Kathlyn Stone is an associate editor with HealthNewsReview.org. She tweets at @KatKStone
Now is a good time to prepare for the onslaught of public relations messages about new Alzheimer’s disease (AD) drugs that are coming down the pike.
This fall, at least six phase III AD drug trials will be wrapping up–and it’s a sure bet their findings will be published in both major and minor journals and making the rounds at medical conferences in the coming months. PhRMA, the industry trade group, wrote in a report last year that 77 AD drugs are under development, most of them in phase I safety trials. [A phase I is the first time a drug is being tested in human subjects, and is only intended to test toxicity and establish how the drug is metabolized and how long it stays in the body. Phase II trials, aimed at measuring the effectiveness of a drug using a range of doses as well as assessing side effects, typically include a few hundred people. In phase III trials, a product is tested in larger groups of people — hundreds or thousands — and for longer periods of time.]
According to a coalition of industry-sponsored groups called USAgainstAlzheimer’s, there’s cause for optimism since “27 Phase III and 8 Phase II Alzheimer’s drugs are on track to launch in next 5 years.”
But anyone who reports on health care interventions knows that undertaking a clinical trial and “launching” a new drug into the health care system are two very different things.
Everyone would like to see bona fide advances in Alzheimer’s treatment. About 5 million Americans are now living with the disease, and that number is expected to triple to 14 million by 2050, according to the CDC.
But since 2003, no new drugs have been approved for treating Alzheimer’s disease, and all of the current drugs deal with short-term management of memory loss and confusion. Sadly, for such a devastating and increasingly common disease, there are only four FDA approved medications for treating symptoms: Aricept (donepizil), Razadyne (galantamine), Namenda (memantine), Exelon (rivastigmine) and Namzaric (a combination of donepezil and memantine).
None of these drugs cure or slow progression, although initially, experts and drug-makers claimed that the drugs did slow the advancement of AD. But now that the drugs have been on the market for a dozen years or more–and have been the subject of numerous post-market trials (that are still ongoing)–it has become evident that many of these drugs haven’t lived up to earlier claims.
Along with Dr. Susan Molchan, a geriatric psychiatrist and former clinical researcher with the National Institutes of Health, we’ve put together some key considerations to be aware of and ask your sources about when writing about the new therapies under development.
When reading PR pitches about new drug interventions, consider whether the news release addresses these key questions:
1. Does the study focus on patient outcomes or surrogate markers?
Molchan suggests playing close attention to the outcomes that were measured. “Were they memory tests that perhaps reached some degree of statistical significance or were they measures of function, for example, trying to see if they allowed people to continue to dress themselves longer?” If the assessments looked instead at biomarkers or surrogate markers–such as changes in brain volume observed through imaging tests–it’s important to find out if these markers can reliably be used to claim improvements in patient’s cognitive function or their overall quality of life. Other surrogate markers commonly measured in AD research include tau protein and beta-amyloid in the cerebrospinal fluid (CSF). Tau and amyloid are often theorized to be signs of or causes of AD. The main consideration here should be: What real-life differences did the intervention make for the people taking it?
Was the drug’s main achievement that it appeared to lower levels of tau or amyloid (about which there are still doubts regarding their role)? Or was the drug shown to improve outcomes such as helping people live longer or better, with improved function? In other words, are tau and amyloid meaningful surrogates for the outcomes we really care about, or not?
We’ve written posts about how these surrogate markers have been hyped, about how the amyloid theory doesn’t prove cause-and-effect, and how claims about amyloid being the cause of AD have gone unchallenged.
Our story reviewers found that STAT did a solid job describing amyloid uncertainty, as did NPR in their summary of a study on an experimental Alzheimer’s drug.
2. Does the research rely on a subgroup analysis?
We’ve been dismayed recently to see an increase in the number of study findings based on highly selective subgroups of patients that yield more favorable outcomes. These smaller, homogeneous groups don’t reflect how the intervention will perform in a larger population. This Buzzfeed story illustrates how a subgroup analysis was used to skew results in a trial of a drug designed to dissolve tau protein in patients with AD. And here’s our review of a news release on an experimental AD drug. It focused on a small subgroup analysis from which conclusions can’t be drawn.
3. Who were the patients?
Did the study volunteers have mild or moderate AD or mild cognitive impairment? (AD and mild cognitive impairment are different conditions with different pathologies and paths of progression, and yet they are often referred to interchangeably in PR releases and news reports.) Were the volunteers cognitively OK but genetically at risk for Alzheimer’s? If so, the evidence will not be as reliable as a randomized controlled trial involving only those already diagnosed with moderate AD, for example.
4. Do the benefits justify the costs?
If costs aren’t mentioned, they should be. Based on current trends, drug companies will charge tens of thousands of dollars a year for any new AD treatments that gain FDA approval. If the new drugs are similar to the current treatments–providing only minimal benefits–it will be hard to justify this. Given America’s crisis over health insurance costs and access to health care, “how could we possibly hope to afford this?” Molchan asks. “We will still have people with dementia, only more of them on expensive drugs for years.”
5. Has the research been peer-reviewed and published?
“All of the studies will have corporate sponsorship,” Molchan says, “so peer review will be important.” Peer review is the widely accepted method of research validation whereby work is subjected to scrutiny by peers, mainly through publication. Submitting one’s work at a medical conference is a form of peer review, too, but that work is often preliminary and not yet published. Reporters should look carefully at how study limitations are reported, and follow-up with the researchers and independent sources to capture a true picture of the benefits and the evidence.
It’s possible to write about even preliminary research in a responsible way. Managing editor Kevin Lomangino’s post gives a good and a bad example of writing about a study of resveratrol — an antioxidant compound found in grapes and red wine — in patients with AD. In this case, the news release was measured and balanced, while the news story relied on hype to tell the story. (Often it’s reversed.)
Of course, many of these strategies for reporting accurately about AD drugs also can be applied to research news about nutritional and other alternative therapies for AD patients. We’ve reviewed many news releases and news stories that didn’t cut the mustard. See “Nutritional drink to protect memory in ‘early Alzheimer’s’? Release takes us for a spin,” “CBS News story on Alzheimer’s treatment follows familiar formula,” and “Sleeping on your side to prevent Alzheimer’s? Rat study conclusions applied too broadly to humans.”
If you see headlines that include words like “breakthrough” or “cure,” be skeptical on behalf of newly diagnosed Alzheimer’s patients, their families and caregivers. (See our primer, “7 words (and more) you shouldn’t use in medical news.”)
Receiving a diagnosis of AD is life-altering. Addressing newly diagnosed patients, the Alzheimer’s Association says,
“A diagnosis of Alzheimer’s disease can leave you feeling disconnected, isolated or abandoned from others. You may feel unsure of where to turn and that no one can possibly understand what you’re going through. People living with early-stage Alzheimer’s have stated that one of the most important lessons they learned early on in their diagnosis is this: they could not just wait for others to help them – they had to go out and help themselves to the best of their ability.”
For many people, self-help means researching their disease and the treatments available to them. Journalists can help these patients by providing balanced, hype-free information about new therapies.
Veteran medical journalist Larry Husten, who slices and dices hype in medical news almost every day, suggested there’s a cardinal rule to follow when dealing with health news:
“Don’t believe the hype! That’s the cardinal rule to obey when reading health news. “Breakthroughs” and “cures” are rare, and should always be viewed with caution and skepticism.”