Post on 13-Aug-2020
transcript
Keith Watts. April 2016
White Paper
Corporate Citizenship,
Expanded Access, and
Ethical Pre-License Drug Sales
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 2
Abstract
High public expectations for corporate social responsibility
(CSR) and accountability have directed pharmaceutical
industry practices to influence positive change locally and
globally. Drug companies often respond to issues affecting
patient well-being following commercialization of new
products. Today, pharmaceutical companies are held to
higher standards as corporate citizens and must now
consider how to implement this role throughout a product’s
life-cycle. Channels that allow access to medicines to treat
serious, life-threatening and debilitating disorders must be
balanced with the interest of all stakeholders. Today, this
balancing act takes place earlier in the product life-cycle,
often before commercialization, at the end, and even in
those instances where market authorization is not sought.
There are various ethical channels available to provide
needed medications to patients with no alternatives. Some
of these channels include Early and Expanded Access
Programs (EAP), Named Patient Programs (NPP),
Compassionate Use (CU), and Pre-License Sales (PLS).
Confusion exists because these terms can have varying
meanings throughout the globe. It is important that these
channels are fully understood by pharmaceutical companies
to uphold CSR, lessen risks, and preserve patient safety.
Clearer definitions of these different channels are needed,
as is understanding and expertise to ethically and
compliantly leverage these pathways to benefit those
around the world with rare, serious, and often life-
threatening disorders. While this will surely be a difficult
task, clarifying understanding of these pathways will
eventually harmonize manufacturers, distributors, and
patients.
White Paper
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 3
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales
which roughly only 5% are treatable with medicines
approved by the US Food and Drug Administration (FDA). A
rare disease, as defined by the Orphan Drugs Act of 1983,
affects no more than 200,000 patients. The US was the first
country to set up legislation that provided for marketing
exclusivity and payment incentives for orphan drugs
(Gammie et al., 2015). In the 1990’s Japan and Australia
followed the US in crafting orphan drug legislation that
provided incentives to develop and market drugs for rare
diseases; the European Union (EU) followed in 2000 with
specific orphan drug legislation (Gammie et al., 2015).
Many countries have set up orphan drug legislation that
includes varying ranges of reimbursement. For example,
the Netherlands orphan drug legislation allows for pre-
license access to orphan drugs and reimburses 100% of the
cost for approved orphan drugs (Gammie et al., 2015). At
the other end of the continuum, India, a country with over
one-billion people, has no orphan drug legislation and
payment often comes from self-funded foundations or
funded out-of-pocket by the patient (Gammie et al., 2015).
Source: Phrma website: New Report Highlights a Decade of Innovation in Rare Diseases. Accessed 4/11/16.
Available at: http://catalyst.phrma.org/new-report-highlights-a-decade-of-innovation-in-rare-diseases
Countries around the world have set up regulatory
mechanisms for ethical access to orphan and non-orphan
drugs at varying stages of the product life-cycle. Early and
expanded access are only two of the many names used to
describe access channels outside the more familiar
traditional supply chain. Providers, patients, and advocacy
groups are leveraging early access channels to ensure
potential interventions for life-threatening and life-altering
disorders reach those patients without viable treatment
alternatives. Legislation exists for innovations to treat rare
disorders that provide incentives making it commercially
feasible for manufacturers to take the extraordinary risks
associated with developing drugs for small patient
populations. Advocacy groups and international
organizations such as the National Organization for Rare
Disorders (NORD) in the United States (US) and the
European Organisation for Rare Diseases (EURODIS), are
influential drivers of orphan drug legislation (Gammie, Lu, &
Babar, 2015). There is an estimated 350 million people
around the world who suffer from roughly 7,000 different
rare diseases ("Rare diseases: Facts and statistics," n.d.), of
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 4
This article discusses CSR as it converges with challenges along various
supply channels that exist at varying stages of a drug’s life-cycle.
Considering all relevant stakeholders is important when
deciding whether addressing global demand for early access
is consistent with a company’s CSR initiatives. The decision
is more complicated and risky during early stages of a
product’s life-cycle. Safety, efficacy, and a myriad of
strategic considerations in the mid to late development and
post-research phases must direct responsible decision
making. Pre-approval and post-approval pre-license phases
in countries with established ethical early access legislation,
if competently managed, may provide the greatest societal
and corporate benefit.
