Corporate Citizenship, Expanded Access, and Ethical Pre ...€¦ · Babar, 2015). There is an...

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

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


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.


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


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.


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


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


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.”


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.


References

Ades, F., Zardavas, D., Senterre, C., de Azambuja, E., Eniu, A., Popescu, R., . . . Parent, F. (2014). Hurdles and delays in access to

anti-cancer drugs in Europe. Ecancermedicalscience, 8(474-494), 482. doi: 10.3332/ecancer.2014.482

Bate, A., Juniper, J., Lawton, A. M., & Thwaites, R. M. A. (2015). Designing and incorporating a real world data approach to

international drug development and use: What the uk offers. Drug Discovery Today. doi:

http://dx.doi.org/10.1016/j.drudis.2015.12.002

Berger, M. L., Lipset, C., Gutteridge, A., Axelsen, K., Subedi, P., & Madigan, D. (2015). Optimizing the leveraging of real-world data

to improve the development and use of medicines. Value in Health, 18(1), 127-130. doi: 10.1016/j.jval.2014.10.009

Bertoldi, A. D., Helfer, A. P., Camargo, A. L., Tavares, N. U., & Kanavos, P. (2012). Is the brazilian pharmaceutical policy ensuring

population access to essential medicines? Global Health, 8, 6. doi: 10.1186/1744-8603-8-6

Blommestein, H. M., Franken, M. G., & Uyl-de Groot, C. A. (2015). A practical guide for using registry data to inform decisions

about the cost effectiveness of new cancer drugs: Lessons learned from the pharos registry. Pharmacoeconomics, 33(6),

551-560. doi: 10.1007/s40273-015-0260-4

DeMuro, C., Clark, M., Doward, L., Evans, E., Mordin, M., & Gnanasakthy, A. (2013). Assessment of pro label claims granted by the

fda as compared to the ema (2006-2010). Value in Health, 16(8), 1150-1155. doi: 10.1016/j.jval.2013.08.2293

Gammie, T., Lu, C. Y., & Babar, Z. U. (2015). Access to orphan drugs: A comprehensive review of legislations, regulations and

policies in 35 countries. PloS One, 10(10), e0140002. doi: 10.1371/journal.pone.0140002

Gutierrez, L., Patris, J., Hutchings, A., & Cowell, W. (2015). Principles for consistent value assessment and sustainable funding of

orphan drugs in Europe. Orphanet Journal of Rare Diseases, 10(1), 53. doi: 10.1186/s13023-015-0269-y

Hintlian, J. T., & Kelly, R. (2014). A roadmap for risky territory. Pharmaceutical Processing, 29(8), 18-20.

Hohman, R., Shea, M., Kozak, M., Roberts, S., Allen, J., & Sigal, E. (2015). Regulatory decision-making meets the real world. Science

Translational Medicine, 7(313), 313fs346. doi: 10.1126/scitranslmed.aad5233

Hyry, H. I., Manuel, J., Cox, T. M., & Roos, J. C. P. (2015). Compassionate use of orphan drugs. Orphanet Journal of Rare Diseases,

10(1), 1.

Iudicello, A., Alberghini, L., Benini, G., & Mosconi, P. (2016). Expanded access programme: Looking for a common definition. Trials,

17(1), 21. doi: 10.1186/s13063-015-1108-0

Kohler, J. C., Mitsakakis, N., Saadat, F., Byng, D., & Martinez, M. G. (2015). Does pharmaceutical pricing transparency matter?

Examining brazil's public procurement system. Global Health, 11(1), 34. doi: 10.1186/s12992-015-0118-8

Mackey, T. K., & Liang, B. A. (2012). Combating healthcare corruption and fraud with improved global health governance. BMC

International Health and Human Rights, 12. doi: 10.1186/1472-698X-12-23

Matten, D., Crane, A., & Chapple, W. (2003). Behind the mask: Revealing the true face of corporate citizenship. Giving voice in a

culture of silence. from a culture of compliance to a culture of integrity, 45(1/2), 109-120.

Rare diseases: Facts and statistics. (n.d.). Retrieved March 18, 2016, from http://globalgenes.org/rare-diseases-facts-statistics/

Salton, R., & Jones, S. (2015). The corporate social responsibility reports of global pharmaceutical firms. British Journal of

Healthcare Management, 21(1), 21-25 25p.

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


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.


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


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: [email protected]

One World, Inc.

Tucker, GA 30084

Date post:	13-Aug-2020
Category:	Documents
Upload:	others
View:	0 times
Download:	0 times

Corporate Citizenship, Expanded Access, and Ethical Pre ...€¦ · Babar, 2015). There is an...

Documents