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Life sciences consultant Sheela Upadhyaya believes that the rare disease community is being held back by red tape. The partnerships between patient groups and industry that should be creating meaningful benefit for patients are being compromised by bureaucratic processes. She suggests some changes that will help the pharmaceutical industry truly put the patient voice at the centre of its work

By Sheela Upadhyaya, a life sciences consultant specialising in rare disease

Partnerships and collaboration – the heart of healthcare

Partnerships and collaboration should be at the heart of any healthcare system. This is especially important in rare diseases where there is paucity of knowledge and very small patient numbers. These factors, combined with often limited knowledge or data within the clinical and patient communities, make it more difficult to develop new, effective care pathways and treatments that are truly fit for purpose.

For some time now the rare disease stakeholder community has called for more collaboration with patients and patient groups to help identify the most important elements of their conditions that need to be addressed. From clinical trial design, treatment pathway design, service design through to patient educational materials, peer support and beyond, all would benefit from patient-community insights. Additionally, doing this well would support the healthcare ecosystem to be better prepared for approvals in both the regulatory and health technology assessment processes.

The ABPI code: a potentially helpful framework to facilitate partnerships

There is commitment from the pharmaceutical industry and patient communities to work in partnership, but why is it that each party has to overcome a huge number of hurdles for the collaboration to be productive and fruitful for all?

This is where the Association of the British Pharmaceutical Industry (ABPI), the self-regulatory body of the pharmaceutical industry, has developed a code of practice that is a helpful guide to those involved.

“The industry considers that provided the requirements of the Code are met, working with patients and patient organisations can bring significant public health benefits.” – page 6

Four key principles underpin the code: commitment to benefiting patients, integrity, transparency and respect. The code outlines what engagement can take place and how; how to construct it and what roles each stakeholder can play. 

The bureaucratic reality of implementing the framework

However, the reality of implementing this framework can be difficult, arduous and bureaucratic, and at times add more barriers than value. Those that have tried to engage in collaborative interactions will attest to the fact that while the code provides a framework on how collaboration can be undertaken, the local interpretation by overly cautious legal / compliance teams in the industry can often hinder progress, reduce meaningful engagement and in some cases halt the ability to engage at all. 

This generates engagement which can seem meaningless or come across as lip service, or be too complicated to undertake, particularly for the smaller patient groups who have limited resources to deal with the mountain of paperwork involved.

So, what makes implementation so problematic?

Over the course of my career, I have had numerous conversations with patient groups and industry who praise the code and its structure; however, frustration arises in interpretation. Each pharmaceutical company’s legal and compliance teams interpret the code and its application differently, causing inconsistency and confusion, creating lack of clarity and understanding for all parties and resulting in stakeholders not seeing the value or being able to contribute.

One example I have seen is contracts being developed with patient groups that are complex and difficult for them to penetrate (patient groups are often small and do not have capacity or resource to seek legal advice on contracting). Complex contracts can make organisations feel overwhelmed and unable to make decisions or make them seek advice from parties that may not be well placed to support them. Such contracts create uncertainty and questions; organisations may not be confident about what they are signing up for and, more importantly, the consequences that may occur. In these situations, it is natural for patient groups to look at the consequences of contract failures, which can seem daunting and worrying.

Another example is situations where pharmaceutical companies are providing an unrestricted grant to the patient group. In line with the code, this may involve some sort of agreement or contracting, but the development of these contracts—for what may be very simple engagement—can take a very long time, resulting in the patient group being unable to progress or deliver activities that may be time-sensitive. Consequently, this wastes resources, creating no output for anyone. These situations do not add value to patients and their care.

It’s important to acknowledge that there is a need for legal and compliance teams in this situation, to protect the independence of each party involved in the engagement, and generally they do a great job of applying the code. However, in some situations the over-zealous application of the code creates evermore complex documents with clauses and paragraphs that are not clear. 

The need for a more structured and consistent approach

We need to come up with a more structured, consistent approach to implementing the framework. This will create efficiency and transparency while maintaining integrity and ensuring that patients remain at the heart of the objective.

The code in clause 23.2, for example, clarifies what the agreement must contain when giving a donation or grant to a patient organisation:

• description of the donation or grant
• objective of the donation or grant. how it will support healthcare, scientific research or education must also be included
• the names of the organisations/parties involved and their respective roles
• the type of activity and the nature of the company’s contribution
• the time frame
• the amount of funding and/or a description of indirect/non-financial, in-kind donation and the nature of that donation. where possible a full breakdown of costs should be included
• a statement that all parties are fully aware that the donation or grant must be clearly acknowledged and apparent from the start
• the signatories to the agreement
• the date of the agreement

This is clear and simple. However, the actual agreement can be many pages long, use complicated language and talk about consequences and sanctions. This leads to slow development of the agreement and even slower sign-off as signatories spend time trying to understand what is needed and what happens if it fails.

What should we do to address this and ensure patient engagement adds value and is meaningful? I would suggest a few things:

• Develop a collective template with relevant stakeholders that all the industry can apply when engaging patient groups and that is easy to understand and apply. If needed, an appendix or appendices can be added—but these too must be simple and clear to understand.
• Share examples of agreements that patient groups are comfortable using on the ABPI website for industry to adopt.
• Educate legal and compliance teams so they deliver simpler, more penetrable documents.
• Develop examples of good and bad agreements.
• Provide clarity about scenarios where sanctions could be applied, and give examples.
• Where possible, the pharmaceutical industry should consider committing to transparency around these agreements, in order that they can be used as examples of best practice.

The time to face the problem is now

The situation I have described is a global issue and not confined to the UK setting. If we can solve this problem in the UK, we can use the solution as a blueprint for other countries to use worldwide—as we have done on many other issues involving the rare disease community.

I am pleased that some steps have already been taken in the UK.

The ABPI published an updated patient sourcebook in September 2022 “to support pharmaceutical companies, patients and patient organisations work together successfully, with relationships that are in the interests of patients and meet the standards set out in the 2021 ABPI Code of Practice”.

Additionally, the PCPMA, along with the ABPI, is undertaking two workshops in March 2023. The first is with patient organisations, to help build a deeper understanding of the code and explore some of the common challenges faced when working together. The second is with industry, where the day will focus on exploring the code in relation to working with patients, patient organisations and the public—along with sharing insights into some of the common challenges in working with patient groups and patients. 

I recognise that overcoming the challenges is not easy and that commitment is needed from many stakeholders, but we owe it to patients and the public to be able to demonstrate that the engagement we undertake is done well and with meaning. So, these workshops and activities are a really encouraging start. I hear there is more to come on this subject from PMPCA and ABPI, and I look forward to providing you with updates.

About Sheela

Sheela Upadhyaya is a consultant to the life sciences industry. She led the NICE Highly Specialised Technology Programme, the programme to evaluate medicines and technologies for rare and ultra-rare conditions for commissioning in the NHS. She was NICE’s Rare Disease and COVID-19 strategic adviser. She is chair for Together for Rare Diseases (Together4RD), an initiative to support collaboration between European Reference Networks (ERNs) and industry.

Sheela is an adviser to the European Haemophilia Consortium Think Tank, chair for the ISPOR Rare Disease special interest group and a trustee of the My Name’5 Doddie Foundation.

sheelaupadhyaya@gmail.com

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