Authentic voice of patients needed in drug development

Meaningful, early and transparent patient involvement throughout the drug development and approval process is needed to avoid tokenism and prevent raising false hopes about new treatments, according to a panel of experts.

Scotland’s inaugural seminar on patient access to medicines heard that the way decisions on new medicines are taken often fails to fully consider the views of people living with the conditions concerned.

Panel members warned that involving patients too late in the process can leave them feeling exploited and disillusioned, especially when promising treatments then fail to reach the market due to financial or regulatory hurdles.

They also described how hype around early-stage research – often driven by universities and pharmaceutical companies seeking investment or justifying how funds have been spent – can mislead patients into believing that new treatments will be available for them soon.

Dr Jane Haley, Director of Research at MND Scotland, said this has left charities to correct the narrative:

“It arises from a tension between the need for universities to champion the impact of their research. Press departments are extremely keen to champion every little thing as a breakthrough, and that it’s going to have an immediate impact.

“But because the internet is open to everyone, patients can also see all of these attempts to leverage funding … and they actually think it means they’re going to get something in the next few months, or the next couple of years, which isn’t true.

“So the charity has found itself in a position where we have to become the trusted communications person. But of course, it’s really hard to compete [with the national media].” 

Patients as people with rights, not puppets

Dr Haley was speaking at a Patient Access Seminar hosted by AXIS, a firm of reimbursement experts, in partnership with the Ethical Medicine Industry Group (EMIG).

Fellow panel member Kirsty Hoyle, CEO at Metabolic Support UK, called for drug development to be grounded in patient rights, with a focus on education and empowerment to ensure patients can play a genuine and informed role in decision-making.

She told attendees: “I’ve seen many examples where our community are ‘puppeted’ in to meet the hopes of a pharmaceutical company with a particular molecule discovery.

“But that person’s hopes are dashed because the treatment gets pulled because the financial, economic model doesn’t work out, or something like there’s competition.

“We’re very supportive of drug development, but we also want our community to be a more authentic part of this, which means education.

“We want our community to understand the system in which they are part of and not just be responsive to it but be able to have a rights-based model.”

Choice, control and conversations

Many of the organisations represented on the panel said they have forums designed to bring together people with lived experience and the researchers they fund to support early collaboration.

However, they said many researchers and companies still fail to engage with the people living with conditions relevant to their research.

Caroline Donoghue, Chair of the Scottish Cancer Coalition’s Medicine Subgroup, said patients should be fairly compensated for their time and expertise, with their input measured from bench to trial design and beyond:

“Good patient engagement requires budget to deliver it. It can sometimes feel quite tokenistic, if you’re just like, “oh, we’ve got to include a patient, we’ll bring one person along.”

“Are we monitoring how well we’re doing in terms of patient involvement, whether that’s getting their voice in the design, or that’s actual involvement in, the trials and in the pathway? How are we monitoring it and are we measuring it in the right way?”

The Scottish Medicine Consortium (SMC) acts as the medicines gatekeeper in Scotland, advising NHS Scotland on which medicines to fund based on cost and clinical effectiveness.

Two Alzheimer’s drugs were recently not recommended for use in Scotland, due to concern around their effectiveness and cost – decisions Alzheimer Scotland says came as “devasting” blows for people living with dementia, their families and carers.

The charity is now calling for a new approach to dementia drug decision-making, saying the current SMC process doesn’t fully consider the innovative nature of these new treatments nor the scale of need.

Speaking to seminar attendees, Alzheimer Scotland CEO Henry Simmons explained that, while clinicians may be cautious about new medicines, people with conditions like dementia want to be able to weigh up the risks of a new treatment themselves:

“There was so much discussion across the clinical community about resistance to some of the potential of these new drugs around the fact they were risky.

“Most drugs are risky. Our community, people with dementia: they want the right to make that choice, to take that risk, and not be left so far down the discussion point that it becomes meaningless.”

Image: Copyright Anatoli / Adobe Stock