Time-Limited Trials Near the End of Life

Clinicians, patients, and families frequently face scenarios in which they must make decisions near the end of life about whether to initiate major interventions in circumstances in which the outcome is uncertain. They do not want to prematurely forgo treatments that might help, but they also may not want to risk indefinite exposure to burdensome treatments. The possibility of a time-limited trial (TLT) of treatment may provide a way forward.

A TLT is an agreement between clinicians and a patient/family to use certain medical therapies over a defined period to see if the patient improves or deteriorates according to agreed-on clinical outcomes. If the patient improves, disease-directed therapy continues. If the patient deteriorates, the therapies involved in the trial are withdrawn, and goals frequently shift more purely to palliation. If significant clinical uncertainty remains, another TLT might be renegotiated.

A TLT begins with an assessment of the patient's current clinical status, preferences, and prognosis with or without the treatment in question. In addition to disease-related factors, the patient's cognitive and functional status is generally relevant.¹ If the patient is unable to participate in decision making, surrogates should use “substituted judgment” to keep the patient's voice alive.² The benefits and burdens of initiating or forgoing the intervention, and whether knowledge about its efficacy for the patient can be ascertained within a specified time frame, should be carefully weighed.

Clinical uncertainty impacts these decisions. For some patients, a 5% chance of surviving in any functional state will be enough to go forward, whereas for others any significant prospect of functional deterioration would preclude any trial of treatment. Some patients, as they get sicker, will accept treatments more aggressive than they thought they would when healthy.³ Physicians contribute to uncertainty as well, tending to err systematically in either optimistic and pessimistic directions when presenting poor prognostic information.⁴ Although patients and families generally want honesty with compassion when discussing prognosis, their desire to be told the truth may waver as prognosis gets worse.⁵ Many patients and families prefer their physicians to assist and often make challenging medical decisions on their behalf.⁶ Moreover, some patients are too ill to make these decisions themselves, so the burden of decision making falls on family members who may be hesitant about limiting any treatment.⁷

A strategy for discussing TLTs is outlined in the Box. TLTs have 5 key sequential elements:

Preparation

Select a main medical communicator and key clinicians to involve

Identify key patient and family decision maker(s)

Seek consensus among medical teams about clinical condition and prognosis

Identify clear clinical markers of improvement or deterioration

Beginning of the Family Meeting

Each person should introduce himself or herself, including how he or she relates to the patient

Review purpose of meeting

Solicit family members' views of patient's situation

Reconcile clinicians' understanding with that of the patient or family

Consider a TLT

Propose key components of TLT

Discuss how progress will be measured and communicated

Negotiate time frame for reevaluation

Schedule a follow-up meeting

Follow up at Scheduled Intervals Depending on the TLT

Regularly inform family about progress

If treatment is working, propose next steps

If treatment is not working, next steps might include (1) negotiating a different TLT or (2) proposing a plan for treatment withdrawal

Framing challenging medical decisions as TLTs when patients have a chance of responding to treatment but a greater chance of deteriorating can allow families to become better informed about how physicians are trying to proportionately balance benefits and burdens of medical treatment. This process also helps the medical teams reach consensus so they can speak with a unified voice. TLTs help establish mutual expectations, guidance, and a regular structured dialogue about how the patient is progressing, lessening the chance of conflict among treatment teams and the patient or family. TLTs also may provide a way of finding a middle ground with patients who want “everything” done vs unilaterally trying to limit treatment.⁹

However, TLTs also have limitations. If clinical deterioration is rapidly unfolding, there may not be sufficient time to formally initiate a TLT. Furthermore, in today's fragmented health care environment, the risk of health care professionals disagreeing with one another and thereby undermining the trial is substantial. Some families also disagree among themselves, making adhering to a complex TLT difficult. Managing conflict and strong emotions from family members when the patient is deteriorating clinically is one of the more challenging parts of this process. Successful TLTs ultimately require considerable trust and a meaningful relationship between the health care team and the family, and maintaining such relationships in these circumstances can be challenging.

There is little empirical evidence as to the benefits and risks of TLTs. These “trials” are analogous to the “Plan-Do-Study-Act” method of quality improvement and could be integrated within disease management programs.¹⁰ They also could be incorporated into the care of patients as part of continuous improvement (an alternative term such as “time-limited therapy” might be used to prevent interpretation of their intent as research). The effects of framing these challenging decisions as a TLT vs usual care, however, could be studied in terms of effects on decisions made, clinical outcomes, and effects on patients, families, and health care professionals.⁷ Critical questions remain about how TLTs might appropriately alter treatment practices for patients with life-threatening illness and make variations more patient-centered.