Module 9
9.1: Introduction to Specialty Medications
In general, specialty medications are high cost medications used for treating complex disease states. They can be
challenging to both manufacture and administer, and they often require significant patient education and close
monitoring to ensure their safe and appropriate use. Although specialty medications were once a very small piece of the
pharmaceutical industry, there has been a significant change over the last 20 years and especially within the last decade.
Specialty medications are now the fastest growing segment of the pharmaceutical industry. Common characteristics
associated with specialty medications are listed in Table 9.1.
Table 9.1 Specialty Medication Characteristics
High Cost Potential for limited or exclusive availability for
distribution
Complex treatment regimen that require ongoing Treat rare diseases
monitoring and patient education
Special handling, storage, or delivery requirements Treat diseases known to have long term or severe side
effects or increased fatality
Biologically derived and available in injection, infusion, or Payers may define what they consider to be a specialty
oral form medication for reimbursement and contracting purposes.
With the increase in specialty medications over the last two decades, a whole new approach to dispensing these
medications emerged called specialty pharmacies. Generally, specialty medications are not available at typical
community pharmacies, so patients must obtain them through specialty pharmacies. There are many reasons for this:
some practical, some financial, and some clinical. Traditional retail pharmacy is not designed to handle these complex,
costly medications. First, the high cost alone, in many cases, would prohibit retail pharmacies from stocking the
medication. Second, the often-busy nature of a retail pharmacy does not align itself well with being able to appropriately
manage and support the needs of patients with complex disease states. For these reasons, the first specialty pharmacies
began in the early 2000s. Specialty pharmacies are typically required, through their contract with health insurance
companies, to meet unique requirements that are not part of their contracts with retail pharmacies. Examples of these
requirements are listing in Table 9.2.
Table 9.2 Examples of Specialty Pharmacy Services
Coordinating care and facilitating the drug access Case management- disease state management
Facilitating mail order delivery logistics Product device training when applicable
Working with health insurance to determine coverage Data management of technical and clinical patient care
and help coordinate any requirements of the insurance services.
company
Investigating patient assistance programs for patients Call center development
without insurance or lack of coverage
Patient Experience
A patient receiving a prescription for a specialty medication should be aware that the process for getting the medication
is going to look different than the normal retail pharmacy experience. However, the system is designed so that the
patient has the best chance of successfully obtaining and using the medication safely and appropriately. The prescription
would first be sent to the specialty pharmacy. The specialty pharmacy then takes responsibility for making sure the
patient gets the medication, understands the risks and benefits, is able to afford it, able to take it appropriately, and will
follow through with any necessary monitoring. The patient may be connected with a case manager that will call them on
a monthly basis to make sure there are no issues with the medications, check to see if they had any required monitoring
, completed, provide any necessary education, and/or answer patient questions. Patient follow-up often happens every
month prior to sending out the next months’ worth of medications in order to minimize waste, ensure safety, and assess
for efficacy. As specialty pharmacies are generally not local to the patient they are caring for, care and coordination are
often provided telephonically. The process is centralized, and the medications are shipped to the patient’s house or, in
some cases, to the facility that will be administering the medications.
Brief Overview of Common Disease States Treated with Specialty
Medications
Common disease states managed by specialty pharmacies include (1) oncology, (2) multiple sclerosis, (3) rheumatoid
arthritis, (4) Crohn’s disease, (5) hepatitis C, and (6) HIV/AIDs. This module with introduce some of the most commonly
used specialty medications that most health care professionals would come into contact with, regardless of their
specialty. Many of these conditions represent an entire specialty in medicine and are very complex. Therefore, for the
purposes of this module, these conditions will be introduced briefly.
Oncology is the study and treatment of cancer. Cancer is a disease process that involves the development and
proliferation of abnormal calls. Cancer cells are marked by both a structural change and a loss of function from the
original healthy cell. They are often characterized by multiplying at a faster than normal rate. As this collection of
abnormal cells, called a tumor, grows it can become life threatening as it deprives normal body cells of the nutrients they
need to function.
Multiple Sclerosis (MS) is an often unpredictable, disabling disease of the central nervous system. It disrupts the flow of
information both within the brain as well as between the brain and the body. Damage to the myelin coating around the
nerve fibers in the CNS causes the nerve signals to be disrupted. This damage ultimately leads to the symptoms of MS
that can vary between individuals with the disease. Some of the more common symptoms include fatigue, difficulty
walking, numbness or tingling, stiffness in the limbs, weakness, vision problems, cognitive changes, pain, depression, and
emotional changes.
