Isaac MB1*, Isaac EL2 and Isaac MT2
1European Medicines Agency (EMA), Netherlands
2Department of Geography, UK
*Corresponding author: Maria B Isaac, European Medicines Agency (EMA), Amsterdam, Netherlands
Submission: September 18, 2019; Published: March 06, 2020
Volume2 Issue1 March 2020
More than one billion people in the world live with some form of disability, of whom nearly
200 million experience considerable difficulties in functioning as reported by the WHO [1]. In
the next years this will growth due to ageing populations and the higher risk of disability in
people living with chronic health conditions such as diabetes, cardiovascular disease, cancer,
infectious diseases and mental health disorders.
As example, an estimated 43.8 million people globally live with dementia (2016 figures).
Nichols et al. [2] highlighted potentially preventable associated links with global disease
burden, such as high BMI, high fasting plasma glucose, smoking, and a high intake of sugarsweetened
beverages. We continue to be confronted with unwelcome negative findings of
new medical treatments of the Alzheimer’s disease (AD) with trials based only on the clinical
pharmacology of amyloid pathology. In 2019, Knopman et al. [3] reported the failure of BACE
inhibitors and monoclonal antibodies in the treatment of mild cognitive impairment (MCI),
mild and moderate AD, where the cognitive decline of a patient can be discerned at as early as
13 weeks of taking the trial drug. The results using MRI biomarkers suggested a decrease in
cortical thickness due to diminution of inflammatory modulation following administration of
drugs that target amyloid levels.
For example, vector-spread infectious diseases, diet, climate change and environmental
contamination are well defined epidemiological factors, of which there is a need of finegrained
measurement. Current vaccines and advances genetic therapies in Spinal Muscular
Atrophy [4], cancer and dementia developed have not included any of these new technologies
or methodologies to develop a better understanding of prevention, or what is the best time to
use them in the natural history of the disease. The primary interests of scientific regulators are
public health and providing patients with access to the right drugs, at the right time and with
the right dose. There is also great focus on facilitating patient access to medical treatments
for people suffering of neurodegenerative disorders and intellectual disability which are in
growing global need, among other medications for a variety of illnesses and disorders. In
an ever-evolving process, the European Medicines Agency (EMA) and the Food and Drug
Administration (FDA), have made positive progress and have updated their respective
guidelines on the qualification of novel methodologies for biomarkers and clinical outcomes
[5-8] and new regulatory indications to reflect an improved methodological approach and
ensure better regulatory clinical trials of neuropsychiatric disabilities in mental health.
Of course, regulators are members of the public, and share a common interest in the
incorporation of new research ideas to facilitate global clinical trials. There is significant
interest in the facilitation of discussion between global research funding agencies to increase
transparency of the evaluation process, as well as to stimulate collaborative research in order
to decrease the regulatory burden on researchers [9]. In the case of degenerative disorders
, both the EMA and FDA have harmonized the proposed use of biomarkers, MRI , digital
technologies and to describe people medical needs and collaborate with IMI-AUIMS and IMIPRISM
to improve the digital tools to measure behaviors in neuropsychiatric disorders [10]
However, current brain degenerative disorders models are
predicated on the presence of pathology in the brain and we have no
other information about what other environmental factors, healthy
lifestyle habits, climate changes impact in degenerative disorders.
It is apparent that novel approaches are required to resolve this
impasse, with trans-disciplinary, non-silo, thinking, involving
are age of disciplines - geographers, statisticians, physicians,
and regulators? We suggest that a conceptual parallel can be
found in existing modelling of infectious disease distribution and
development of vaccines. This is an important response to climate
change, where focused models of disease vector distribution
as a function of increasing global temperatures, environmental
factors can be measure by digital data and can inform policies of
vaccination targeting [11].
The biological rationale of diagnosis is a good starting point
to tackle the early diagnosis of degenerative disorders, and clearly
identifies strategic priorities for the study of these disorders.
There is much at stake. Not only are degenerative intellectual and
development disabilities of critical importance in our society, but
an accurate evaluation of the personal risk of developing these
illnesses also reflects the mounting demand for individualized
healthcare. It is vital that no stone is left unturned in the
investigation and search for robust treatments, especially for and in
the earlier stages of this devastating condition; and the sooner this
is done, the better. Regulators and clinicians can play their part and
are doing so in facilitating robust investigation using updated and
evolving guidelines and fora for stakeholders.
© 2020 Isaac MB. This is an open access article distributed under the terms of the Creative Commons Attribution License , which permits unrestricted use, distribution, and build upon your work non-commercially.