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.

 

 

 

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