Author: David Bennett-Orphan diseases/UK specialist
Working with industry. Messages from the patients

Working with industry. Messages from the patients

Working with industry. Messages from the patients Date:04 February 2018
As a rookie pharmaceutical company marketer many product launches ago my world consisted largely of clinicians and pharmacists who constituted the different ‘stakeholder’ groups to be addressed in the marketing plan. The medical disorders addressed were common.

Fast forward three decades andrare disorders have taken centre stage in the pharmaceutical industry. Two additional interest groups now command their own sections in the company marketing plan: reimbursement authorities and patient advocacy groups. Navigating the reimbursement hurdle can be tortuous but at least the conditions for the dialogue are defined. In contrast, the rules for engagement with patient groups are still evolving. Practice varies between companies. In order to understand the state of play and share best practice, Findacure held a one day workshop in London in autumn 2017 which brought together patient groups with some industry representatives.  The case for industry to listen to the patient voice is underscored by the results of a large survey of senior clinical development professionals conducted by Covance. The majority of respondents see a strong and growing role for the patient voice in clinical development programmes.  

This article highlights some of the important themes that arose during workshop and shares discussion which helps bring clarity to some of the questions that persist in the industry/patient group relationship, notably around the major topics of clinical research and funding. 

Before jumping into details, what is the basis for the interaction between pharmaceutical companies and patient groups? As in most transactions, each has something the other values. Industry has a need to understand in great detail the patient’s journey through the healthcare system and their treatment needs. When the new treatment is licensed for marketing the company can benefit from the testimony of the patient advocacy organisation. The patient group in turn has a need to improve awareness of ‘their’ disease and attract new members who can benefit from the group’s service.  Many patients with rare genetic diseases suffer considerable delay in gaining an accurate diagnosis. Shortening the ‘diagnostic odyssey’ is a vital goal for companies and patient organisations.

Let’s see how these broad interests play out in practice according insights shared during the workshop.

Clinical research and patient recruitment

Consider the options for a patient organisation with relatively few members. The ability to provide the information and advice required by patients and their families is restricted, as is the ability to act as the voice of the patient population. For a company developing a treatment, recruitment of study patients is one of the most urgent priorities. In the case of biotech companies, failure to recruit patients to a pivotal study can threaten the future of the organisation. And for any drug developer, each day’s delay in recruiting the last patient into the regulatory study can cost millions of dollars per day in lost sales.

A well-developed patient group offers an industry partner the possibility to progress the clinical programme at a faster pace and eventually make an effective treatment available. During the workshop some of the specific benefits that patient groups offer drug developers were shared.

* Patient journey. Typically, rare disease patients travel a rocky road from their community and initial consultation with a generalist to eventual definitive diagnosis at a specialist tertiary centre. Understanding the patient journey allows a company to work with all interested parties to intervene and improve the pathway.  Importantly, patients can be identified at a stage suitable for the treatment protocol. An example of an ‘extreme’ protocol was that for XLHED (X-Linked Hypohidrotic Dysplasia), which required infusions with the experimental treatment during the first two weeks of life.

* Clinical study recruitment. An impressive example of the ability of a patient organisation to supplement patient recruitment was presented by Dr Julie Vallortigara, Ataxia UK. Ataxia UK has a patient registry including over 3,500 patients. The patient group can also be a trusted source of comfort for patients eligible for a drug study. Although the accepted wisdom is that patients with a serious rare disorder are particularly motivated to enter studies, patient survey data presented by Rebecca Starkie (Covance) questions this belief. Presenting results from a survey of patient attitudes to the practicalities of taking part in clinical trials (e.g. willingness to travel, time spent on site), Rebecca noted the surprising finding that patients with a rare condition did not differ in their attitude from patients with a common disorder such as diabetes (see Covance study, below). (For full presentation see

Covance surveyed 150 patients with a rare disease and revealed that they share similar concerns about entering studies as patients with common disorders.
For example additional clinic visits  reduces willingness to take part in a study from 96% for one additional visit  to only 45% for three additional visits, with the prospect of a fourth visit reducing willingness to a mere 26%.Additional hours spent on site also reduces willingness by a similar degree. Concern about side effects of treatment is reported by almost half of patients, again a similar level to that reported by patients with a common diagnosis.

* Enabling clinical studies. Other than identifying suitable trial candidates, patient groups can enable the development of a study protocol and the study progression. Dr Julie Vallortigara shared several examples of the benefits Ataxia UK has been able to offer pharmaceutical developers: ‘our organisation can bring significant resources and infrastructure to a therapeutic development programme.  Our established network of global research experts and easily accessible patient populations cuts preclinical and clinical development time to a minimum and so help bring treatments to market faster and at a lower overall cost’. (For full presentation see


The financial relationship between industry and patient organisations was a theme that recurred throughout the workshop, not as a problem, but as a sensitive aspect of the overall partnership which needs managing. Dr Richard White (Oxford PharmaGenesis) stimulated the discussion with the question, ‘how can industry fund patientorganisation activities without it being deemed as exerting undue influence.’ Guidelines are available to industry, such as the Code of Practice (ABPI) in the UK and companies tend to have a standard operating procedure to cover relationships with patient groups (as well as with healthcare professionals).Equally, patient groups have the obligations of the Charity Commission to guide ethical behaviour. Jill Prawer noted that: 'conflict of interest is often a problem more of perception rather than reality, and if funding helps improve the lot of the patients - and independence is assured - then go for it'.(For full presentation see

What can patient groups and companies do to help avoid the charge of inappropriate influence? There is no single answer but a variety of options were discussed.

1. Funding from more than one source.  Where more than one company is active in researching a treatment for a medical disorder each should share the interests of the advocacy group in patient identification and disease awareness. Additionally, it is in the interest of companies, particularly in the case of a sole industry funder, to help a patient group seek additional sources of income. Large charitable organisations including the National Lottery, Jeans for Genes, the Wellcome Trust are examples of potential sources of funding for specific projects.

2. Written agreements. It is in the interests of the charity to have a written agreement with funders to set out the purpose of the grant and clarify the expectations on both sides, notably the independence of the charity in developing the project for which the grant has been awarded.

3. Purpose of funding. For both practical and ethical reasons, funding a patient group’s general running costs is unattractive to a commercial company. The activity for which funding is sought should fit the description of a project and be in line with the purpose of the charity.Advocacy groups are also well advised to think of the time scale for project in relation to the company’s priorities according to the stage of product development or marketing.

Jill Prawer explained the requirements of a grant application in some detail and emphasised the need - particularly for small, volunteer-led charities - to ensure they were able to deliver the project for which the funding was requested.  She commented that funding like this should involve a contract between the two parties.


To avoid the charge of bias I acknowledge the role of diverse professionals beyond patient advocacy organisations in contributing to the patient voice: healthcare professionals, regulators, clinical research organisations and medical communications professionals. Speaking as a communications professional, Dr Richard White noted that 61% of patient information in England is written at too complex a level (if literacy and numeracy are taken into account).

The distinguishing feature of the rare disorders sector within pharmaceuticals is the existence of communities consisting of patients, clinicians, academic scientists and commercial companies. These communities are long-lasting and comprised of highly committed individuals. My personal involvement with one patient interest group stretches back to my early days in the pharmaceutical industry and has persisted long beyond my commercial involvement. In this regard I am far from unique. An objective for pharmaceutical companies then is to find a way to provide continuity given the inherent turnover of staff. If such continuity can be achieved, the meaningful engagement demanded by patient groups is more likely to be met.

About the author

David Bennett is a consultant in the rare diseases sector and is a public representative for a variety of NHS organisations.