Let AA be a treatment, YY be an outcome with potential outcomes Y(a)Y(a) and UU be an unobserved confounder of AA and Y.Y. When UU is unobserved the treatment is not ignorable given the observed covariates, and the adjustment formula does not identify the causal effect.

Let AA be a treatment and YY be an outcome. An instrument for the effect of AA on YY is a random variable ZZ that satisfies the following assumptions:

  • relevance: ZZ is associated with A,A, so that cov[Z,A]≠0;\mathrm{cov}\left[Z,A\right]\neq 0;
  • exclusion restriction: ZZ affects YY only through A,A, so that the potential outcome Y(a,z)Y(a,z) under treatment aa and instrument zz does not depend on zz and is written Y(a);Y(a);
  • independence or instrument exogeneity: ZZ is independent of the potential outcomes and potential treatments, Z⊥ ⁣ ⁣ ⁣⊥(Y(a),A(z))Z\perp\!\!\!\perp\left(Y(a),A(z)\right) for all a,z,a,z, possibly given covariates.

In the causal graph an instrument is a vertex with an edge into A,A, no other directed path to YY and no open backdoor path to Y.Y. Relevance is testable from the data, whereas the exclusion restriction and independence are not.

Let ZZ be an instrument for the effect of AA on Y.Y. The linear structural model is the structural equation for YY with a constant treatment effect and an error term correlated with AA but uncorrelated with Z.Z.

Y=α+βA+U,cov[Z,U]=0,cov[A,U]≠0Y=\alpha+\beta A+U,\qquad\mathrm{cov}\left[Z,U\right]=0,\qquad\mathrm{cov}\left[A,U\right]\neq 0

Under the linear structural model the treatment effect is identified by the ratio of covariances, since cov[Z,Y]=β cov[Z,A].\mathrm{cov}\left[Z,Y\right]=\beta\,\mathrm{cov}\left[Z,A\right].

β=cov[Z,Y]cov[Z,A]\beta=\frac{\mathrm{cov}\left[Z,Y\right]}{\mathrm{cov}\left[Z,A\right]}

Least squares regression of YY on AA estimates β+cov[A,U]var[A]\beta+\frac{\mathrm{cov}\left[A,U\right]}{\mathrm{var}\left[A\right]} rather than β,\beta, and the difference is the endogeneity bias.

Let ZZ be a binary instrument for the effect of AA on Y.Y. The Wald estimator of the treatment effect is the ratio of the difference in mean outcomes to the difference in mean treatments between the two instrument values.

β^W:=Y‾Z=1−Y‾Z=0A‾Z=1−A‾Z=0\hat{\beta}_{\mathrm{W}}:=\frac{\overline{Y}_{Z=1}-\overline{Y}_{Z=0}}{\overline{A}_{Z=1}-\overline{A}_{Z=0}}

The Wald estimator is the sample analogue of the ratio of covariances for a binary instrument.

Let a‾∈Rn\underline{a}\in\mathbb{R}^n be the vector of treatments, y‾∈Rn\underline{y}\in\mathbb{R}^n be the vector of outcomes, Z∈R n×qZ\in\mathbb{R}^{\,n\times q} be the matrix of q≥1q\geq 1 instruments and exogenous covariates, X∈R n×pX\in\mathbb{R}^{\,n\times p} be the matrix of the treatment and exogenous covariates with p≤q,p\leq q, and PZ:=Z(Z⊺Z)−1Z⊺P_Z:=Z\left(Z^\intercal Z\right)^{-1}Z^\intercal be the projection onto the column space of Z.Z. Two-stage least squares (2SLS) is the method that regresses XX on ZZ in a first stage, then regresses y‾\underline{y} on the fitted values X^=PZX\hat{X}=P_ZX in a second stage.

β‾^2SLS:=(X⊺PZX)−1X⊺PZy‾\hat{\underline{\beta}}_{\mathrm{2SLS}}:=\left(X^\intercal P_ZX\right)^{-1}X^\intercal P_Z\underline{y}

Two-stage least squares has the following properties:

  • with one instrument and one treatment it equals the ratio of covariances, and with a binary instrument it equals the Wald estimator;
  • it is consistent and asymptotically normal under relevance and exogeneity, but biased in finite samples towards the least squares estimator;
  • the standard errors must be computed from the second-stage residuals y‾−Xβ‾^,\underline{y}-X\hat{\underline{\beta}}, not from y‾−X^β‾^;\underline{y}-\hat{X}\hat{\underline{\beta}};
  • the model is just identified if p=qp=q and overidentified if p<q.p<q.

