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Why Parents Are Often The Best Health-Care Experts For Special-Needs Kids


After President Obama signed the 21st Century Cures Act in December, media coverage whiplashed from glowing reports of the disease-fighting portions of the law to criticism of the provisions modernizing the Food and Drug Administration drug review and approval process. In attacking the Cures Act provisions related to the FDA, opponents predictably cast “Big Pharma” as a bogeyman that had “hijacked” this otherwise wonderful piece of legislation.

Another undercurrent to the criticism was hard for me to place, until one naysayer laid bare his target: parents. Now, I am one of those parents. I have a seven-year-old son with cystic fibrosis. With that in mind, back to the criticism.

People Are Not Statistics

In his Slate piece, Alan Levinovitz charged: “[P]harma companies could sponsor patient advocacy groups’ campaigns for approval, using testimony from mothers whose children participated in experimental, low-quality trials with miraculous results. Trump tweets something about how the FDA needs to get its act in gear so autistic children don’t suffer. So, they do, and a potentially unsafe, ineffective, and highly profitable medication is approved. Rinse and repeat. . . . This will be the model for medication approval driven by outrage and desperation, rather than evidence and reason.”

While few will push back against those demonizing “Big Pharma,” criticizing parents of sick and dying children smacks of insensitivity, so the attack against us is more restrained. Instead of implying we’re evil, parents are branded as desperate—read irrational. Of course there’s the perfunctory caveat: “I cannot imagine anything more painful than watching a loved one suffer or die, only to find out that speedier approval might have saved them.”

No, you may never know what it is like to receive a telephone call from the state’s Newborn Screening Office when you and your six-day-old son had just fallen asleep. You may never know what it is like to “hear” that pregnant pause when you ask if anything is wrong. And you may never know what it is like to learn that your only child has a genetic disease that—absent a cure—will eventually kill him.

But something else you will never know: the significance of a drug’s impact on the physical (and yes, mental and emotional well-being) of a patient. It is not desperation to “pressure” the FDA to approve a drug to treat a devastating disease where there is no current treatment, even if the benefit is only “minimal.” The FDA sees statistics. On paper, those figures may appear minimal, which is why those living with the disease must put study results in context. Such testimony is not unscientific and emotive, it is pragmatic; parents and patients are not asking the FDA to ignore clinical results, but to understand them.

Numbers Need the Context Our Lives Provide

For instance, when the FDA considered Vertex’s application for approval of Orkambi, a drug to treat the underlying cause of cystic fibrosis, the top-line results appeared minimal: the drug improved lung function only 2.6 to 4.0 percent. But a person with cystic fibrosis (or a parent) can explain what that “minimal” improvement means in the daily life of an individual suffering from cystic fibrosis. They can put into context the other benefits, such as the 30 to 39 percent reduction in exacerbations for study participants: fewer days missed of work and school, fewer days on antibiotics or in the hospital.

Similarly, parent and patient testimony provides the FDA with a perspective on how much risk is appropriate. Every drug has a risk. Those suffering (or loving someone who suffers) are best able to convey the acceptable level of risk—something for which there is no scientific litmus test. And no one is more concerned about the dangers of drugs than the parents.

Whether a risk is outweighed by the benefit is a value judgment, and parents and patients are best positioned to guide the FDA in its analysis because, as NPR rightly recognized in its coverage of the FDA’s approval of eteplirsen for Duchenne muscular dystrophy, “[i]t is, after all, the patients who face the risks and the benefits.” The Cures Act recognizes that reality and makes clear that the FDA must consult with patients, parents, and patient-interest groups.

Treat Parents and Doctors Like Sensible Humans

While patient input is relevant to the review and approval process, what is not relevant is cost—at least under our current regulatory system. Yet critics of the Cures Act FDA provisions inevitably support their arguments by stressing the high cost of drugs. While other countries commingle the questions of value (which considers both benefit and cost) and safety, in the United States, Congress has limited the FDA’s authority to questions of drugs’ safety and efficacy.

