Why great therapies still fail to reach patients - EMJ GOLD

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Why great therapies still fail to reach patients

A broken ladder, with a hand holding the missing piece

A great therapy can clear every scientific hurdle and still fail to reach the patient. Often, the difference is friction: a series of small barriers that can have major consequences

Words by Isabel O’Brien

The alarm goes off at 6am. You need to be at the gym for 6:30. In theory, there is plenty of time. Then there is the small matter of getting out of bed. You have to find your shoes, fill your water bottle, make coffee and locate your keys. Perhaps it is raining. Perhaps, after all that, you decide that missing one session isn’t all that bad.

None of these is a serious obstacle. That is precisely why they matter. Friction is rarely a wall. It is a succession of small impediments between an intention and an action, each easy to dismiss until there are enough of them.

Speaking on the EMJ GOLD podcast, behavioural scientist Richard Shotton, Author, Hacking the Human Mind, describes friction as the accumulation of “seemingly trivial barriers” that can have a “disproportionate effect on behaviour”. The same applies after a medicine has cleared the scientific and regulatory hurdles. Access depends on what happens next, and every hurdle creates another opportunity for failure.

When approval isn’t access

At the broadest level, the question is whether an approved medicine reaches patients in a country at all. The European WAIT Study 2025 found that, on average, a new medicine takes 597 days to travel from European marketing authorisation to a patient, while 49% of those approved between 2021 and 2024 were not available in Europe.

“Not available” seldom means rejected. More often, pricing and reimbursement negotiations simply take time, with weeks quickly becoming months. Some of this is within the industry’s control: where and when a company files is a decision, not a fixed constraint. That said, concept such as “day one” access models can attack the delay directly by allowing patients to begin treatment while price negotiations continue.

As Nathalie Moll, Director General, EFPIA, puts it, ensuring medicines reach patients wherever they live in Europe is “a shared ambition and a shared responsibility”. At country level, access can be lost not because the therapy fails, but because the process around it does.

When the best treatment can’t be reached

Clear the border and the patient can still be no closer. In precision oncology, a medicine can be licensed, funded and on a hospital formulary, yet an eligible patient may never receive it because the drug cannot be prescribed until a biomarker is found. The question is no longer whether the treatment exists, but whether the patient can reach the best, most tolerable option for their disease. 

“The gap can open at several points,” says Dr Michael Zaiac, Head of Oncology Medical Affairs, Europe & Canada, Daiichi Sankyo. Clinicians may be unaware of a targeted treatment and therefore not request the relevant test. Results presented at a major oncology congress may change what is possible, but the clinician treating a particular patient may never see them. 

The IQVIA Institute’s 2026 report, Advancing Precision Oncology, found the biggest testing barriers were usable tissue samples, test cost and reimbursement, turnaround time and patients’ willingness to be tested. A Diaceutics analysis published in JCO Precision Oncology found that, in advanced lung cancer, only around 356 of every 1,000 eligible patients were estimated to benefit, with most losses occurring around testing. As Zaiac puts it, “the reality of testing determines the real-world population that can benefit”. 

That makes pathway design as important as the medicine itself. A 2016 JAMA Internal Medicine study by researchers at the University of Pennsylvania found that changing the electronic health record default lifted generic prescribing from around 75% to 98%. Nobody had to be persuaded: the easier option became automatic. Reflex testing applies the same principle, triggering biomarker testing at diagnosis rather than relying on someone to remember to request it. 

When a potential cure never gets discussed

The stakes rise again with cell and gene therapies. Here the question is whether a patient can reach a treatment that could potentially cure them. These are among the most expensive medicines ever made, so it is natural to assume price is what stops them. It is, but only second. In sickle cell disease, Sarah Hendry, Franchise Head, Hematology, Spherix Global Insights, says the first filter is perceptual: “Physicians are making judgment calls about who is and isn’t a good candidate for gene therapy,” she says, “often excluding the patient from that consideration discussion.” 

An audit of patient charts cited by Hendry found that around 80% were considered good candidates, yet only about 10% had active plans to proceed, with “patient motivation” and “likelihood to comply” among the reasons cited. At the 2026 EBMT Annual Meeting, a survey of 138 US clinicians reported that 86% were likely or very likely to refer an eligible child for gene therapy, yet only 18% routinely discussed specific products during referrals. Willingness in principle does not always become a real conversation. 

Some reluctance is rational. The process is long, costly and available at only a handful of accredited centres, so clinicians must consider whether a patient can realistically complete it. But that judgement can still be made before the patient has a say. Hendry argues the responsibility is shared: developers too must “ensure their therapies are accessed by patients who need them most, not just by those who are deemed to be appropriately motivated”. That makes candidacy one of the most complex frictions of the three. The answer is not simply better referral pathways, but clearer candidacy criteria, better education and, crucially, including patients in their own care decisions.

Making access the easier choice

Follow an innovative therapy from approval to a patient and hurdles appears at every stage. First a border, where reimbursement stretches out. Then a pathway, where a specialised test goes unordered. Then a consulting room, where a candidacy judgement is made before the patient is part of the decision. 

These are all frictions, but often unconscious behaviours behind them are what make each easy to miss. The remedy is the same at every level: name the friction, examine its impact and make the better choice easier. For pharma, the task is not to argue the evidence harder, but to find the friction and remove it.  

It is not unlike making that 6:30 session. Shoes by the door, bottle filled the night before, keys where you can find them and don’t look out of the window. 

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