1
chapter 1 Healthcare Landscape and
Drug Development
Ruchi Gupta, MS
H
ealthcare always has been – and is even more so today
– a vital aspect of human life. The healthcare landscape
continues to evolve and transform hand in hand with
evolution in the medicinal product development field, now highly
influenced by payer reform, technology, scientific advances, con-
sumer demand, and more.
These new realities and challenges impact how medicinal
products are developed and approved. Previously, we lacked
effective treatments for many life-threatening diseases now,
despite having many more treatments available, public scrutiny of
healthcare has intensified. Patients and their families want new
treatments sooner with accurate and understandable information
on how to use them. While this has led health authorities to
support innovation and advances in science and technology, it has
also increased the complexity in the global regulatory landscape.
The Food and Drug Administration (FDA) and the Euro-
pean Medicines Agency (EMA) often are the regulators who first
review these innovative treatments and lead the way in bringing
efficacious and cost-effective treatments to the general and broad-
er population.
In addition, the International Council for Harmonisation
of Technical Requirements for Pharmaceuticals for Human Use
(ICH) plays a critical role in global drug development by devel-
oping guidelines based on scientific discussions among regulatory
authorities and the pharmaceutical industry. ICH guidelines are up-
dated continuously and applied by an increasing number of health
authorities worldwide. The mission of ICH is to achieve greater
harmonization worldwide and ensure that safe, effective, and
high-quality medicines are developed, registered, and maintained in
the most resource-efficient manner while meeting high standards.1
Food and Drug Administration
FDA, an agency of the US the Department of Health and
Human Services (HHS), is responsible for protecting the public
health by assuring the safety, efficacy, and security of human
drugs and biological products, as well as other products outside
the scope of this book. FDA’s mission includes advancing public
health by supporting innovations that make medicines safer, more
effective, and more affordable. The agency is tasked with providing
the public with the accurate, science-based information they need
to use medicinal products to maintain and improve their health.
FDA plays a significant role in US counterterrorism efforts. The
agency collaborates with other US agencies, international regula-
tors, academia, trade associations, consumer groups, and others.2
Role in Global Regulatory Landscape
Many of the medicinal products consumed by people of leading
nations such as the US are produced in other countries. In the US,
FDA-regulated products are produced in over 130,000 facili-
ties in more than 150 countries.3 The agency faces challenges in
determining and confirming that its standards and requirements
have been applied in the manufacture, distribution, and storage of
medicinal products imported into the US. Given that the man-
ufacture of a product may involve multiple parties from different
countries, there are chances for the product to be improperly
formulated or packaged, contaminated, diverted, counterfeited, or
adulterated. Thus, compliance and surveillance activities overseen
by regulatory bodies across the product life cycle are a critical part
of the drug development process.4
Inspections are a big part of such surveillance activities and
depend on the stage of drug development. In situations where
noncompliance is identified, FDA may issue one of several types
of regulatory action letters, such as warning letters, administrative
license action letters, and license revocation to cease practices that
violate regulations and promote corrective action. If warranted,
the FDA also has the authority to impose civil enforcement
actions, including seizure, injunction, and prosecution.
FDA oversees the import and export of medicinal products
to ensure that the FDA-regulated products comply with the
requirements of the Food, Drug, and Cosmetics Act (FD&C
Act) and the regulations promulgated under these statutes.5
Imported products regulated by FDA are subject to inspection
at the time of entry by the US Customs and Border Protection
(CBP). Imported products not in compliance with US regulations
are subject to detention. Moreover, FDA verifies with CPB a
company’s licensure for imports, may perform random sampling,
and will issue import alerts for noncompliant products.6 A foreign
manufacturer must have a US license to import a biological prod-
uct into the US.