The Slate piece was not alone in its slighting of parents and patients. But whereas Slate portrayed us as desperate, in an opinion piece for, patients (and presumably their parents) come off as dolts: “Despite all of their clinical and scientific knowledge, health professionals need a way to convey to patients that treatments are safe and effective. The imprimatur of the FDA is currently a kind of Good Housekeeping Seal of approval. Weakening the FDA’s standards would undermine our trust and leave patients at a loss.”

To illustrate their concern, the authors highlighted a patient who had not heard of a medication suggested for her chronic pain, noting that “[a]lthough it is a commonly used drug, she had not heard of it. But she was reassured when told that it is FDA approved for this condition.”

You know what patients find equally reassuring? When their doctor explains that while a drug is not approved for a specific condition, it is extremely safe and studies have shown it is very effective in treating that condition. Or when the physician explains that the drug has been used safely for 20 years “off-label” for children because it is difficult and expensive to test the medication on children younger than six. The Cures Act will allow the FDA to also consider such “real world” evidence and, where appropriate, expand the labels.

But what about the dangers? Doctors already inform patients of risks. While package inserts often make the cure sound worse than the disease, doctors understand the likelihood of the risk, the seriousness of the risk, and the benefit. Most of the time, the risk is minimal and not even broached.

Other times, the risk is mentioned and discussed and, if serious, the doctor helps the patient weigh the benefits versus the risks. Sometimes a doctor will refuse to prescribe a medication because the doctor believes the risk outweighs the benefit. If the FDA approves a medication based on a shorter or faster approval process or based on “minimal” benefits, the doctors will know this and discuss it with their patients, too. It’s not just insulting and condescending, it’s wrong to assume that parents and patients can only understand a “stamp of approval.”

We’re Not In the Twentieth Century Anymore

Those condemning the FDA provisions of the Cures Act are failing to understand both twenty-first-century science and twenty-first-century patients (and their parents). With the advent of gene-mapping (identifying and understanding genes’ function and relationship to diseases), toxicogenomics and toxicity markers (assessing toxicity of chemical compounds), cellular reprogramming (converting skin cells into other cells—such as heart, lung, or brain cells), high-throughput screening (automated testing of chemical compounds), researchers can more readily test and identify safe compounds that will target the underlying disease.

Parents are doctors, nurses, biologists, academics, and pharmacists, too.

With the advent of technology, easily accessible scientific publications, patient advocacy groups that hire scientists and physicians, online communities, and the streaming of national and international scientific conferences, parents and patients can read, study, and learn of these advancements and join together to share their knowledge while pulling from their varied backgrounds. Parents are doctors, nurses, biologists, academics, and pharmacists, too.

This allows parents and patients to understand a disease and potential treatments in ways not possible in the 1960s. In fact, it is not uncommon for parents to learn of promising research (or evidence of safe and effective off-label treatments) before their children’s doctors: I only have one child and one disease on which to focus, doctors have thousands.

The father of a boy with Duchenne muscular dystrophy who testified before the FDA made this exact point, noting the now-dated 1962 FDA reforms were “meant to protect the consumer who was uneducated from unscrupulous drug companies. I think today we have clear evidence that we have an educated consumer here who is asking you seriously consider this given the merits of the drug.”

The mother of another son suffering from Duchenne added: “We’re not asking the FDA to lower its standards or grant wishes to a desperate community. We are a community that is well-informed, a community that funds and drives research, a community that writes draft guidance for drug development. We are here in large numbers because eteplirsen has met the safety and effectiveness standards for accelerated approval.”

In the same ways that the 21st Century Cures Act accounts for the advances in science by providing the FDA flexibility for reviewing and approving drugs, it also recognizes the advances in parents’ and patients’ knowledge. Taking into account such knowledge and perspective is not dangerous—it is the right thing to do. It is nice to see for once that Washington politicians have rejected the head-patting, experts-know-best attitude. Opinion journalists should do the same.

Disclosures: As the mother of a young son with cystic fibrosis, Cleveland has a vested interest in the continued development of drugs to treat and eventually cure this disease. Her family’s 401(k) and retirement savings include diverse holdings, including mutual funds in both the health care and pharmaceutical sectors; it also includes immaterial investments in Vertex and several individual biotech stocks.