What makes a compliant Phase III and pre-launch patient advocacy strategy?


What makes a compliant Phase III and pre-launch patient advocacy strategy?

Nick Hicks, MSc1*, Keith Allan, C.Biol, MBSB, BSc Hons2, Christoph Thalheim, Dipl Paed3 and Paul Woods, B.Pharm. M.A4

1Commutateur Advocacy Communications, Paris, France; 2Medical and Pharmaceutical Consultant, Leicester, UK; 3European Multiple Sclerosis Platform, Brussels, Belgium; 4Paul Woods Compliance, Stockport, UK


Background: A key task for the pharmaceutical industry is to understand the compliance implications of engaging with a patient advocacy group (PAG). This presents challenges for the industry to negotiate the ethical and reputational issues that can arise when working with a PAG.

Objective: To gain the views of pharmaceutical industry executives on future compliance challenges when working with PAGs.

Study design: We conducted two surveys among two sets of industry executives: one group focussed on market access roles and the other focussed on non-market access roles.

Results: Transparency was identified as the biggest challenge, followed by project rationale and then by project ownership.

Conclusion: We explore how this can be overcome and make recommendations on how best to work compliantly with PAGs.

Keywords: pharmaceutical industry; patient advisory group; transparency; compliance; ethics; reputation.

Citation: Journal of Market Access & Health Policy 2016, 4: 33177 - http://dx.doi.org/10.3402/jmahp.v4.33177

Copyright: © 2016 Nick Hicks et al. This is an Open Access article distributed under the terms of the Creative Commons Attribution 4.0 International License, allowing third parties to copy and redistribute the material in any medium or format and to remix, transform, and build upon the material for any purpose, even commercially, provided the original work is properly cited and states its license.

Received: 17 August 2016; Revised: 3 November 2016; Accepted: 8 November 2016; Published: 13 December 2016

Competing interests and funding: The authors have not received any funding or benefits from industry or elsewhere to conduct this study.

*Correspondence to: Nick Hicks, Commutateur Advocacy Communications, C/O Destination conseil 16 rue pierre fontaine, FR-75009 Paris, France, Email: nick@commutateuronline.com


The pharmaceutical industry endeavours to understand patient experiences and address unmet patient needs by engaging with patient advocacy groups (PAGs). However, this raises important compliance, ethical and reputational considerations.

Compliance encompasses the endeavours of pharmaceutical companies to conform to relevant legislation and code of practice governing advertising, communications and interactions with customers. These have been described in a recent review (1).

In the United Kingdom, code of practice breaches are governed by the Association of the British Pharmaceutical Industry (2). Additional guidelines usually apply in the member countries of the International Federation of Pharmaceutical Manufacturers and Associations (3), which also issues universal minimum standards wherever international companies operate – even if there is no effective local regulatory framework.

This article reviews two online surveys of pharmaceutical industry executives on the compliance challenges when working with PAGs as part of a market access programme during Phase III. From these results, we present a five-part model for successfully working with a PAG in a sustainable and ethically compliant manner. To the best of our knowledge, this is the first practical discussion to make recommendations on this subject.



Two online surveys were conducted in December 2015 and January 2016. The first group included 35 pharmaceutical executives with varying geographical responsibility (Table 1) involved in non-market access functions (Tables 2 and 3). The second group comprised 18 executives, primarily responsible for market access (Table 2). The aim was to evaluate the groups’ understanding of the compliance considerations (Table 4) and challenges (Table 5) facing a pharmaceutical company when partnering with PAGs over the next 5 years.

Table 1. Geographical responsibility
What’s your geographical responsibility?
  National market
  Regional market
  European market
  Global market
  Established market
  Emerging or developing market

Table 2. Commercial responsibility
Which of the following best describes your function within your organisation?
  Medical affairs
  Policy/public/governmental affairs
  Other (please specify)

Table 3. Main role of respondents
Survey 1 (non-market access roles) N Survey 2 (market access responsibilities) N
Advocacy 6 Advocacy 1
Compliance 5 Compliance 1
Communications 6 Clinical development 1
Market access 2 Health outcomes 1
Medical affairs 11 Market access 13
Public affairs 5 Medical affairs 0
    Public affairs 0
Total 35 Total 18

