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Overview of N-of-1 study suite – lessons learned from three aggregated N-of-1 trials.

Overview of N-of-1 study suite – lessons learned from three aggregated N-of-1 trials. Geoff Mitchell*, Jane Nikles, Sue-Ann Carmont, Janet Hardy, Phillip Good, Meera Agar, Katherine Clark, Carol Douglas, Rohan Vora , Hugh Senior, David Currow. Current N-of-1 Trials in PC.

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Overview of N-of-1 study suite – lessons learned from three aggregated N-of-1 trials.

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  1. Overview of N-of-1 study suite – lessons learned from three aggregated N-of-1 trials. Geoff Mitchell*, Jane Nikles, Sue-Ann Carmont, Janet Hardy, Phillip Good, Meera Agar, Katherine Clark, Carol Douglas, RohanVora, Hugh Senior, David Currow

  2. Current N-of-1 Trials in PC • Methylphenidate for fatigue in advCa • Paracetamol for pain in advCa • Pilocarpine for dry mouth in advCa • (also known as single patient trials)

  3. What are N-of-1 Trials • Multiple Cross-over RCTs • Provides evidence on treatment effect for an individual patient • METHOD: multiple paired treatment periods with random treatment order, e.g.,

  4. Benefits of N-of-1 trials • Possible to aggregate a series of n-of-1 trials to get a population estimate. • N-of-1 like a trial where participant is in both arms of a randomised controlled trial • Participant is: • Blinded to which arm is going at any one time • Contributes data to each side of an RCT • “control” and “intervention” participation perfectly matched.

  5. When are n-of-1 trials justified? • Medicine short acting • Immediate treatment effect and short half life • Treatment expensive, important side effects or controversial • Validated measure of effect available • Question is important

  6. Usefulness in palliative care • RCTs very hard to conduct – very expensive • Many fail because sample size is not met • Aggregating N-of-1s trials could reduce sample size dramatically, make the gathering of evidence easier

  7. What Worked Well • MPH exceeded recruitment target • Caresearch Database was an efficient method of data management • PaCCSC network of sites and skilled research staff facilitated: • Trial establishment • Liaison with pharmacy • Recruitment • Protocol Compliance • Data Integrity

  8. What Didn’t Work Well • Lower than anticipated recruitment rate and study withdrawals due to: • Very unwell participants • Competing trials in a limited population • Medications are not commercial, so had to be compounded • Delays due to complex ethics process, multiple ethics applications and SSAs, and legal contract review by lawyers • Some patients did not like the taste of pilocarpine or it produced over-salivation

  9. What we would do differently • We would continue with the Caresearch database and the PaCCSC network • Have more realistic timelines on trials that are difficult to recruit in grant applications • Ensure eligibility criteria is less restrictive if possible • Look at different recruitment strategies including through the community rather than solely hospital based

  10. Results • Paracetamol for pain pilot trial had major difficulty in recruiting as participants had to not take paracetamol, 7 patients recruited, data still to be analysed • Pilocarpine for dry mouth, 22 patients recruited, data still to be analysed • MPH for fatigue, see next slide

  11. Methyphenidatevs placebo on fatigue (FACIT-F) individual participant scores • (Mean difference (95% credible interval)) • Did not improve fatigue on a population level, • 8 of 33 participants clinically important improvement in fatigue, • 1 showed important worsening

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