FDA works closely with external organizations and foreign
governments to promote product safety and regulatory consisten-
cy. Its efforts include:
• developing new enforcement and regulatory tools
• conducting more foreign inspections
chapter 1 Healthcare Landscape and
Drug Development
Ruchi Gupta, MS
H
ealthcare always has been – and is even more so today
– a vital aspect of human life. The healthcare landscape
continues to evolve and transform hand in hand with
evolution in the medicinal product development field, now highly
influenced by payer reform, technology, scientific advances, con-
sumer demand, and more.
These new realities and challenges impact how medicinal
products are developed and approved. Previously, we lacked
effective treatments for many life-threatening diseases now,
despite having many more treatments available, public scrutiny of
healthcare has intensified. Patients and their families want new
treatments sooner with accurate and understandable information
on how to use them. While this has led health authorities to
support innovation and advances in science and technology, it has
also increased the complexity in the global regulatory landscape.
The Food and Drug Administration (FDA) and the Euro-
pean Medicines Agency (EMA) often are the regulators who first
review these innovative treatments and lead the way in bringing
efficacious and cost-effective treatments to the general and broad-
er population.
In addition, the International Council for Harmonisation
of Technical Requirements for Pharmaceuticals for Human Use
(ICH) plays a critical role in global drug development by devel-
oping guidelines based on scientific discussions among regulatory
authorities and the pharmaceutical industry. ICH guidelines are up-
dated continuously and applied by an increasing number of health
authorities worldwide. The mission of ICH is to achieve greater
harmonization worldwide and ensure that safe, effective, and
high-quality medicines are developed, registered, and maintained in
the most resource-efficient manner while meeting high standards.1
Food and Drug Administration
FDA, an agency of the US the Department of Health and
Human Services (HHS), is responsible for protecting the public
health by assuring the safety, efficacy, and security of human
drugs and biological products, as well as other products outside
the scope of this book. FDA’s mission includes advancing public
health by supporting innovations that make medicines safer, more
effective, and more affordable. The agency is tasked with providing
the public with the accurate, science-based information they need
to use medicinal products to maintain and improve their health.
FDA plays a significant role in US counterterrorism efforts. The
agency collaborates with other US agencies, international regula-
tors, academia, trade associations, consumer groups, and others.2
Role in Global Regulatory Landscape
Many of the medicinal products consumed by people of leading
nations such as the US are produced in other countries. In the US,
FDA-regulated products are produced in over 130,000 facili-
ties in more than 150 countries.3 The agency faces challenges in
determining and confirming that its standards and requirements
have been applied in the manufacture, distribution, and storage of
medicinal products imported into the US. Given that the man-
ufacture of a product may involve multiple parties from different
countries, there are chances for the product to be improperly
formulated or packaged, contaminated, diverted, counterfeited, or
adulterated. Thus, compliance and surveillance activities overseen
by regulatory bodies across the product life cycle are a critical part
of the drug development process.4
Inspections are a big part of such surveillance activities and
depend on the stage of drug development. In situations where
noncompliance is identified, FDA may issue one of several types
of regulatory action letters, such as warning letters, administrative
license action letters, and license revocation to cease practices that
violate regulations and promote corrective action. If warranted,
the FDA also has the authority to impose civil enforcement
actions, including seizure, injunction, and prosecution.
FDA oversees the import and export of medicinal products
to ensure that the FDA-regulated products comply with the
requirements of the Food, Drug, and Cosmetics Act (FD&C
Act) and the regulations promulgated under these statutes.5
Imported products regulated by FDA are subject to inspection
at the time of entry by the US Customs and Border Protection
(CBP). Imported products not in compliance with US regulations
are subject to detention. Moreover, FDA verifies with CPB a
company’s licensure for imports, may perform random sampling,
and will issue import alerts for noncompliant products.6 A foreign
manufacturer must have a US license to import a biological prod-
uct into the US.
FDA works closely with external organizations and foreign
governments to promote product safety and regulatory consisten-
cy. Its efforts include:
• developing new enforcement and regulatory tools
• conducting more foreign inspections