This article discusses CSR as it converges with challenges
along various supply channels that exist at varying stages of
a drug’s life-cycle. These channels have many labels
including EAP, post-approval PLS, NPP, among others.
These channels represent ethical pathways set up by host
countries to address the issues facing those patients
awaiting a drug’s commercial approval or marketing
authorization to treat a life-threatening or life-altering
disorder.
Corporate Social Responsibility
Pharmaceutical companies are becoming aware that
intentional initiatives to support CSR can improve their
competitive advantage. CSR affects many stakeholders
across sociocultural, political, economic, and regulatory
environments. In the past, a medium to large
pharmaceutical company could donate to various charities
and claim to be a good corporate citizen (Matten, Crane, &
Chapple, 2003). Companies are now expected to engage in
programs that affect the health and well-being of patients
as well as influence sustainable positive change in local and
global communities. Company reputation and public
opinion improves when decisions are directed by
transparent determinants used to guide product access
through ethical pathways designed to meet unmet medical
needs. Salton and Jones (2015) argued that pharmaceutical
companies should be held to higher standards of ethical
social behavior. Pharmaceutical and biotech firms share the
common purpose of improving the health and quality of life
for people around the world (Salton & Jones, 2015). It is
critical to balance this purpose, and the extraordinary
possibility to positively affect lives against the possible
negative consequences flowing from misdirected or poorly
considered product access implementation. Ensuring the
security and integrity of the supply chain is critical when
making these access decisions and the process begins with
choosing the right distribution partner for the right stage of a
product’s life-cycle.
EAP exist to provide needed medicines to patients with
special circumstances in countries with established regulatory
mechanisms for access before market authorization. Some
companies set up extensive programs to provide drugs
outside clinical trials if convinced there may be some benefit
to patients and clinicians before seeking market
authorization. There are pharmaceutical firms that have set
up policies denying or ignoring the need for early access to
medications in countries where they do not intend to seek
market authorization. The reason for restrictive policies
include lack of product supply, uncertainty of the supply
channel regulations, and other reasons that could be
addressed by collaborating with the right strategic partner.
These policies often exist even in the presence of host
country regulatory mechanisms allowing access and
reimbursement for needed drugs approved in another
country. Risks of negative public opinion and failure of CSR
are high when a company refuses to engage the proper early
access channel to help patients in need. The risks of negative
perception is higher when there is no financial or regulatory
risk to the company. Licensed, professional, and dedicated
distribution companies exist with the sole business mission to
ethically address early access needs of patients around the
world, commercially or by CU, while securing the supply chain
and protecting stakeholder interests. The small number of
companies dedicated to this business model, such as One
World, Inc. (OWI) can professionally address the common
issues given for withholding product from smaller less
commercially attractive regions around the world.
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 5
Drug supply forecasts for approved drugs eligible for early access are usually
more robust and can accommodate channel demand more easily than
research stage supply. Companies such as OWI can help with estimates of
treatment demand in various regions, and by securing the supply chain from
secondary wholesalers and specialty pharmacies likely selling into those
regions with neither accountability or the manufacturer’s knowledge. Clinical
and market insights from real-life experiences communicated by providers and
key network partners, where allowed by existing regulatory mechanisms, can
be obtained for strategic use to help guide commercialization. Delays in
market authorization also create disadvantages for patients outside the US
that can be greatly reduced by authorized specialty distributors
knowledgeable in EAP regulations. For example, in the EU, once a drug
application is filed, it takes an average of 418 days for the drug to receive EMA
approval (Ades et al., 2014). Patients in the US typically have more rapid
access to new products, possibly because of expedited review processes
available in the states (Ades et al., 2014). Patients with life-threatening and
life-altering disorders are placed at extreme disadvantage and risk when
manufacturers withhold available product from an approved, licensed and
compliant specialty distributor that can ethically access regulated channels
where the drug is awaiting market authorization, but is approved for early
access.