Table 4. Compliance considerations
Rank the most important considerations for a compliant partnership project between a pharmaceutical company and a patient group
  Transparency (source of funding, degree of involvement of pharmaceutical company)
  Rationale for project (the primary purpose of the project)
  Project implementation (primary contact, countries involved, measurement)
  Project ownership (patient group, pharmaceutical company or shared ownership)
  Involvement of third party (medical society or patient group medical advisory board)
  Involvement of multiple sponsors
  Other (please specify)

Table 5. Compliance challenges
In the coming 5 years, what do you foresee as the single greatest compliance challenge for a pharmaceutical company when working with patient groups?
  Providing patient insight into early drug development
  Ownership of patient registries
  Shared access to research data from partnership projects
  Markets which do not have an industry code of practice
  Single sponsorship of patient groups
  Involvement of patients/patient groups in HTA
  Late involvement of compliance in the strategic planning process
  Correct or appropriate level of funding to patient groups
  Other (please specify)
HTA, health technology assessment.


Transparency was seen as the most important common consideration for a compliant partnership (Table 6), followed by project rationale and then by project ownership (Table 7). The additional areas raised in the survey are listed in Table 8.

Table 6. Most important considerations for a compliant partnership project between a pharmaceutical company and a patient group (pooled results)
Most important consideration (%) N
Transparency 58 28/48
Rationale for project 35 17/48
Project ownership 23 11/48

Table 7. Most important considerations for a compliant partnership project between a pharmaceutical company and a patient group (by job function)
Executives with non-market access roles Number of responses (%) Executives with market access responsibility Number of responses (%)
Transparency 25/35 71 Rationale for project 7/18 39
Rationale for project 10/35 29 Transparency 3/18 17
Project ownership 9/35 26 Project ownership/involvement of third party 2/18 11

Table 8. Additional areas highlighted as important
Primary job responsibility Area identified as important
Medical affairs Effective collaboration and credibility
Publication plan and authorship
Market access Adaptability both ways along the evolution of the project
Advocacy Contribution of parties to project concept, for example, is the opportunity of shaping the project balanced?
Public affairs Involvement of patient organisation from the start
Publication on Internet of the project and progression
Being strategically aligned to the business objectives (not a commercial business plan)
Communications Common goal
Communication towards patients around this project and towards physicians to get their buy-in

Involvement of PAGs in health technology assessment (HTA) was the most significant compliance challenge in the next 5 years, followed by appropriate level of PAG funding and then shared access to research data (Table 9).

Table 9. Greatest compliance challenge for pharmaceutical companies in working with patient groups (pooled results)
Greatest compliance challenge (%) Number of responses
Involvement of PAGs in HTA 27 13/48
Correct level of funding PAG 17 8/48
Shared access to research data 13 6/48
HTA, health technology assessment; PAG, patient advocacy group.

There was a difference between the two groups about the most important compliance challenge (Table 10). The wider group gave almost equal importance to appropriate funding of patient groups and involvement in HTA. Those working primarily in market access identified the involvement of PAGs in HTA as the priority challenge, followed by shared access to research data, with correct funding of PAGs ranked third.

Table 10. Greatest compliance challenge for pharmaceutical companies in working with patient groups (by job function)
Executives with non-market access roles Number of responses (%) Executives with market access responsibility Number of responses (%)
Correct level of funding PAGs 8/35 23 Involvement of PAGs in HTA 5/13 38
Involvement of PAGs in HTA 7/35 20 Shared access to research data 2/13 16
Shared access to research data 4/35 11 Correct level of funding PAGs 1/13 8
HTA, health technology assessment; PAG, patient advocacy group.


Research limitations

The principal limitations were small survey size and the multiple roles of respondents. The questions were developed with senior compliance executives, yet the possibility of response bias from question phrasing remains.


The approaches to compliance fall into two broad types: a legally driven approach and those founded on well-respected codes of practice.

Both laws and codes exist in all developed countries, but the balance between the two can be very different. Compliance and ethics experts should be an integral part of the team developing advocacy strategy. Ideally, executives who interact with PAGs should hold a non-commercial function, such as medical affairs or advocacy.

In disease areas where there are no PAGs, or where few pharmaceutical companies are operating, companies should beware of being a single or dominant sponsor, since this creates questions over independence. The European Federation of Pharmaceutical Industries and Associations (EFPIA) code prohibits this (4).

