Q1 — 2016 :
Chapter 03
Culture and Purpose, Mentorship for Stronger Societies, and Innovation in Money and Medicine
21st Century Regulatory Systems for Today’s Innovation
Michael Hodin
November 30, 2016

For roughly two decades, experimental Alzheimer’s drugs have targeted beta-amyloid, a protein that is central to the plaques that characterize the disease. Though the beta-amyloid hypothesis is widely supported, yet another beta-amyloid drug, Solanezumab, recently failed to show a significant impact in Phase 3 clinical trials, thus joining a long line of unsuccessful Alzheimer’s therapies.

This is distressing, but sadly unsurprising news. Since the early 2000’s, Alzheimer’s drugs have faced a failure rate of over 99%. Even so, Solanezumab demonstrated the best of science and humanity, as Lilly’s scientists and researchers worked tirelessly for years with the goal of improving patients’ lives and our understanding of dementia’s underlying causes. Alas, failure was victorious yet again, but these persistent efforts emphasize the need for a breakthrough – despite the odds – to address the severe social and economic consequences of Alzheimer’s.

To continue these efforts, we must take lessons from the Solanezumab failure:

Lesson 1: We have an ethical responsibility and our own self-interest to support those leading the fight

First, we are past the tipping point to recognize the world’s profound need for an Alzheimer’s cure. By mid-century, this disease will directly affect over 130 million people worldwide and have a devastating impact on many more caregivers and families. This makes Alzheimer’s not just an epidemic, but a social crisis as loved ones will be called on to provide high-intensity care and sacrifice their own health, income, and savings.

Dementia is also a fiscal nightmare due to its strain on public healthcare systems, family resources, and the workforce. Dementia’s cost today is about one trillion dollars – roughly 1% of global GDP – and this will explode concomitant with increased prevalence, itself nearly perfectly correlated with the otherwise miraculous 21st century achievement of longevity. This spike will be overwhelming: it’s predicted that the cost of dementia will hit $2 trillion by 2030.

There are no easy solutions to relieve this immense social and economic burden. Yet, continuing to strive for a therapeutic breakthrough is both an ethical and economic imperative. This could mean exploring beta-amyloid drugs in even earlier stages of the disease, or moving towards new biomarkers and underlying scientific theories.

Lesson 2: While we wait for a cure, we must innovate in care

The harsh reality of the Solanezumab failure is that we are years and even decades away from a cure for Alzheimer’s. So, for those affected in the here and now, we must re-double our efforts to improve caregiving models. Our existing approaches are unsustainable. There are currently 7 potential family caregivers for each senior over 80; AARP estimates that number will fall to 4 by 2030. And, families are increasingly moving farther away from their parents and grandparents. The alternative to family care – moving the patient to a care facility – is a massive expense for families, and in the U.S., also a cost to our Medicare, and Medicaid programs (the average annual cost for a semi-private room in a nursing home is over $70,000 a year). And in addition to being costly, care within a facility is often improperly suited for the needs of people with Alzheimer’s.

Fortunately, there are other options – solutions that can keep people with Alzheimer’s in their homes amongst familiar surroundings for as long possible. While still a young industry, private-pay in-home care is poised to help fill the care gap, and play an important role in mitigating the impacts of the Alzheimer’s crisis. Research shows that older adults with paid in-home care have 25 percent fewer doctor visits per year, and that the home care industry saved hospitals as much as $25 billion over the last decade. This model also helps seniors to protect their dignity and independence by remaining in their own home, which is favored by 90 percent of older Americans. This is a shining example of innovation in response to America’s growing aging population. And it’s a great American export as the demands are global.

Lesson 3: Our regulatory process must stand up to 21st century aging and health realities

Like healthcare itself, medical regulations are stuck in the 20th century model – one defined by communicable diseases, which, while still with us, are of a different character than today’s health needs. We must update our regulatory approach to medicine development to get ahead of the 21st century health challenges of aging characterized by non-communicable diseases and conditions of aging. In addition, our updated approach should also be consistent with larger changes in public health. For example, the World Health Organization has its brand new strategy for this past years’ World Health Assembly to better meet the needs of older adults. We must do the same for regulatory bodies and pathways.

In the past, effective therapies for communicable diseases resulted from long clinical trials with placebo comparators against the real thing at advanced stages in the disease process. This worked for communicable diseases. But this approach simply doesn’t work today, not when our biggest threats are NCDs such as Alzheimer’s, and we increasingly need treatments to help achieve wellness and functional ability as we age. Dementia researchers recognize that their potential innovations are likely being applied far too late in the disease progression and measured against biomarkers, thus clouding efficacy measures society needs.

Yet, we now have the capacity for earlier detection and diagnosis of Alzheimer’s – perhaps even before symptoms emerge. These tools could revolutionize regulatory pathways with the right policy innovations. Draft guidance on Alzheimer’s drug development from the Food and Drug Administration and the European Medicines Agency are a step in the right direction, as they reflect the value of intervention in the preclinical stage. To incorporate this value in clinical trials, we should consider including patients who have a high risk for Alzheimer’s, but may not even show symptoms yet.

So while the Solanezumab results are disappointing, they also point to a way forward. As the impact of Alzheimer’s increases, so must our efforts and innovation. We must continue to push for scientific and pharmaceutical advances, improve care for those already affected, and update regulatory paradigms. The most recent setback is just one of many steps toward innovation.

Q1 — 2016 : Chapter 03 > 21st Century Regulatory Systems for Today’s Innovation by Michael Hodin