The Food and Drug Administration (FDA) views aging as a natural process. This makes it difficult to obtain FDA approval for drugs that are intended to slow or reverse the biological aging process. Instead, drugs that target aging must combat a disease that is often the result of aging in order to prove their effectiveness and obtain approval.
However, there is increasing consensus among scientists and efforts to convince the FDA that aging itself should be considered a disease and an appropriate target for drug development.
This could be an important milestone not only for industry but also for society. If the FDA can be influenced, the resulting regulatory shift could result in the approval of drugs or treatments that generally slow down or reverse the aging process before a patient develops a disease.
Researchers who regard aging as a disease aren’t just referring to the inevitable passage of time. Instead, they regard aging as a process of degradation of our structure and function at the cellular level, whose characteristic features are genomic instability and damage to our DNA.
And the World Health Organization (WHO) supports this view — WHO describes the aging process as “… the effect of accumulating a wide range of molecular and cellular damage over time.”
Advanced chronological age is widely considered to be the biggest risk factor for developing diseases, but the aging process itself leads to fragility and mental decline, even in the absence of illness. As the WHO describes it, aging is “a gradual decline in physical and mental capacity.” In fact, WHO recently included the “age-related decline in intrinsic capacity” as a disease code in the 11th edition of the International Classification of Diseases (ICD).
This is particularly important because diseases are diseases that can be treated or are at least worth the effort, meaning that WHO regards age-related decline as a treatable condition.
The FDA is likely to revisit this issue as there is increasing scientific pressure to declare aging a drug target. Part of the problem is that the FDA needs more guidance to determine how “aging” should be measured and that it needs evidence that targeting “aging” biomarkers actually has positive effects.
In addition to pressure from industry, the FDA is also facing pressure from Congress to clarify the regulatory path for new regenerative therapies. Seven years have passed since the FDA approved the Targeting Aging with Metformin (TAME) clinical trial, the first study to combat the effects of aging. TAME is a double-blind, placebo-controlled, multi-center study aimed at investigating whether the diabetes drug metformin can protect against age-related diseases such as cancer, dementia or cardiovascular disease in study participants without diabetics. The aim of the study is to identify an indication for aging. The American Federation of Aging Research calls this a “proof of concept” that aging is an appropriate drug target. In this way, the aim of TAME is political rather than scientific.
The scientific community continues to discuss next steps, including potential age-related biomarkers, which should serve as acceptable targets for regenerative therapies. The acceptance of biomarkers to represent the aging process is not as big a regulatory leap as it seems. For example, the FDA recently stated its willingness to consider biomarkers for identifying early-stage Alzheimer’s disease.
We may only be a few well-designed trials away from FDA’s acceptance of aging as a drug target, especially as researchers bridge the gap between the TAME target and better-accepted disease targets. The emerging possibility of a regulatory process for anti-aging therapies warrants an assessment of how a new regulatory path will impact the market.
Here’s what we could expect:
- The market for regenerative therapies will expand to almost the entire adult population. Regenerative therapy companies that target the biological aging process are currently limited to treating specific diseases or conditions in order to obtain FDA approval. Medicines or therapies that come onto the market are generally only approved to a limited extent for a disease. Approval for other diseases is often granted years later.
- Eliminating disease-specific regulatory barriers, regenerative therapies would be available as preventive treatment solutions. According to David Sinclair, professor of genetics at Harvard Medical School and a leading company in regenerative medicine, the development of drugs that could prevent many diseases is progressing far more slowly than they should, as aging is not recognized as a disease.
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Put simply, therapies that target the aging process itself will have an almost limitless market compared to therapies using the same mechanism that target a single disease.
- “There is little doubt that approving drugs and therapies to treat aging in general would have a significant impact on research funding,” says Mark Mirkin, corporate and securities lawyer at Rimon PC, the firm where I work. “The venture capital industry, for example, would be highly susceptible to entrepreneurial ventures involving diagnostics, therapeutics and devices aimed at broad markets and resulting from an FDA-approved sector for age-related diseases. Start-ups, in particular university spin-offs, with federal subsidies will be attractive seed financing options for individual angel investors, angel funds and family offices, etc… early-stage venture capital funds looking for exciting new paths will follow. Such financing is sure to attract the attention of strategic investors, including Big Pharma.”
Finally, let’s talk about the prospect of democratized access to preventive care and, as a result, better communication between manufacturers and clinicians.
Disease-specific drug approvals force clinicians to deal with the prohibited use of a drug themselves to treat diseases or conditions that are not approved by the FDA. Except to warn of contraindications, manufacturers cannot talk to doctors about prohibited use. As a result, many patients outside the class for which a drug has been approved cannot receive the drug until years have passed and millions of dollars more have been spent on clinical trials as part of a disease-by-disease evaluation.
FDA approval of regenerative therapies to treat aging would ultimately put important safety information in the hands of local clinicians, provide access to reimbursement from health insurance and thus make the treatment available to the general population.
It is an unfortunate truth of healthcare in America that wealthy patients have greater access to both prevention and disease treatments than less privileged patients. This economic dichotomy would be mitigated to a certain extent by a regulatory shift to aging as a treatment indication.
Amy Baker is a partner in Rimon PC’s Orlando office and serves life sciences industry clients, including legal issues related to emerging technologies, regulatory compliance, and Food and Drug Administration and Federal Trade Commission regulations.