Posted in Advocacy, Health Communication, Political communication, Uncategorized

NORD Press Release on AHCA

NORD Issues Statement on Today’s Passage of the American Health Care Act

Washington, D.C., May 4, 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 the passage of the American Health Care Act:

“Today the House of Representatives passed American Health Care Act (AHCA). We are very disappointed the House pursued and passed this legislation, and we will continue to do everything we can to prevent this legislation as it is currently written from being enacted.

The AHCA jeopardizes the health and wellbeing of rare disease patients across the country. The bill allows states to opt-out of critical pre-existing conditions protections, including the prohibition on annual and lifetime caps and community rating.

Community rating prohibits insurers from charging individuals higher premiums based on health status. Without a community rating, pre-existing conditions protections are rendered moot, as insurance companies will once again be capable of charging higher premiums to those with pre-existing health conditions. Community rating is essential to maintaining access to quality and affordable healthcare coverage for rare disease patients.

In addition, the bill allows states to set their own Essential Health Benefits (EHB) categories rather than complying with the ten Federal EHB categories. Both the prohibition on annual and lifetime caps as well as the maximum out-of-pocket caps apply only to the EHB categories. If the EHBs are turned over to states, states could choose to allow insurers to once again set annual and lifetime caps on medically necessary services for rare disease patients by only mandating coverage of certain EHBs. States could also allow insurers to set prohibitive cost-sharing amounts without any out of pocket cap. We strongly oppose this measure as it is unacceptable to once again expose rare disease patients to these draconian caps and cost-sharing requirements.

Under the AHCA, federal funding of Medicaid would be substantially weakened by per capita caps and block grants, resulting in states potentially delaying or outright refusing coverage for necessary care under Medicaid. Rare disease patients seeking expanded Medicaid coverage would be turned away after 2019, and additional Federal assistance for the 1915(k) Community First Choice program would be removed. Many rare diseases are financially catastrophic with families often having no choice but to become single-income households because either an adult is sick, or a parent must stop working in order to become a sick child’s primary caregiver.

We remain unconvinced that the continuous coverage proposal will adequately incentivize healthy individuals to enter the market to stabilize premiums, or that the tax credit structure will provide enough assistance to low-income individuals with rare diseases seeking coverage.

We have said throughout this process that we are committed to assisting Congress and the Administration as they navigate ACA repeal and replace. We remain committed today to ensuring that Congress enacts legislation that improves the health and wellbeing of rare disease patients across the country. With continued dialogue and collaboration, we hope to be able to support legislation that improves the lives and wellbeing of rare disease patients.”

Retrieved from https://rarediseases.org/nord-issues-statement-house-passes-american-health-care-act/?utm_source=policystatement_05042017&utm_medium=email

Posted in Advocacy, Health Communication

International Pompe Day

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Today is International Pompe Day, a day to raise awareness about Pompe disease. Pompe is a rare genetic neuromuscular disease, and most of us who have it can share frustrating stories of misdiagnosis, including being told that nothing is wrong with us (when we clearly know there is). My diagnostic journey took me through just about every specialty department in medicine. Quite a few years and dollars were involved. By raising awareness, we might be able to help other Pompe patients and medical professionals reduce the time from onset of symptoms to diagnosis. This is important because, as I learned at the Duke Adult Onset Pompe Conference last week, “time is muscle.” While there is no cure, a treatment is available that slows the progression of muscle damage. The sooner treatment begins, the better.

To learn more about Pompe disease, please visit Pompe Community.

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 https://www.forbes.com/sites/matthewherper/2017/03/01/would-trumps-fda-deregulation-create-an-age-of-miracles-dont-bet-on-it/#4c1a81443883

Huron, J. (2017, March 1). NORD issues statement on President Trump’s Address to Congress. National Organization for Rare Disorders. Retrieved from https://rarediseases.org/nord-issues-statement-president-trump-address-congress/

Johnson, C. (2017, Feb. 28). The backstory behind the rare disease patient Trump highlighted to Congress. The Washington Post. Retrieved from https://www.washingtonpost.com/politics/2017/live-updates/trump-white-house/real-time-fact-checking-and-analysis-of-trumps-address-to-congress/the-backstory-behind-the-rare-disease-patient-trump-highlighted-to-congress/?utm_term=.0c32e803b570

Raymond, L. (2017, March 1). Trump’s disingenuous attack on the FDA highlights the precarious future of medical research funds. ThinkProgress. Retrieved from https://thinkprogress.org/if-trump-really-cared-about-medical-advances-hed-increase-funding-not-slash-regulations-e59a78e30647#.ri1r1dk7f

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, 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.”

[i] https://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/UCM542141.pdf

[ii] ibid

Huron, J. (2017, March 1). NORD issues statement on President Trump’s Address to Congress. National Organization for Rare Disorders. Retrieved from https://rarediseases.org/nord-issues-statement-president-trump-address-congress/

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 Advocacy, Health Communication

World Rare Disease Day 2017

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February 28 is World Rare Disease Day, a day dedicated to raising awareness of rare diseases and improving access to care for patients and their families. For American patients and their families, this observance is especially needed, and especially meaningful, this year, as some members of the current US administration aim to dismantle the Affordable Care Act (ACA). The protections of the ACA, like coverage for preexisting conditions and the elimination of caps on coverage, are critical to patients with rare diseases. Let’s remember the needs of people with rare diseases during health care policy conversations. Better yet, let’s have people with rare diseases IN those conversations!