Let ZZ be a binary instrument and AA be a binary treatment with potential treatments A(1),A(0)A(1),A(0) under the two instrument values. The compliance type of a unit is the pair (A(0),A(1)),\left(A(0),A(1)\right), which takes one of four values:

  • always-takers have A(0)=A(1)=1;A(0)=A(1)=1;
  • never-takers have A(0)=A(1)=0;A(0)=A(1)=0;
  • compliers have A(0)=0A(0)=0 and A(1)=1;A(1)=1;
  • defiers have A(0)=1A(0)=1 and A(1)=0.A(1)=0.

The compliance type is not observed, since only one potential treatment is observed for each unit.

Let ZZ be a binary instrument and AA be a binary treatment. Monotonicity is the assumption that the instrument does not decrease the treatment for any unit, so that there are no defiers.

A(1)≥A(0)A(1)\geq A(0)

Let ZZ be a binary instrument and AA be a binary treatment. The intention-to-treat effect is the average effect of the instrument on the outcome, E[Y∣Z=1]−E[Y∣Z=0],\mathbb{E}\left[Y\mid Z=1\right]-\mathbb{E}\left[Y\mid Z=0\right], and the first-stage effect is the average effect of the instrument on the treatment, E[A∣Z=1]−E[A∣Z=0].\mathbb{E}\left[A\mid Z=1\right]-\mathbb{E}\left[A\mid Z=0\right]. Under independence the first-stage effect is the proportion of compliers when monotonicity holds.

Let ZZ be a binary instrument and AA be a binary treatment. The local average treatment effect (LATE) or complier average causal effect is the average treatment effect among the compliers.

τLATE:=E[Y(1)−Y(0)∣A(1)=1,A(0)=0]\tau_{\mathrm{LATE}}:=\mathbb{E}\left[Y(1)-Y(0)\mid A(1)=1,A(0)=0\right]

Let ZZ be a binary instrument satisfying relevance, the exclusion restriction, independence and monotonicity. The LATE theorem states that the local average treatment effect is identified by the ratio of the intention-to-treat effect to the first-stage effect.

τLATE=E[Y∣Z=1]−E[Y∣Z=0]E[A∣Z=1]−E[A∣Z=0]\tau_{\mathrm{LATE}}=\frac{\mathbb{E}\left[Y\mid Z=1\right]-\mathbb{E}\left[Y\mid Z=0\right]}{\mathbb{E}\left[A\mid Z=1\right]-\mathbb{E}\left[A\mid Z=0\right]}

The LATE theorem has the following properties:

  • the Wald estimator is a consistent estimator of the local average treatment effect without assuming a constant treatment effect;
  • the always-takers and never-takers contribute nothing to the numerator, since the instrument does not change their treatment, and monotonicity removes the defiers, whose contribution would enter with the opposite sign;
  • the local average treatment effect is specific to the instrument, since different instruments have different compliers, and it equals the average treatment effect only under additional assumptions such as a constant treatment effect;
  • the distributions of Y(1)Y(1) and Y(0)Y(0) among the compliers are also identified.

Let ZZ be an instrument for the effect of AA on Y.Y. The instrument is weak if and only if its association with the treatment is small relative to the sampling variability of the first stage. The first-stage FF-statistic is the FF-statistic for the hypothesis that the instruments have zero coefficients in the first-stage regression. Weak instruments have the following properties:

  • the two-stage least squares estimator is biased towards the least squares estimator, with bias approximately 1F\frac{1}{F} of the endogeneity bias;
  • the normal approximation to its sampling distribution fails and confidence intervals undercover;
  • a first-stage FF-statistic below about 1010 is the conventional indication of a weak instrument.

Let ZZ be an instrument for the effect of AA on YY under the linear structural model and β0\beta_0 be a hypothesised value. The Anderson–Rubin test of H0:β=β0H_0:\beta=\beta_0 is the test of whether ZZ has zero coefficient in the regression of Y−β0AY-\beta_0A on Z.Z. The Anderson–Rubin test is valid whatever the strength of the instrument, and inverting it gives a confidence set for β\beta that may be unbounded when the instrument is weak.

Let the linear structural model be overidentified with q>pq>p and u‾^\hat{\underline{u}} be the two-stage least squares residuals. The Sargan–Hansen test or JJ-test of overidentifying restrictions is the test of the hypothesis that all instruments are exogenous, with statistic J=n R2J=n\,R^2 from the regression of u‾^\hat{\underline{u}} on Z,Z, which has an approximate χq−p2\chi^2_{q-p} distribution under the null hypothesis. The JJ-test can detect that some instruments are invalid but cannot identify which, and it has no power when all instruments are invalid in the same way.