FYI - MEM for binary response with one obs/individual

5 messages 3 people Latest: Sep 18, 2001
From: Lewis B Sheiner <lewis@c255.ucsf.edu> Subject: FYI - MEM for binary response with one obs/individual Date: Sat, 15 Sep 2001 10:22:40 -0700 So, perhaps it can be done after all, but Stuart is not sanguine ...\
Quoted reply history
-------- Original Message -------- Date: Sat, 15 Sep 2001 04:21:02 -0700 (PDT) From: stuart@c255b.ucsf.edu To: dennis.fisher@durect.com, lewis@c255b.ucsf.edu >I'm basing my "can't be done" on the idea that the omega is unidentifable: >if you have 1 success, and 1 failure, how can you >know if the success will be a success the next time with p>.5 >(=> omega > 0) or with p=.5 (=> omega = 0)? > > Lewis The highly nonlinear nature of the logistic-type model often allows the inter- and intra-individual variability to be separated. In one example I just ran, I got legitimate estimates 90% of the time. However, as we have learned since the time of the Bailey-Gregg article in 1997, and as we report in our JPP article in last month's issue: When the amount of data per individual is very small, the estimates are not as good as those obtained when interindividual variability is omitted from the model. In the example I just ran, the estimates are indeed quite poor, so poor in fact as to suggest that it would be ill-advised to include interindividual-variability in the model. Stu

1 binary response/person

From: Lewis B. Sheiner Date: September 17, 2001 technical
From: LSheiner <lewis@c255.ucsf.edu> Subject: 1 binary response/person Date: Mon, 17 Sep 2001 11:29:23 -0700 All - There's been an exchange recently about whether one can estimate a hierarchical model given only a single binary response from each individual. I claimed it couldn't be done, by which I meant that OMEGA would be meaningless. Stu Beal, in a personal reply to me (which I shared with nmusers) pointed out that my claim that it couldn't be done was not strictly true, in the sense that NONMEM will not necessarily terminate in rounding errors. But he did not claim that the estimate of the structural parameters (theta's) would be good, and he made no claim whatever about OMEGA. Perhaps there are circumstances in which taking a MEM view of single binary response per person data is helpful, but experience to date indicates that if such circumstances exist, they are hard to find. All my experience to date suggests that estimates of those parameters that are common to both the hierarchical and non-hierarchical formulations are actually more precisely estimated under the latter, and estimates of the parameters unique to the hierarchical formulation, namely OMEGA, are meaningless. LBS. -- _/ _/ _/_/ _/_/_/ _/_/_/ Lewis B Sheiner, MD (lewis@c255.ucsf.edu) _/ _/ _/ _/_ _/_/ Professor: Lab. Med., Bioph. Sci., Med. _/ _/ _/ _/ _/ Box 0626, UCSF, SF, CA, 94143-0626 _/_/ _/_/ _/_/_/ _/ 415-476-1965 (v), 415-476-2796 (fax)

Re: 1 binary response/person

From: Nick Holford Date: September 17, 2001 technical
From: Nick Holford <n.holford@auckland.ac.nz> Subject: Re: 1 binary response/person Date: Tue, 18 Sep 2001 07:19:57 +1200 Lewis, Would you please confirm that your comments about OMEGA being meaningless are restricted to the single binary response per person case? How exactly do you know they are meaningless? Is your assertion that they are meaningless based on theoretical considerations or on the results of simulation? Are your comments about the precision of estimates from hierarchical vs non-hierarchical methods based on (misguided) attempts to estimate OMEGA or in the case where OMEGA is fixed to zero? What is your opinion/experience of the meaningfulness of OMEGA estimates for repeated measures binary responses? I am not familiar with other non-hierarchical methods for logistic regression. Do they exist for repeated measures? The key advantages of using NONMEM for binary and other categorical responses is that one is not restricted to estimating parameters of linear (or linearized) models and one can perform joint estimation of PK parameters with the PK predictions driving the model for the binary response. And of course given a hammer everything looks like a nail. Nick -- Nick Holford, Divn Pharmacology & Clinical Pharmacology University of Auckland, 85 Park Rd, Private Bag 92019, Auckland, New Zealand email:n.holford@auckland.ac.nz tel:+64(9)373-7599x6730 fax:373-7556 http://www.phm.auckland.ac.nz/Staff/NHolford/nholford.htm

