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What patients want

Study
Results
Comment

The informed patient is one who is aware of treatment options available to them, in terms of efficacy, harm, cost, and how much treatments may interfere with their daily activities. The informed patient has been a rarity, but is becoming less rare thanks to easier access to information through the Internet (though with misgivings about how reliable Internet information can be). Studies of treatment choices informed patients may make are very rare. One such [1] in knee osteoarthritis makes for interesting reading.

Study

This was a study at Yale in 100 consecutive patients living in the community with osteoarthritis of the knee. They had pain in one or both knees on most days of the month, had neither gout nor rheumatoid arthritis, and had not had a knee replacement.

In face-to-face interviews preference data on treatment choices and utilities were collected using conjoint analysis software that presented questions in a random order. Patients were given information about treatment efficacy, common adverse events, route and frequency of administration, onset of action and risk of ulcer for five treatments: nonselective NSAIDs, cox-2 inhibitors, opioid preparations, glucosamine and/or chondroitin (all oral), and capsaicin cream. The percentage of patients benefiting was set at 50% for all four oral medications, and 25% for topical capsaicin. Paracetamol was not included because most of these patients had already taken it.

Results

The average age of the 100 patients was 70 years, and 80% were women. Half had a current health status of fair or worse, a third had had dyspepsia from nonselective NSAIDs, 22% had a previous ulcer and 5% had been admitted to hospital for gastrointestinal bleeding. Most of them were using or had used nonselective NSAIDs, Cox-2 inhibitors, glucosamine and analgesic creams, but only a third had previously used opioid preparations. They were therefore experienced patients.

Patient values for medication characteristics are shown in Table 1. Utilities relating to cost or availability have been omitted because they are probably distinct for the USA. High utility values were placed on once daily oral dosing, onset of action within a week, a higher level of patients benefiting, and low risk of common adverse events and low risk of ulcer.



Table 1: Utility values for different medication characteristics



Medication characteristics
Mean utility
One pill once a day
41
One pill four times a day
19
Cream three times a day
10
Onset 1-2 hours
50
Onset 1 week
34
Onset 2 weeks
20
Onset 4 weeks
2
100% benefit
56
75% benefit
39
50% benefit
18
25% benefit
1
Well tolerated
70
Nausea, diarrhoea, heartburn
15
Nausea, constipation, dizziness
10
No added risk of ulcer
72
1% added risk of ulcer
49
2% added risk of ulcer
24
8% added risk of ulcer
1


The relative importance of these various characteristics is shown in Table 2. The distribution of these implies that patients considered multiple characteristics, but highest on the list were common adverse events and ulcer risk. Together these rated much higher than chance or timing of benefit, or route of administration.



Table 2: Relative importance of different treatment characteristics



Characteristic
Relative importance (%)
Gastrointestinal ulcer
19
Common adverse events
19
Chance of benefit
15
Time to benefit
14
Route of administration
12


All these things come together when patients are asked for their preference for treatment. Table 3 shows this for an example of where patients pay a monthly co-payment ($10 per month for NSAIDs, cox-2 inhibitors, and opioid preparations; $25 per month for glucosamine or capsaicin). Capsaicin was the most preferred, but none preferred NSAIDs. Decreasing the risk of ulcers made little difference, but increasing the efficacy of nonselective NSAIDs and cox-2 inhibitors from 50% to 75% of patients benefiting made cox-2 inhibitors the first choice.



Table 3: Patient choices for different treatments, assuming a minimum copayment



Option
Base case
Ulcer risk decreased
Efficacy of anti-inflammatory drugs increased
Nonselective NSAIDs
0
0
0
Cox-2 inhibitors
23
24
36
Opioids
25
25
20
Glucosamine/chondroitin
18
18
15
Capsaicin
34
33
29


Much the same results occurred when patients paid the full cost of their medicines. Patients with health status rated by them as fair or worse were less likely to choose capsaicin (26%) than those who felt well or very well (56%).

Comment

There is much to say about how informative this study is. That patients prefer rapid action, good effect, and few adverse events (common or rare) from a once a day medicine is predictable. That adverse events (common or rare) dominate over efficacy is predictable. Bandolier would not have predicted that topical capsaicin would be preferred over other choices, nor that traditional NSAIDs would be chosen by none. Where patient choice is meant to be increasingly important in healthcare, the difference between these results and most guidelines for treatment is stark. For most of those topical capsaicin would not get a look in, and NSAIDs would be preferred over Cox-2 inhibitors on grounds of acquisition costs.

It is worth questioning the assumptions patients were given over treatment scenarios rather than validity of the results. For topical capsaicin, for instance, the scenario may have been over-generous. A recent systematic review showed that there were only 368 patients in three randomised trials in musculoskeletal pain, and topical capsaicin was barely better than placebo [2].

For NSAIDs or Cox-2 inhibitors, the assumption that half patients benefit may be right, but the context is difficult. Even in clinical trials, 30% of patients with either treatment discontinue by about a year (Bandolier Internet review), and it is more with NSAIDs in clinical practice. There is a difference between short and long term benefit.

Missing from the analysis is how choices would be different if topical NSAIDs had been included. We know they work in knee arthritis, at least in short term trials. Longer term trials are coming that add to present evidence that topical and oral NSAIDs have equal efficacy. They are safer by far, but not available in the USA, so were not included. Had they been, choices may well have been even more in favour of topical over oral drugs.

But these are quibbles. What we know is that what patients want may well be different from what we think they want. We could certainly do with more studies like this, and more information about what to do with this information when we have it.

References:

  1. L Fraenkel et al. Treatment options in knee osteoarthritis. The patient's perspective. Archives of Internal Medicine 2004 164: 1299-1304.
  2. L Mason et al. Systematic review of topical capsaicin for the treatment of chronic pain. BMJ 2004 328: 991-994.

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