Abstract
Context: When feasible, randomized, blinded single-patient (n-of-1) trials are uniquely capable of establishing the best treatment in an individual patient. Despite early enthusiasm, by the turn of the twenty-first century, few academic centers were conducting n-of-1 trials on a regular basis. Methods: The authors reviewed the literature and conducted in-depth telephone interviews with leaders in the n-of-1 trial movement. Findings: N-of-1 trials can improve care by increasing therapeutic precision. However, they have not been widely adopted, in part because physicians do not sufficiently value the reduction in uncertainty they yield weighed against the inconvenience they impose. Limited evidence suggests that patients may be receptive to n-of-1 trials once they understand the benefits. Conclusions: N-of-1 trials offer a unique opportunity to individualize clinical care and enrich clinical research. While ongoing changes in drug discovery, manufacture, and marketing may ultimately spur pharmaceutical makers and health care payers to support n-of-1 trials, at present the most promising resuscitation strategy is stripping n-of-1 trials to their essentials and marketing them directly to patients. In order to optimize statistical inference from these trials, empirical Bayes methods can be used to combine individual patient data with aggregate data from comparable patients.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 533-555 |
| Number of pages | 23 |
| Journal | Milbank Quarterly |
| Volume | 86 |
| Issue number | 4 |
| DOIs | |
| State | Published - Dec 2008 |
Fingerprint
Keywords
- Bayesian methods
- Clinical effectiveness
- N-of-1 trial
- Personalized medicine
- Research policy
- Treatment effects heterogeneity
ASJC Scopus subject areas
- Public Health, Environmental and Occupational Health
- Health Policy
Cite this
What ever happened to N-of-1 trials? Insiders' perspectives and a look to the future. / Kravitz, Richard L; Duan, Naihua; Niedzinski, Edmund J.; Hay, M. Cameron; Subramanian, Saskia K.; Weisner, Thomas S.
In: Milbank Quarterly, Vol. 86, No. 4, 12.2008, p. 533-555.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - What ever happened to N-of-1 trials? Insiders' perspectives and a look to the future
AU - Kravitz, Richard L
AU - Duan, Naihua
AU - Niedzinski, Edmund J.
AU - Hay, M. Cameron
AU - Subramanian, Saskia K.
AU - Weisner, Thomas S.
PY - 2008/12
Y1 - 2008/12
N2 - Context: When feasible, randomized, blinded single-patient (n-of-1) trials are uniquely capable of establishing the best treatment in an individual patient. Despite early enthusiasm, by the turn of the twenty-first century, few academic centers were conducting n-of-1 trials on a regular basis. Methods: The authors reviewed the literature and conducted in-depth telephone interviews with leaders in the n-of-1 trial movement. Findings: N-of-1 trials can improve care by increasing therapeutic precision. However, they have not been widely adopted, in part because physicians do not sufficiently value the reduction in uncertainty they yield weighed against the inconvenience they impose. Limited evidence suggests that patients may be receptive to n-of-1 trials once they understand the benefits. Conclusions: N-of-1 trials offer a unique opportunity to individualize clinical care and enrich clinical research. While ongoing changes in drug discovery, manufacture, and marketing may ultimately spur pharmaceutical makers and health care payers to support n-of-1 trials, at present the most promising resuscitation strategy is stripping n-of-1 trials to their essentials and marketing them directly to patients. In order to optimize statistical inference from these trials, empirical Bayes methods can be used to combine individual patient data with aggregate data from comparable patients.
AB - Context: When feasible, randomized, blinded single-patient (n-of-1) trials are uniquely capable of establishing the best treatment in an individual patient. Despite early enthusiasm, by the turn of the twenty-first century, few academic centers were conducting n-of-1 trials on a regular basis. Methods: The authors reviewed the literature and conducted in-depth telephone interviews with leaders in the n-of-1 trial movement. Findings: N-of-1 trials can improve care by increasing therapeutic precision. However, they have not been widely adopted, in part because physicians do not sufficiently value the reduction in uncertainty they yield weighed against the inconvenience they impose. Limited evidence suggests that patients may be receptive to n-of-1 trials once they understand the benefits. Conclusions: N-of-1 trials offer a unique opportunity to individualize clinical care and enrich clinical research. While ongoing changes in drug discovery, manufacture, and marketing may ultimately spur pharmaceutical makers and health care payers to support n-of-1 trials, at present the most promising resuscitation strategy is stripping n-of-1 trials to their essentials and marketing them directly to patients. In order to optimize statistical inference from these trials, empirical Bayes methods can be used to combine individual patient data with aggregate data from comparable patients.
KW - Bayesian methods
KW - Clinical effectiveness
KW - N-of-1 trial
KW - Personalized medicine
KW - Research policy
KW - Treatment effects heterogeneity
UR - http://www.scopus.com/inward/record.url?scp=56649123410&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=56649123410&partnerID=8YFLogxK
U2 - 10.1111/j.1468-0009.2008.00533.x
DO - 10.1111/j.1468-0009.2008.00533.x
M3 - Article
VL - 86
SP - 533
EP - 555
JO - Milbank Quarterly
JF - Milbank Quarterly
SN - 0887-378X
IS - 4
ER -