Diversion and drug counterfeiting is an ongoing concern that risks patient
safety and lives (Hintlian & Kelly, 2014). Diversion appears to be more
common as a mechanism to meet the international demand for needed
medications in instances where manufacturers have decided to withhold
product, pre- and post-approval, from territories where no commercialization
is planned. This is often the case, even when ethical distribution partners
specializing in compliance driven supply of unlicensed and pre-license
medications using established regulatory mechanisms offer to assume
financial risk. Drug diversion by secondary wholesalers also serving domestic
markets further complicates the ethical access to needed medicines and that
lead to higher costs and risk patient safety (Hintlian & Kelly, 2014; World
Health Organization, 2014). Unauthorized companies entering the
international distribution chain with poor quality management also create
risks from diversion and counterfeit product (Kohler, Mitsakakis, Saadat, Byng,
& Martinez, 2015; Wilcock & Boys, 2014).
“A small number of
companies, such as One
World, Inc. (OWI), can address
the common issues given for
withholding product from
smaller, less commercially
attractive regions around the
world.“
Risks of negative public opinion and
failure of CSR are high when a company
refuses to engage the proper early access
channel to help patients in need.
Keith Watts
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 6
Unauthorized companies entering the international distribution chain
with poor quality management also create risks from diversion and
counterfeit product.
These risks include negligent environmental controls and
monitoring combined with poorly implemented export-
import strategies that run counter to regulatory
requirements and risk patients safety (Hintlian & Kelly,
2014). These situations exist when manufacturers’ policies
ignore legitimate pathways that can be legitimately
accessed by working with companies such as OWI to reach
patients where commercialization is delayed or not
planned. Providers and patients are forced to source
needed medications outside legitimate supply chain
channels when manufacturers refuse for fail to use ethical
and regulated channels by working with specialty
distributors to supply pre-licensed and unlicensed
medicines. Unauthorized distribution creates an
environment where patients either do not receive the drug,
or must pay very high prices for access to drugs with no
traceability, educational support, or recall procedure
offered by the unauthorized seller. Further risks to patient
safety occur when there are no assurances that
environmental requirements have been met (Hintlian &
Kelly, 2014).
CSR is evaluated today based on the Global Reporting
Initiative (GRI) that is robust, but does not target issues
specifically relevant to the pharmaceutical industry. Of five
major pharmaceutical companies evaluated, Johnson &
Johnson and Roche reached the highest CSR scores
achieving 65% and 64% GRI guideline coverage, respectively
(Salton & Jones, 2015). Sanofi, GlaxoSmithKline achieved
58% and 44% GRI coverage, followed by Pfizer at 28%
(Salton & Jones, 2015). Some companies focus more
heavily on one or two of the 10 GRI measures, thereby,
understating their contribution to society in the broader
measures, as reported in the Salton and Jones (2015) study.
Despite the ratings from GRI, Pfizer is considered a good
corporate citizen and has devoted significant resources to
setting up regulatory compliant early access for needed
medicines before regulatory approval.
Some companies will devote significant resources to engage
in ROW CSR initiatives. Merck created a unique supply
chain model to supply contraceptives in Senegal that is
replicable for other products in territories that could
produce commercial value while addressing human need
(Wright, 2016). Merck’s initiative is a great example of
CSR. Other large pharmaceutical companies focus on
different CSR initiatives with no attention given to product
access in countries where no market authorization is
planned. Patients with life-threatening diseases living in
regions with no existing or expected commercialization
value face significant disadvantage unless manufacturers
partner with companies such as OWI to ensure ethical
access to needed medications.
OWI is among a few companies dedicated to ensuring
access, no matter how small or difficult the challenges and
can help support CSR for companies able but unwilling to
serve these markets. There are clearly supply chain issues
keeping needed drugs from patients in hard to reach or
commercially unattractive markets (Wright, 2016), but
competent distribution partners specializing in EAP and PLS
can support managed access initiatives around the world.
The EU can be challenging for those unfamiliar with early
access where reimbursement policies differ and market
authorization lag times vary among member nations (Ades
et al., 2014; Gammie et al., 2015; Hohman et al., 2015).
Companies often ignore these territories, because no
market authorization is sought or regulatory expertise is
missing to ensure proper distribution. Merck, Pfizer, and
other manufacturers can help expand compliant global
access to needed medications throughout the product life-
cycle in regions with small patient populations and unique
regulatory mechanisms by partnering with dedicated
distributors specializing in these supply channels.
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 7
A product can receive regulatory approval in one country and be
available for CU or PLS prior to market authorization in other countries.
