Posted in Advocacy, Communicating for Social Change

NORD Statement on President’s Address

Today, the National Organization for Rare Disorders issued a statement in response to the President’s address to Congress on February 28:

Washington, D.C., March 1, 2017—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement in response to President Trump’s first speech to Congress:

“Yesterday evening, President Trump recognized Rare Disease Day and the 30 million Americans living with a rare disease in his first address to Congress. We are grateful for his recognition of the day that raises awareness for all individuals with rare diseases and their families. We are elated that he is joining us in our efforts.

President Trump also recognized Megan Crowley, and her father John, as shining examples of the untiring, steadfast commitment individuals with rare diseases and their families bring to finding treatments and cures for their disease. The Crowleys are one of many superstar families that deserve recognition, and we are thrilled that their tireless work was recognized on such a large stage.

The President continued, stating, ‘…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.’

We agree that FDA review processes can be improved upon to expedite the development and review of orphan drugs. Yet we disagree with the President that restraints must be slashed, or that the approval process at the FDA is preventing advances from reaching those in need.

Between 2008 and 2013, 87 percent of the 113 rare disease treatments reviewed by the FDA received an expedited review, compared to 35 percent of treatments for common diseases.[i] Seventy-eight percent of rare disease treatments were approved using one or more flexible development approaches (generally defined as an approach that does not include two adequate and well-controlled trials or uses novel endpoints).[ii]

For patients with immediately life-threatening illnesses who cannot participate in clinical trials, the FDA approves 99.5 percent of all expanded access requests submitted by physicians and companies.

We also believe that the current safety and effectiveness standards for drugs and biologics are crucial to ensuring individuals with rare diseases receive therapies that will positively impact their lives. Our patients deserve the same quality therapies as everyone else, and to weaken the standards will only threaten our population with unsafe, ineffective therapies.

The FDA already shows an incredible amount of flexibility in reviewing and regulating orphan drugs. But we can still improve the process.

First, we can adequately fund the FDA to allow them to hire and retain the experts needed to quickly and thoroughly review orphan drugs. Without experts on staff, the review of orphan therapies, and consequently the delivery of orphan therapies to the patient population, may be slowed. The Center for Drug Evaluation and Research (CDER) has hundreds of unfilled positions, and we support a paradigm in which the FDA can freely hire and retain the expert reviewers our patients deserve.

Second, we can reauthorize the critical user fee agreements that fund a substantial portion of FDA’s budget. These user fees must be reauthorized this year.

Finally, we can enact the critical reforms included in the Prescription Drug User Fee Act (PDUFA) and Medical Device User Fee Act (MDUFA) commitments. For orphan drug review, we can greater integrate the patient perspective into drug development review through the further use of patient preference information and patient-reported outcomes. We can also ensure the unique rare disease experience is reflected in orphan drug review through the integration of the FDA Rare Disease Program into orphan drug reviews. Both of these key reforms are under consideration in Congress to be included in the drug user fee act reauthorization.

We at NORD again wish to emphasize our appreciation of the attention President Trump has brought to the rare disease community. Moving forward it is our hope to partner with President Trump and his administration in supporting the needs of our community, such as with the passage of these key user fee agreements.”


[ii] ibid

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

Posted in Communicating for Social Change, Inspiration, Role Models

In Memory of Hans Rosling

I was sad to hear that Hans Rosling died today. I appreciated his unique approach to analyzing and using research data (especially qualitative data). He produced animations that made complex statistics lively and meaningful. As the BBC noted:

Facts, Mr Rosling believed, could correct “global ignorance” about the reality of the world, which “has never been less bad”.

As his son, Ola Rosling, said on Twitter,  Hans Rosling had a “dream of a fact-based worldview.” We have never needed it more. The “fact tank” he helped found, Gapminder, will carry on his dream and his work.



Posted in Communicating for Social Change, Inspiration, Role Models

David Harbour at SAG Awards

The subject of this blog is “difference-making” communication. David K. Harbour gave a stunning example in his acceptance speech at last night’s Screen Actors Guild Awards ceremony:

In light of all that is going on in the world today, it is difficult to celebrate the already-celebrated Stranger Things. But this award from you, who take your craft seriously and earnestly believe — like me — that great acting can change the world, is a call to arms from our fellow craftsmen and – women to go deeper and through our art, to battle against fear, self-centeredness, and the exclusivity of a predominantly narcissistic culture and through our craft to cultivate a more empathetic and understanding society by revealing intimate truths that serve as a forceful reminder to folks that when they feel broken and afraid and tired, they are not alone.

We are united in that we are all human beings and we are all together on this horrible, painful, joyous, exciting, and mysterious ride that is being alive. Now, as we act in the continuing narrative of Stranger Things, we 1983 Midwesterners will repel bullies. We will shelter freaks and outcasts, those who have no home. We will get past the lies. We will hunt monsters. And when we are at a loss amidst the hypocrisy and casual violence of certain individuals and institutions, we will, as per Chief Jim Hopper, punch some people in the face when they seek to destroy what we have envisioned for ourselves and the marginalized. And we will do it all with soul, with heart, and with joy. We thank you for this responsibility!



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