Focus on the patient, not the pill
The UK’s MHRA has launched a pilot scheme to make patient involvement a more central part of clinical trials and drug development. Natalie Healey speaks to the regulator about the aims behind the new initiative and what it hopes to do with the evidence it generates.
While patient-centred research is an increasingly important concept for the pharmaceutical industry, it is arguably not an intrinsic part of getting a new therapy approved. But this could be set to change.
The Medicines and Healthcare Products Regulatory Agency (MHRA), the UK’s medical regulator, has announced that applications for new medicines must now provide evidence of patient involvement activities undertaken during the drug development process.
The initiative – which is being rolled out as a pilot scheme from March 2021 – applies to marketing applications for all new active substances.
Daniel O’Connor, a medical assessor at the MHRA, explains that the agency has made this move to better understand the pharmaceutical industry’s current scope of activities that focus on the patient. “It’s a really great time to ensure that agency is fulfilling its ambition to bring the patient right to the heart of everything we do,” he says.
He reveals that the idea for the pilot sparked from responses to a 12-week public consultation in 2019 on how to engage and involve patients in the agency’s work.
“There was a very clear messaging that patients really need to be embedded across the different functions of the agency,” says O’Connor.
Then in January 2021, the government launched the Innovative Licensing and Access Pathway (ILAP), a scheme aiming to accelerate the time to market for new therapies.
When MHRA discussed ILAP with pharmaceutical companies, it realised that many organisations were already prioritising patient engagement activities. But that activity had never been recorded by the regulator before.
Value in the patient voice
“We’ve always had a sense that companies are doing this,” says MHRA medical assessor Lisa Campbell. “But we’ve never had a systematic, robust way of acknowledging it, learning from it, and taking it forward in a meaningful way.”
Campbell believes companies should be thinking about the patient from the very first stage of drug discovery. “Otherwise after that long ten-year path of drug development, you may have amazing results, but it could mean nothing to the people you are trying to help,” she says.
Three arguments support patient involvement in clinical research, according to a paper from Oxford University researchers in Health Expectations.
The first is that patients have a right to have an input into research on their condition – and that researchers have a moral duty to reduce the power imbalance between scientists and patients.
We’ve always had a sense that companies are doing this, but we’ve never had a systematic, robust way of acknowledging it.
Another is that an individual’s lived experience of their condition can help scientists create more relevant studies and improve trial recruitment. Thirdly, patient involvement increases accountability and transparency of medical research.
But it can be challenging for pharmaceutical companies to know what types of patient involvement activities will be most effective. Sponsors could look to the Innovative Medicines Initiative (IMI), suggests O’Connor – a public-private scheme launched to speed up the development of effective and safe medicines.
IMI’s Paradigm project was set up in 2018 to create a framework for “structured, effective, meaningful and ethical patient engagement” and to demonstrate the “return on engagement” for all stakeholders.
It focuses on patient involvement at three points across the research and development process for a new therapy: research and priority setting, clinical trial design, and early communication with regulators. “It’s created a suite of tools for supporting developers to embed engagement into the clinical development programme,” says O’Connor.
Promoting people power
Engagement activities can help companies better understand how patients view the risks and benefits of a drug. O’Connor recalls a recent consultation on whether tamoxifen should be used for breast cancer prevention in women who have the highest risk of developing the disease.
“Clearly the benefits and risks of taking the medicine were different to these women so we held a dedicated patient group meeting to try and get a better feel for what patients really want from patient information,” he says. “This is understanding you can only really get by talking to the end user.”
Listening to patients early in the development process can also help companies design the most appropriate clinical trials, says Campbell. You can have a study design that looks brilliant on paper, but if the end point isn’t relevant to the patient, your trial could be for nothing. She strongly encourages patient representatives to attend company meetings. O’Connor agrees.
“A couple of weeks ago, a company brought a patient with them to the scientific advisory meeting, and it was an incredibly valuable experience,” he says. “I could ask the patient directly about the endpoint that the company was proposing.”
During the pilot, pharmaceutical companies will voluntarily provide information about their patient engagement activities. However, anything they share about patient-centricity won’t affect the outcome of their application at this stage.
Campbell says clinical trial applicants should summarise their patient engagement activities in the protocol or cover letter.
If the end point isn’t relevant to the patient, your trial could be for nothing.
For drug licencing applications, the regulator will also ask companies about the impact of their patient engagement activities, such as whether it influenced the design of a clinical trial.
The scheme forms part of the UK’s wider vision for medical research. The government says a key theme is to make access to clinical research as easy as possible for everyone across the country, including rural, diverse and under-served populations.
It has pledged to help sponsors easily access patient groups who can support the development of their studies.
O’Connor hopes that there will be enough evidence from the MHRA pilot by late summer to determine whether the agency should consider patient involvement as part of regulatory decision making in future.
“It’s the first step but it's really important for us to understand the level of work that's already going on in terms of patient engagement,” he says.