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Integrity, Independence and Transparency

Recent years have seen increased interactions between patient groups and pharmaceutical companies. Despite this, external stakeholders may view such partnerships with suspicion. Patient groups can be accused of “selling out” when working with pharma, and industry can be accused of “ticking a box” to show that they work with patients. Due to this negative feedback, some groups are reluctant to share details of partnerships, which can lead to further scepticism.

To avoid this negative reaction, pharmaceutical companies and patient groups should aim to be as transparent as possible about their intentions and collaborations and ensure they maintain their organisation’s integrity. The relationship should be collaborative with open communication to protect the independence of each party and prevent either taking over when it comes to setting and achieving end goals.

The Association of the British Pharmaceutical Industry (ABPI) and the Association of Medical Research Charities (AMRC) have each produced useful guidance on how to manage such relationships. It is important to read this guidance before initiating a partnership with industry.

Industry Funding

Many pharmaceutical companies look to support patient organisations through grants and sponsorship programmes. They may have a clear section of their website, under headings such as “Corporate Responsibility” or “Partnering with Patients”, where they outline a formalised application process and the types of projects that they are interested in.

Industry funding usually revolves around improving patient care, patient outcomes, or patient health education and can support a range of projects such as family days for the patient community, condition awareness leaflets, website development or a helpline for patients.

Many charities set an internal limit for the maximum amount of funding they are willing to accept from industry. If a charity receives too much funding from industry, stakeholders may begin to question the organisation’s independence and integrity, particularly if patient groups are advocating for access to drugs which the company markets. Patient groups should aim to source funds from projects with multiple funders in order to maintain independence. EURORDIS, an alliance of rare disease patient organisations in Europe, recommends that a patient group does not accept more than 50% of its income from pharmaceutical companies, and this is a good starting point for your own policy.

Transparency in funding is particularly important for groups advocating for increased access to drugs. If the drugs in question are manufactured by companies who you have received funding or support from, this must not be hidden or obscured to ensure that your group is not seen to be biased towards a particular company.

More information about fundraising can be found in our “How to fundraise successfully” course.

Advising Industry in the Early Stages

When pharmaceutical companies look to move into a new disease area, they often reach out to patients to better understand patient needs and experiences. There is no formal structure for this across the industry; each company differs in how they consult with patients. Consultation could be in the form of a focus group, workshop or individual consultations. As a patient group, it can be good to facilitate this interaction by:

  • Helping to coordinate the process, perhaps by identifying the most appropriate individuals or families to get involved.
  • Following-up with the company to see how the comments have been utilised and whether any further information can be provided.
  • Ensuring that the interests of the wider patient community are respected throughout the process; negotiate on behalf of your patients to help reduce patient burden e.g. time commitments, travelling to meetings.

Supporting drug development

Figure 1. How can patient groups be involved in the drug development process?

The drug development process can be split into pre-clinical, clinical, and approval and launch phases. During pre-clinical development, patient groups can help raise disease awareness and encourage research. During clinical development, patient groups can be involved in optimising a patient-friendly trial design, supporting retention of participants during trials and helping effectively communicate analysis results. Finally, during the approval and launch of the drug, patient groups can help raise awareness and provide information/advise during decision-making processes.

Patient groups can get involved with the drug development process at a number of different stages:

  1. Pre-Clinical Development

Raising awareness of your disease and its impact on patients may encourage research into this condition. In the early drug development stages, collaboration with a wide range of stakeholders can help drive the work forward. Projects during the pre-clinical development might involve reviewing natural history study data and research proposals.

  1. Clinical Development

Clinical trials need enough patients for the results to be meaningful, however, rare disease clinical trials often struggle to recruit and retain an adequate number of patients as rare disease patient populations are typically small. Patient groups can help minimise this issue by providing insights before, during, and after the trial.

a) Trial Design

Clinical trials are often designed around ensuring the trial will be acceptable to regulatory bodies, rather than being designed around the patient. Clinical trials may demand too much from patients or overlook important aspects of the disease, resulting in difficulty recruiting and retaining patients. It is becoming more common, especially for rare diseases, for patient representatives to sit on clinical trial steering committees and to have a clear voice in clinical trial design. Patient groups make sure the patient is considered from the start of trial design by:

  • Reviewing the trial protocol: To ensure that the trial is not too burdensome for participants and that clinically meaningful trial endpoints are included so that the drug is ultimately beneficial for patients.
  • Supporting with recruitment:
    • Patient groups can utilise their networks of known patients and personal relationships to help with recruitment. This can be challenging when rare disease patients are dispersed around the country.
  • Patient groups can be a friendly face that reach out to eligible patients and neutrally address questions about the trial.
    • Patient groups can help to make trials more patient-friendly to boost the number of patients interested in getting involved and reduce recruitment time.
  • Maximising the site set-up, ensuring that:
    • The sites meet any accessibility requirements the patients may have.
    • Trial appointments are at convenient times of the day.
    • Travel required to reach a trial site from the patient’s home is limited.
    • Processes are in place to allow carers or family members to accompany patients if necessary.

b) During the Trial

  • Retention:
    • Patient groups can look after participants in the trial and ensure that their mental health and wellbeing is maintained and supported.
    • Effective communication is essential for ensuring maximum patient retention. Studies have suggested that patients enrolled in clinical trials feel that regular updates influence their participation and that feeling part of a community is the main reason to remain in a clinical trial.
    • Making patients comfortable in the trial will increase the chances of participants on the trial staying until the end, making the trial more clinically meaningful by retaining a larger patient population.
  • Patient information:
    • Patient groups can help make sure that the information for patients is accessible, relevant and understandable.


c) Trial Analysis

  • Sharing results and progress with patients is important, so that they know what to expect going forward.
  • Interpreting the real-world meaning of clinical outcomes for regulators and demonstrating how clinical trial endpoints translate into real benefits for patients.
  • Highlighting barriers patients might face when accessing the drug.


  1. Approval and Launch of the Drug

It is often thought that once a drug has successfully passed through clinical trials the hard work is over; this is not the case. Before a drug can be prescribed by clinicians it needs to:

  1. Gain a marketing authorisation from a regulatory body (in Europe the regulator is the EMA). The marketing authorisation is the license required for a drug to be legally sold to patients.
  2. Be paid for by the healthcare system; this usually means that companies have to prove the drug is effective, safe and good value for money. In England, the National Institute for Health and Care Excellence (NICE) evaluates whether new drugs, including high cost drugs for rare conditions, should be reimbursed by the National Health Service (NHS). In this process, NICE look to see if the drug is cost-effective given its impact on patients’ lives. More information on the cost-effectiveness of a drug can be found in our “What is health economics” and “Health economic modelling” courses.

Patient groups can help during these regulatory processes by:

  • Explaining to decision-makers what it is like to live with the disease and how the drug has improved the lives of patients who took part in clinical trials.
  • Putting together patient evidence reports, which contain surveys from patients about the value of the drug and its impact on their lives.
  • Attending decision committee meetings to make the case for the importance of a treatment being approved.

By working closely with pharmaceutical companies throughout the drug development process, patient groups can ensure that the treatment will have a positive impact on their patients, and therefore can be a strong partner in convincing decision-makers like NICE of the value the drug would have if approved.