Ethical Access to Needed
Medicines
Defining ethical EAP and trying to apply the definition
uniformly across continents can be complicated. It is
occasionally unclear on one continent across multiple
industry participants. In the US the term EAP and CU
programs can be used interchangeably, while in Europe the
terms do not have the same meaning (Iudicello, Alberghini,
Benini, & Mosconi, 2016). CU typically refers to a process
where a pharmaceutical manufacturer temporarily allows
access to treatment for a patient who has run out of
alternative treatments (Hyry, Manuel, Cox, & Roos, 2015).
A product can receive regulatory approval in one country
and be available for CU or PLS prior to market authorization
in other countries. Some companies offer the drug at no
charge while others seek out payment programs (Hyry et
al., 2015). CU often represents a commitment to CSR that
can build early market presence yielding useful real world
data associated with the expanded access program (Hyry et
al., 2015). Manufacturers worry about allowing CU, mainly
because they want to avoid creating expectations of free
product.
The US and EMA definitions for EAP are similar; however,
the differences are enough to require regulatory
specialization when carrying out EAP at varying stages of
development and approval. Open-label extension, which is
clearly delineated from EAP in the US definition, but is not
so clear within the EMA, can cause confusion (Iudicello et
al., 2016). A pharmaceutical manufacturer may use CU to
carry out specific internal programs designed to provide
some form of early access to an investigational treatment.
CU may also be tied to a patient assistance program, NPP,
or any number of other programs fitting outside the normal
supply chain. These programs are often unfamiliar to
pharmaceutical manufacturers in certain decision making
roles. Fear of the EAP and PLS channels often stems from
differing regulatory constraints and requirements for these
channels compared to those of the commercial supply
chain. The EMA allows companies that make promising
medicines to run CUs to allow early access to their medicine
and to extend use to patients who can benefit from it. The
EU regulatory environment has many nuances that vary
among member nations. These differences merit discussion,
but are beyond this article’s scope. Future articles will
address some of the differences between open-label studies
and EAP in the EU.
The Brazilian Universal Health System (SUS), with guidance
from the Ministry of Health, has a unique system with the
goal of providing needed medicines free-of-charge to close to
75% of its population (Bertoldi, Helfer, Camargo, Tavares, &
Kanavos, 2012). Different meanings for terms that describe
legislated access to unapproved investigational drugs, post-
research pre-approval drugs, and unlicensed medicines
awaiting authorization has persuaded researchers of the
need for clarity through common definitions for proper
access mechanisms (Iudicello et al., 2016). Clearer definitions
that distinguish EAP from clinical trials should reduce
confusion for patients, providers, and companies dealing with
these complex topics. A global definition is a tall order as the
challenge is to coordinate all terms associated with various
EAPs. Clarity will help direct support of access to needed
medications at varying stages of the product life-cycle
(Iudicello et al., 2016).
Pre-License Sales
Pre-license access allows importation of orphan drugs
available in other countries but currently unauthorized in the
host country (Gammie et al., 2015). NPP is often used to
describe pre-licensing access and is often the most common
method for patients accessing orphan drugs in many
countries (Gammie et al., 2015). NPPs can occur through
approved pre-licensed sale (PLS) of a product access granted
to an individual or a group of patients with a serious or life-
threatening disorder in cases where existing treatment
alternatives have been exhausted. Pre-license regulatory
mechanisms for product access typically require a physician
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 8
Companies with dedicated EAP/PLS models can support PLS and CU
channels in instances where a pharmaceutical manufacturer is not
seeking market authorization in specified countries.
responsible for the patient’s care to assess the need and
open a request for a drug, on behalf of the patient (Gammie
et al., 2015). A manufacturer, or cooperation between the
manufacturer and a competent EAP/PLS distribution
partner, will assess whether to honor the request for
access, based on fixed guidelines from the manufacturer. A
regulatory mechanism for pre-license access may grant
ethical access, although, reimbursement by public payers
may not be the rule (Gammie et al., 2015). Turkey is a
unique contrary example of a country without orphan drug
legislation that has a national reimbursement program
allowing importation and access to orphan drugs in a pre-
license setting where these drugs are unavailable or
unauthorized (Gammie et al., 2015).
