable result of the disease, though. Laurie died as a result of a deliberate health care policy decision by the New Zealand government.

There has been a treatment for late-onset Pompe Disease since 2010 — bi-weekly intravenous infusions with Lumizyme®, a genetically engineered enzyme replacement. While not a cure, research shows that the infusions can both extend the lives of Pompe patients and improve their quality of life. Before Lumizyme®, Pompe was a fatal disease; since Lumizyme®, it has been a treatable disease. Laurie’s government, which is responsible for health care policy and funding, refuses to cover the treatment in its health plan. In fact, when Laurie went to Parliament to appeal for treatment for the ten Pompe patients in his country, the prime minister’s security guards turned him away (Harvey, 2017). In effect, his government told Laurie to just go ahead and die.

New Zealand, through its health care policy, put Lumizyme® treatment out of the reach of ordinary patients, thereby rendering Pompe Disease a fatal disease for its citizens. Why? The short answer is that Lumizyme® is expensive. Although the precise cost of treatment depends on the patient’s weight, the annual cost per patient is estimated at approximately a half million dollars (Murphy et al., 2012). Thus, the New Zealand government’s decision to deny the drug to Laurie and the other Pompe patients reflects an economic calculation that, ultimately, their lives are just not worth the cost. I cannot imagine how it feels to be on the losing end of that calculation, but I may be finding out soon.

There are between 5,000 and 10,000 Pompe patients in the world. I am a member of the community of Pompe patients in the United States. Right now, we live in fear of what the incoming administration will do to limit our access to health care. Like Laurie Hill, we can be sentenced to death by government policy. The Affordable Care Act has been critical to securing our access to treatment because, among other consumer protections, it provides an annual out-of-pocket maximum for our medical costs, eliminates annual and lifetime caps on benefits, and prevents insurers from excluding those with pre-existing conditions from coverage. Eliminating the protections of the Affordable Care Act could well mean that, in the U.S., Pompe Disease will be once again a fatal disease, simply because the government put treatment out of reach of the patients.

On the same day that I received, through my Google Alerts, the story about Laurie Hill’s death in New Zealand, I also received an investment report that discussed the expectations for growth in the market for Pompe Disease treatment in coming years and the revenue-generating potential in this market. Apparently, Pompe Disease is a good investment — as long as you are not a patient. The U.S. government encourages research and development for treatments and cures for rare diseases like Pompe, most notably through such legislation as the Orphan Drug Act and the 21st Century Cures Act. Legislation provides various incentives to researchers and drug companies to develop treatments for diseases whose populations are too small to be of much interest otherwise. I am grateful for these incentives and the miracles they have produced. At the same time, I have to note that the focus of policy is heavily on the supply side.

Patients with rare diseases are on the frontier of research and development, too. It is the patients who have these rare diseases who participate in the clinical trials to test new treatments. Patients put their own bodies and lives on the line in hopes of finding treatments and cures, even if not for themselves, at least for others. Moreover, as patients undergo treatments that extend their lives, scientists gain the opportunity to learn more about their diseases. This has been the case with Pompe Disease, where improved patient survival has led to a better understanding of the mechanisms and effects of the disease, which, in turn, has generated research into new treatments. For Pompe, Lumizyme® is already considered a “first generation” treatment and “second generation” treatments are in development. Notably, too, Jules Berman (2014) argued that advances in understanding and treating many of the common diseases have resulted from, and will continue to come from, the insights researchers gain through studying people with rare diseases. None of this progress would be possible without the patients.

Patients with rare diseases do not set the prices for the drugs or any other aspect of the health care that we need. We are simply trying to live our lives as best we can, with symptoms most people do not understand. We are vulnerable to the quality of the policy decisions made by our elected officials, which could abruptly change or even end our lives. For example, a decision to reinstate caps on benefits or exclusions for preexisting conditions, or to eliminate annual out-of-pocket maximum payments, could turn Pompe Disease back into a fatal disease, not because there is no treatment, but because patients cannot afford it. Moreover, such policy decisions would create a bitter paradox in which the government uses tax dollars to support scientists and corporations in the development of treatments for rare diseases with one hand, while with the other hand making treatments inaccessible to the very patients who need them (and who pay those taxes) (Murphy et al., 2012).

The incoming Republican administration wraps itself in pro-life language. The hypocrisy is palpable. A truly principled pro-life position is one of compassion for all, one that demands keeping health care accessible and affordable, even for the rarest among us. We Pompe patients are just one segment of the larger community of patients with rare diseases. There are approximately 7000 rare diseases, defined in the U.S. as any disease that affects less than 200,000 people. Combined, about 25-30 million people in the U.S. suffer from rare diseases. And we are all at risk of suffering the same tragic fate as Laurie Hill, just so politicians can score political points.



Berman, J. J. (2014). Rare diseases and orphan drugs: Keys to understanding and treating the common diseases. London, UK: Academic Press.

Harvey, H. (2017, Jan. 5). Much-loved teacher and counselor, Laurie Hill, loses battle with Pompe Disease. Stuff. Retrieved from

Murphy, S. M., Puwanant, A., & Griggs, R. C. (2012). Unintended effects of orphan product designation for rare neurological diseases. Annals of Neurology, 72(4), 481-490. doi:10.1002/ana.23672