Re: 1 binary response/person

From: Lewis B. Sheiner Date: September 18, 2001 technical
From: LSheiner <lewis@c255.ucsf.edu> Subject: Re: 1 binary response/person Date: Mon, 17 Sep 2001 17:08:46 -0700 Nick Holford wrote: > > Lewis, > > Would you please confirm that your comments about OMEGA being meaningless are restricted to the single binary response per person case? How exactly do you know they are meaningless? Is your assertion that they are meaningless based on theoretical considerations or on the results of simulation? I am talking about a single binary response per person. It actually applies more widely. Ikuko Yano, Stu and I have a paper in press in JPP ("The need for mixed effect modeling with population dichotomous data") which deals with the matter more extensively. Despite the title, it also p makes some points about continuous 1-observation/person data. > > Are your comments about the precision of estimates from hierarchical vs non hierarchical methods based on (misguided) attempts to estimate OMEGA or in the case where OMEGA is fixed to zero? When OMEGA is set to zero, and there is 1 binary obs/person, then NONMEM does exactly the same thing as standard logistic regression. > > What is your opinion/experience of the meaningfulness of OMEGA estimates for repeated measures binary responses? > I am not familiar with other non-hierarchical methods for logistic regression. Do they exist for repeated measures? If there are repeated measures, then at least in theory, it makes sense to try to estimate a hierarchical model. The to-appear paper I referred to above indicates that even with several observations per person which truly arise from a MEM, a MEM analysis may still not be better than a NPD analysis (i.e. essentially viewing all of the binary observations as independent). > > The key advantages of using NONMEM for binary and other categorical responses is that one is not restricted to estimating parameters of linear (or linearized) models and one can perform joint estimation of PK parameters with the PK predictions driving the model for the binary response. And of course given a hammer everything looks like a nail. This is a feature of NONMEM and not of ML for binary data. As I said above, if you set OMEGA = 0 then NONMEM does what standard logistic regression would do, and, as you point out, makes it more convenient to implement non-linear models for the logistic model parameters. -- _/ _/ _/_/ _/_/_/ _/_/_/ Lewis B Sheiner, MD (lewis@c255.ucsf.edu) _/ _/ _/ _/_ _/_/ Professor: Lab. Med., Bioph. Sci., Med. _/ _/ _/ _/ _/ Box 0626, UCSF, SF, CA, 94143-0626 _/_/ _/_/ _/_/_/ _/ 415-476-1965 (v), 415-476-2796 (fax)

RE: 1 binary response/person

From: Vladimir Piotrovskij Date: September 18, 2001 technical
From: "Piotrovskij, Vladimir [JanBe]" <VPIOTROV@janbe.jnj.com> Subject: RE: 1 binary response/person Date: Tue, 18 Sep 2001 10:22:05 +0200 Even if OMEGA is not fixed to zero and the random effect is at the level of logit [e.g., LOGIT = INT + SLOPE * LOG(CONC) + ETA(1)] the program converges to a negligible value of OMEGA in case of a single binary observation per individual. If more than one observation are available and they are independent, the following control works fine: $PROB dichotomous response: mutiple obs per individual $DATA nmd.ssc $INPUT ID CONC DV $PRED EC50 = THETA(1)*EXP(ETA(1)) SLOPE = THETA(2)*EXP(ETA(2)) INT = -LOG(EC50) * SLOPE LOGIT = INT + SLOPE * LOG(CONC) A=EXP(LOGIT) P=A/(1+A) IF (DV.EQ.1) THEN Y=P ELSE Y=1-P ENDIF $THETA (0,200,300) (.5,2,15) $OMEGA .1 .1 $EST METHOD=COND LAPLACE LIKE MAX=500 PRINT=10 $COV The number of observations/individual should be substantial. I tested it with 10 obs/individual and 50 individuals, and it was fine. I presume if the number of obs/individual is relatively small, the IIV in SLOPE (which corresponds to Hill coeff) cannot be estimated, although it has not been tested. Best regards, Vladimir ------------------------------------------------------------------------ Vladimir Piotrovsky, Ph.D. Research Fellow Global Clinical Pharmacokinetics and Clinical Pharmacology (ext. 5463) Janssen Research Foundation B-2340 Beerse Belgium Email: vpiotrov@janbe.jnj.com