Rheumatoid Arthritis (RA) is a chronic autoimmune disorder that causes inflammation and tissue damage in the joints. It
is a very painful and (often) disabling disease. The common symptoms of RA include pain, stiffness, and reduced range of
motion. Generally, the treatment would not start with a specialty medication. The initial medications fall under the class
disease modifying antirheumatic drug (DMARD). However, these are not always effective or in some instances, patients
may no longer respond as they once did. In either case, a patient would be then switched to one of the specialty
medications reviewed below.
Crohn’s Disease is an inflammatory bowel disease caused by inflammation in the digestive tract which can lead to
symptoms such as abdominal pain, severe diarrhea, fatigue, weight loss, and malnutrition. The inflammation can spread
deep into the layers of the affected bowel tissues making the symptoms extremely painful and often hard to control.
Hepatitis C is a viral infection that causes liver inflammation which can sometimes lead to serious liver damage. The virus
is spread through infected blood. There are often no symptoms associated with chronic hepatitis C until the virus
damages the liver significantly enough. Symptoms of hepatitis C then include bleeding and bruising easily, fatigue, poor
appetite, dark urine, swelling, weight loss, and confusion. Interestingly, acute hepatitis C often goes undiagnosed
because of the lack of or self-limiting nature of the symptoms. Additionally, some people that contract hepatitis C never
go on to develop chronic hepatitis C because their body is able to clear the virus on its own. However, those that do
progress to chronic hepatitis C, will likely develop liver failure and need a liver transplant should they live long enough if
the disease is left untreated. Recent treatments have made the possibility of a cure very likely with only three months of
treatment.
HIV/AIDS is a notorious virus with incredible advancements in treatment over the last 30 years. HIV stands for human
immunodeficiency virus and is the causative agent of acquired immunodeficiency syndrome (AIDS). The virus is spread
through sexual contact, perinatally from an infected mother, or by injection into the blood. The typical course of the
infection is characterized by an acute clinical illness that varies in presentation followed by a longer clinical latency.
9.1: Introduction to Specialty Medications
In general, specialty medications are high cost medications used for treating complex disease states. They can be
challenging to both manufacture and administer, and they often require significant patient education and close
monitoring to ensure their safe and appropriate use. Although specialty medications were once a very small piece of the
pharmaceutical industry, there has been a significant change over the last 20 years and especially within the last decade.
Specialty medications are now the fastest growing segment of the pharmaceutical industry. Common characteristics
associated with specialty medications are listed in Table 9.1.
Table 9.1 Specialty Medication Characteristics
High Cost Potential for limited or exclusive availability for
distribution
Complex treatment regimen that require ongoing Treat rare diseases
monitoring and patient education
Special handling, storage, or delivery requirements Treat diseases known to have long term or severe side
effects or increased fatality
Biologically derived and available in injection, infusion, or Payers may define what they consider to be a specialty
oral form medication for reimbursement and contracting purposes.
With the increase in specialty medications over the last two decades, a whole new approach to dispensing these
medications emerged called specialty pharmacies. Generally, specialty medications are not available at typical
community pharmacies, so patients must obtain them through specialty pharmacies. There are many reasons for this:
some practical, some financial, and some clinical. Traditional retail pharmacy is not designed to handle these complex,
costly medications. First, the high cost alone, in many cases, would prohibit retail pharmacies from stocking the
medication. Second, the often-busy nature of a retail pharmacy does not align itself well with being able to appropriately
manage and support the needs of patients with complex disease states. For these reasons, the first specialty pharmacies
began in the early 2000s. Specialty pharmacies are typically required, through their contract with health insurance
companies, to meet unique requirements that are not part of their contracts with retail pharmacies. Examples of these
requirements are listing in Table 9.2.