Companies with dedicated EAP/PLS models, such as OWI,
can support PLS and CU channels in instances where a
pharmaceutical manufacturer is not seeking market
authorization in specified countries. In countries where
demand for unavailable medicines exists, but market
authorization strategies are unattractive for pharmaceutical
companies; an experienced distribution partner can support
ethical access to these products. Supply chain quality
management by these specialty distributors is important to
protect against counterfeiting, diversion, and unauthorized
product sales (Kohler et al., 2015; Wilcock & Boys, 2014).
Specialty distributors must return value to manufacturers in
the form of supply chain security and integrity, RWD (in
regulatory compliance), market insights and provider
education. In certain cases, RWD, market insights, and
patient reported outcomes (PRO) can support label claims
approved by US and European regulatory agencies. The
EMA more commonly allows PRO-based claims than the US
FDA (Wilcock & Boys, 2014). The EMA generally will grant
higher order claims, such as health related quality of life
and physical functioning, from PRO (DeMuro et al., 2013).
The FDA mainly allows symptom related claims from PRO
(DeMuro et al., 2013). The two agencies tend to align on
symptoms where, for example, patient reported pain
symptoms can support label claims (DeMuro et al., 2013).
Knowing what to expect from different regulatory bodies
may help identify key RWD elements when collection of such
data is allowed.
Many companies have a stated policy of withholding early
access in countries that are not considered for market
authorization or commercialization. These countries are
challenging and some companies, manufacturers, and EAP
distributors, are either not equipped or prefer not to devote
resources to handle requests from these difficult regions. In
the US a few specialized companies exist whose sole business
as a licensed export distributor centers on ethically serving
rest-of-world (ROW) pre-license product needs through
existing regulatory mechanisms. Specialists tend to be
exceedingly capable and enthusiastic to serve those
challenging regions, whether there are two or 200 patients.
An ethical pharmaceutical distributor with a strong PLS
model, having a developed niche presence and competence
in securing EAP supply chains in ROW markets, can be a
strong ally of the pharmaceutical manufacturer. It is
important the distribution partner thoroughly understands
the host country regulatory and related issues facing
manufacturers and patients. The needs of patients can be
efficiently served by an experienced specialty PLS distributor
that simultaneously protects the related interests of
pharmaceutical manufacturers.
A PLS business model does not exclude servicing a CU or EAP,
but it is best to separate one service provider from another to
avoid conflicts of interest and supply chain confusion. In
territories where an available reimbursement or payment
mechanism for unlicensed medicine exists, and where market
authorization is not practical, a competent specialty
distributor can help ethically supply needed products. These
specialists can navigate the complexities of serving this
channel efficiently within available regulatory mechanisms,
and without risk to manufacturers’ global plans. A company
with a sound and robust PLS model can support socially
responsible distribution of products to providers and patients
with no viable treatment alternatives. This is done by
assuming financial and reimbursement risks of product
acquisition, provider credentialing, shipment security,
tracking, and confirmation
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 9
with follow-up reporting, where indicated and allowed by a
host country. In some countries, reimbursement of orphan
drugs is based on therapeutic value, impact on clinical
practice and budget impact (Gammie et al., 2015).
PLS channel management for ethical access to needed
medications requires a specialized distribution partner with
strong understanding of this channel. Unauthorized and
unmonitored sales by secondary wholesalers not versed in
the regulatory environment of many different countries risk
safety and security of patients, whether it be one or many
(Mackey & Liang, 2012; Wilcock & Boys, 2014). It is also an
advantage to work with a partner whose model leverages
understanding of the issues facing manufacturers,
providers, payers, and patients.
Manufacturer CSR that supports EAP, CU, and PLS, often
relies on internal departments devoted to connecting
needed treatments to providers of patients who have
exhausted existing therapeutic regimens. Setting up an
internal system requires resources to develop networks and
global regulatory expertise. A cost-effective alternative
tying up internal resources is to engage a company like OWI
to bridge that gap for patients who cannot otherwise access
a product before market authorization or where
commercialization is not planned. An ethical PLS program
established with a trusted distribution partner experienced
in multi-national regulatory requirements of this channel
offers an efficient alternative to in-house management of
this role. Companies engaged in clinical trials and research
phase EAPs should use a separate provider for this supply
channel to clearly separate from their CU and related pre-
approval EAP.
PLS and CU are important access channels for needed
products, but it is not a separate marketing channel. There
is no marketing allowed for a product before market.