In emerging markets, PAGs are generally not well developed and some may not have worked with companies. Also, local teams may not be applying the same standards as international companies, and the local culture can be very different from developed countries. Work by Transparency International identifies countries with a high corruption index and can help ethical compliant relationships worldwide (5). International companies should have internal standards that ensure similar compliance and ethics requirements wherever they operate.

Best practice compliance details a company’s engagement with a PAG, ideally over a number of years with an obvious research and development strategy, rather than a sales-motivated relationship, and will also include extensive record-keeping and documentation.

Project rationale

A defined project rationale is essential to create a trust-based PAG partnership. Every interaction with a patient group should have a clearly defined legal contract which sets out why a company is working with a patient group, and what each party will gain from the partnership and in what timescale. This avoids the risk of hidden promotion.

Creating a clear rationale involves understanding the key players and their interactions, and creating an understanding in the company of a PAG’s needs and perspectives. However, agreeing on a rationale means accepting and understanding the long-term nature of patient engagement. This cannot be captured in conventional sales metrics; yet there is equity value in building partnerships, which cannot be measured by traditional means.

Understanding the key influencers’ interactions shape the advocacy strategy. Thorough analysis of each advocacy group’s priorities, policies and positions provides insight into the issues that matter to patients. Advocacy objectives, and therefore a project’s rationale, will be different when a company creates a new partnership, compared with an established institutional presence. For example, the emphasis in a new partnership will be on understanding unmet needs and creating a mutual agenda.

Patient group ownership

As patients are increasingly well-informed, organised and powerful, more and more companies are prioritising patient-centrism. This means integrating patient inputs in each stage of the value chain, from drug development through to commercialisation and lifecycle management. Early engagement during development – in some ways – poses fewer compliance issues, as no drug has been launched. Involvement well before the availability of a drug avoids the perception of a purely commercial interest and shows a commitment to long-term partnering with PAGs.

One of the main reasons for losing trust is companies reaching out to a PAG before there is internal clarity on what a company wants to achieve. Often, these activities are not thought through in sufficient detail in the early stages of the planning process, leading to a mismatch of shared aims.

HTA involvement

The difference between the two groups’ views on the most significant compliance challenge illustrates an important consideration when planning an advocacy strategy; the need to think beyond partnering just for access and instead addressing unmet needs of the PAG (6, 7).

The market access executives saw the PAGs as having mainly an HTA role, whereas sustainable partnering is likely to involve working on common aims which may or may not be related to access. This was also recognised by the other responders. Internally, advocacy must therefore align with other members of the global access team to scope out priorities. How this is done depends on the way the advocacy function is built into the company infrastructure and the corporate compliance culture, and there must be a transparent, legitimate and responsible communication with patient groups, which acknowledge the sources of funding.

PAG funding

This represents an advocacy opportunity to address unmet patient needs and a compliance challenge – especially if there are few companies in the area. Many PAGs rely on pharmaceutical funding, either as unrestricted medical grants or project funding provided for a specific activity, which must be declared by the pharmaceutical company.

Withdrawal of funding can present a PAG with a funding crisis. This is where early transparency, and a clearly defined, detailed, agreed contract, can minimise the risk of reputational damage.


Pharmaceutical company interactions with PAGs must be highly regulated and transparent to avoid the risk of damage to reputation that ensues if companies fail to observe the rules and regulations set out to ensure compliance. The transparency must reinforce the legitimacy of the partnership.

This article provides a useful first step in identifying and planning how to find common points of interest to work together, by focusing on the key compliance challenges now and in the immediate future, and their solutions.

While the results need to be interpreted with caution, they provide useful insights and provoke debate on successful compliant advocacy strategy at the time of Phase III.


This article was based on a lecture given by NH and CT in February 2016 entitled ‘Priorities and challenges: phase III advocacy activities’. The said lecture was given as part of the European Market Access University Diploma (EMAUD) run by the University of Marseille.

Lilian Anekwe assisted in the writing of the manuscript.


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About The Authors

Nick Hicks
Commutateur Advocacy Communications

Keith Allan

United Kingdom

Christoph Thalheim

United Kingdom

Paul Woods

United Kingdom

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