Listening to the myeloma patient voice is difficult – but it still needs to be done

Over the last decade, treatments for the blood cancer myeloma have come on in leaps and bounds – but it remains incurable and unpredictable for individual patients and clinicians.

In Myeloma Awareness Month, we talk to Simon Ridley, director of research at Myeloma UK, about how far we’ve come and how listening to the patient voice can help move the medical community to the next step.

Myeloma: A long-term cancer

Advances in myeloma treatment in the last 10 years have led to a swathe of new treatments, but there’s still a long way to go before it becomes a “condition that people die with, rather than die from”.

To get there, regulators, payers, manufacturers and clinicians need to include patients at every step of the journey, said Simon.

“We are working with NICE, NHS England, clinicians and patients to try to make sure there is a good dialogue, to make sure the right evidence is presented and that the patient voice and patient needs are considered,” he said, adding that understanding unmet need benefited everybody.

He went on: “People who are developing new medications, people who are evaluating, regulating and paying for these drugs all need to understand their real value.

“Going back to basics, if the industry is trying to make products for people with a particular condition, they need to understand that market, and understand the unmet need.”

Incurable and unpredictable

Myeloma is incurable and its relapsing-remitting nature makes it highly variable and unpredictable. Different people respond to different treatments and what worked in one person before won’t necessarily work the next time the disease rears its head.

It currently carries a 10-year survival rate of just over 30%. While this has improved greatly in recent years, we are not at the point of myeloma becoming a chronic, long-term condition.

“There have been a number of new therapies that have shown themselves to be efficacious,” said Simon. “One challenge we have now is knowing how to make the most of the treatments: which is the best combination for each patient at which stage of the disease? That’s really complex.”

Part of the problem was being able to carry out robust testing of different combinations of drugs at different stages of the disease.

Simon explained: “There is growing evidence that we need to use combinations of drugs to hit myeloma cells from different angles at the same time.

“But doing these trials is complicated, not least because the drugs are made by different companies and working together can be difficult. They can’t be seen to be operating as a cartel.”

No single patient voice

The multiple ways in which the cancer affects people means there is “no single, typical patient voice” for industry to tap into, adding another dimension of complexity to the listening process.

Simon commented: “It’s very variable. Some people are diagnosed in their 30s and their experience will be very different from someone who is diagnosed in their 80s.

“Ask someone who is newly diagnosed and someone who has been through this three times already, and they will have very different views.”

Those who do respond well to treatment face a whole slew of quality of life issues, that might not impact a different patient population.

But just because it is difficult doesn’t mean it shouldn’t be done, Simon said, adding that Myeloma UK has been working on ways to better incorporate patient preference into treatment decision pathways.

“For industry, systematically understanding patient needs and experience across the spectrum seems the best starting point.

“Of course, talking to the clinicians is very important, but they also need to listen to patients. You can sometimes find differences between what healthcare professionals think is the most important thing to consider and what patients think it is.

“That’s where an organisation like Myeloma UK can add real value because we have a unique insight into and overview of the patient experience, based on our almost daily contact with patients but also our programme of health services research,” he said.

Precision medicine

A future goal, he said, is in precision medicine. And in cancer there is more optimism in this field than in other therapeutic areas.

“If you can figure out what’s driving the disease you can target this with the right drugs and at the right time. Not only could that improve outcomes, but it means you are not giving people expensive drugs that cause side effects, but don’t help with the problem.

“That’s a grand aim and it is difficult because of the heterogeneity involved in myeloma. The science has to catch up, and we don’t have drugs against all of the pathways driving disease.”

Overall, industry is getting a lot better at producing medicines that work to treat myeloma, and you can’t say that in every other therapeutic area, Simon added.

“We have a range of options and now we need to understand the best use of these. To do that we need to relate that understanding to patient benefit in ways that are meaningful.”

While the huge variation of patient experience makes it difficult to capture unmet need, that’s not to say it shouldn’t be done.

“That will help us move on to look to the next stage when myeloma becomes a manageable, longer-term condition for all patients, with a cure as the ultimate aim,” he said.