Ethical and experienced distribution partners understand
this and have policies in place defining their role to serve an
existing unmet medical need and to avoid marketing a
product approved for pre-licensed sales. Once a product
receives market authorization, the PLS distribution
partner’s job is to help transition to appointed affiliates of
the manufacturer for commercialization.
“PLS channel management for
ethical access to needed
medications requires a
specialized distribution
partner with strong
understanding of this
channel.”
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 10
Payment for orphan and non-orphan drugs in the EU is subject to
individual country policies.
Regulatory Mechanisms
It is important that manufacturers of orphan drugs and
specialty distributors understand the regulatory framework
in place to address product access and reimbursement
around the world. The regulatory mechanisms in place in
the US and EU directing orphan drug marketing
authorization and early access are similar to non-orphan
regulatory mechanisms (Gammie et al., 2015). A similar
regulatory environment exists in non-EU countries (Gammie
et al., 2015). The Committee for Orphan Medicinal
Products (COMP) of the EMA is responsible for decisions to
award an orphan drug label, although marketing
authorization decisions are the responsibility of the
Committee for Medicinal Products for Human Use (CHMP).
Payment for orphan and non-orphan drugs in the EU is
subject to individual country policies. Individual EU
countries are responsible for fixing early access policy
mechanisms pre- and post-market authorization. Individual
EU countries also must set reimbursement mechanisms that
align payers and regulatory mechanisms for early access to
drugs for rare disorders that are sustainable over time
(Gutierrez, Patris, Hutchings, & Cowell, 2015; Hyry et al.,
2015). Many countries require cost effectiveness analysis to
support reimbursement of high cost orphan drugs and the
use of managed entry agreements are becoming more
common for both orphan and non-orphan drug
reimbursement (Gammie et al., 2015). Reimbursement
policies for needed medications vary significantly around the
world.
In Brazil, 25% of the households pay for private insurance
while 75% of the population rely on the Brazilian Universal
Health System even though only roughly 60% of needed
medicines are available when needed (Bertoldi et al., 2012).
Brazil has three channels that allow access to unapproved or
pre-license pharmaceutical and biotech products, two of
which are subject to approval by the National Health
Surveillance Agency (ANVISA). Humanitarian use, which is CU
for importing free product after meeting several conditions, is
a common channel for access to unapproved medicines. The
EAP in Brazil allows a cohort of patients to receive product
without charge, subject to specific conditions and requires
submission of a summary report on all treated patients.
Finally, NPPs in Brazil allow for personal use of an
unapproved product based off a prescription from the
treating doctor - no ANVISA approval is required.
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 11
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Wilcock, A. E., & Boys, K. A. (2014). Reduce product counterfeiting: An integrated approach. Business Horizons, 57(2), 279-288. doi:
http://dx.doi.org/10.1016/j.bushor.2013.12.001
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 12
References
World Health Organization. (2014). Medicines quality. WHO Drug Information, 28(3), 317-323 317p.
Wright, R. (2016). How merck balances the business of corporate social responsibility. Life Science Leader, 8(1), 18-23.
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 13
ANVISA Agência Nacional de Vigilância Sanitária
COMP Committee for Orphan Medicinal Products
CSR Corporate social responsibility
CU Compassionate Use
EAP Early and expanded access program
EMA European Medicines Agency
EU European Union
EURODIS European Organisation for Rare Diseases
FDA Food and Drug Administration
GRI Global Reporting Initiative
NORD National Organization for Rare Disorders
NPP Named patient program
OWI One World, Inc.
PLS Pre-license sales
PRO Patient reported outcomes
ROW Rest-of-world
SUS Brazilian Universal Health System
US United States
Appendix: Table of Abbreviations
Corporate Citizenship, Expanded Access, and Ethical Pre-License Drug Sales 14
Author: Keith Watts
President/CEO KG Pharmaceuticals.
Board Member, One World, Inc.
Adjunct Faculty, Ohio Christian University Health Care
Administration Healthcare Economics and Business Strategy
Lecturer
Corresponding author at: One World, Inc., 4920 North Royal Atlanta
Drive, Tucker, GA 30084. Tel.: 404-371-1745 Ext 312; fax: 404-371-
1774; email: kwatts@one-world-inc.com
One World, Inc.
Tucker, GA 30084