Table 9.2 Examples of Specialty Pharmacy Services
Coordinating care and facilitating the drug access Case management- disease state management
Facilitating mail order delivery logistics Product device training when applicable
Working with health insurance to determine coverage Data management of technical and clinical patient care
and help coordinate any requirements of the insurance services.
company
Investigating patient assistance programs for patients Call center development
without insurance or lack of coverage
Patient Experience
A patient receiving a prescription for a specialty medication should be aware that the process for getting the medication
is going to look different than the normal retail pharmacy experience. However, the system is designed so that the
patient has the best chance of successfully obtaining and using the medication safely and appropriately. The prescription
would first be sent to the specialty pharmacy. The specialty pharmacy then takes responsibility for making sure the
patient gets the medication, understands the risks and benefits, is able to afford it, able to take it appropriately, and will
follow through with any necessary monitoring. The patient may be connected with a case manager that will call them on
a monthly basis to make sure there are no issues with the medications, check to see if they had any required monitoring
, completed, provide any necessary education, and/or answer patient questions. Patient follow-up often happens every
month prior to sending out the next months’ worth of medications in order to minimize waste, ensure safety, and assess
for efficacy. As specialty pharmacies are generally not local to the patient they are caring for, care and coordination are
often provided telephonically. The process is centralized, and the medications are shipped to the patient’s house or, in
some cases, to the facility that will be administering the medications.
Brief Overview of Common Disease States Treated with Specialty
Medications
Common disease states managed by specialty pharmacies include (1) oncology, (2) multiple sclerosis, (3) rheumatoid
arthritis, (4) Crohn’s disease, (5) hepatitis C, and (6) HIV/AIDs. This module with introduce some of the most commonly
used specialty medications that most health care professionals would come into contact with, regardless of their
specialty. Many of these conditions represent an entire specialty in medicine and are very complex. Therefore, for the
purposes of this module, these conditions will be introduced briefly.
Oncology is the study and treatment of cancer. Cancer is a disease process that involves the development and
proliferation of abnormal calls. Cancer cells are marked by both a structural change and a loss of function from the
original healthy cell. They are often characterized by multiplying at a faster than normal rate. As this collection of
abnormal cells, called a tumor, grows it can become life threatening as it deprives normal body cells of the nutrients they
need to function.
Multiple Sclerosis (MS) is an often unpredictable, disabling disease of the central nervous system. It disrupts the flow of
information both within the brain as well as between the brain and the body. Damage to the myelin coating around the
nerve fibers in the CNS causes the nerve signals to be disrupted. This damage ultimately leads to the symptoms of MS
that can vary between individuals with the disease. Some of the more common symptoms include fatigue, difficulty
walking, numbness or tingling, stiffness in the limbs, weakness, vision problems, cognitive changes, pain, depression, and
emotional changes.
Rheumatoid Arthritis (RA) is a chronic autoimmune disorder that causes inflammation and tissue damage in the joints. It
is a very painful and (often) disabling disease. The common symptoms of RA include pain, stiffness, and reduced range of
motion. Generally, the treatment would not start with a specialty medication. The initial medications fall under the class
disease modifying antirheumatic drug (DMARD). However, these are not always effective or in some instances, patients
may no longer respond as they once did. In either case, a patient would be then switched to one of the specialty
medications reviewed below.
Crohn’s Disease is an inflammatory bowel disease caused by inflammation in the digestive tract which can lead to
symptoms such as abdominal pain, severe diarrhea, fatigue, weight loss, and malnutrition. The inflammation can spread
deep into the layers of the affected bowel tissues making the symptoms extremely painful and often hard to control.
Hepatitis C is a viral infection that causes liver inflammation which can sometimes lead to serious liver damage. The virus
is spread through infected blood. There are often no symptoms associated with chronic hepatitis C until the virus
damages the liver significantly enough. Symptoms of hepatitis C then include bleeding and bruising easily, fatigue, poor
appetite, dark urine, swelling, weight loss, and confusion. Interestingly, acute hepatitis C often goes undiagnosed
because of the lack of or self-limiting nature of the symptoms. Additionally, some people that contract hepatitis C never
go on to develop chronic hepatitis C because their body is able to clear the virus on its own. However, those that do
progress to chronic hepatitis C, will likely develop liver failure and need a liver transplant should they live long enough if
the disease is left untreated. Recent treatments have made the possibility of a cure very likely with only three months of
treatment.
HIV/AIDS is a notorious virus with incredible advancements in treatment over the last 30 years. HIV stands for human
immunodeficiency virus and is the causative agent of acquired immunodeficiency syndrome (AIDS). The virus is spread
through sexual contact, perinatally from an infected mother, or by injection into the blood. The typical course of the
infection is characterized by an acute clinical illness that varies in presentation followed by a longer clinical latency.
