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BLOG: Targeting the ‘Right’ Patient — How Pharmaceutical Companies Can Help Physicians Connect Patients to the Right Treatments

Not every drug is right for every patient, but for some — or the “right” patient — treatment can provide significant clinical and financial benefits. For new therapies, identification of the right patient can be a challenging journey. This journey is best started by first targeting the most appropriate patients while additional evidence is developed for expanding that reach of HCPs through extending their comfort to a wider range of patients. Unfortunately, it is becoming increasingly common for groups to simply view medications as “good” or “bad” for all patients.

The headlines out of the latest Institute of Clinical and Economic Review (ICER) meeting on Biogen’s Aduhelm for Alzheimer’s disease fail to identify who the right patient is: “ICER panel unanimously votes Biogen’s Aduhelm lacks clinical benefit.” ICER made the critical error of applying this treatment to all patients with Alzheimer’s disease rather than focusing on the right patient.

The right patient is, by definition, one who can benefit so much from the treatment that the improved health outcomes exceed the costs. Benefits in terms of clinical improvement include quality of life, life expectancy, productivity and other factors. Financial costs refer to the patient, payer and society as a whole.

Determining the point at which costs exceed the benefits with complex disease treatments is extremely difficult. When patients respond uniquely, the fact is that a complete picture is not available yet from the limited clinical studies used to gain approval. This is why the FDA approved Aduhelm under their accelerated approval pathway, which is used for drugs that treat serious or life-threatening illnesses and provide a meaningful therapeutic advantage over existing treatments. Accelerated approval is based on a drug’s effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients, with a required post-approval trial to verify that the drug provides the expected clinical benefit.

In addition to identifying the right patient, it is critical for pharmaceutical companies to point out the “wrong” patients as well. There are patients for which the costs of a treatment in terms of clinical and financial outcomes exceed the benefits. This point can occur when the right patient has started a therapy but, afterward, changes affect the components of the equation, causing a treatment to be negative. These changes can include the following:

  1. The patient has not met their therapeutic goal.
  2. The patient develops an adverse drug reaction.
  3. The patient will have a procedure, which precludes the continued use of the drug treatment regimen.
  4. The patient is not able to appropriately use or adhere to the prescribed drug treatment regimen.
  5. The patient’s physician establishes new therapeutic goals.
  6. The patient is diagnosed with a new clinical condition that would put them at risk for adverse drug reactions, therapeutic failure or other drug-related problems.
  7. New standards of care or other national specialty society-endorsed scientific information is published that changes the efficacy or safety record of the agents.
  8. The identification of actual or potential drug-drug interactions or the addition of another agent to the regimen is contraindicated with the current treatment.

For older adults, changes frequently occur over time as additional medications increase the risk for drug-drug interactions or establishing new therapeutic goals. For these patients, de-prescribing these medications needs to occur to prevent negative outcomes.

My recently published article, Negative Treatments — Changing Our Words to Drive Deprescribing, in the Journal of the American Geriatric Society further highlights the need for de-prescribing medications that no longer carry a benefit for a specific patient.

The bottom line is that targeting the right patient is critical not only at the start of a treatment but on an ongoing basis as well. Medications need to be evaluated based on the right patient, and this definition needs to be refined over time with the addition of real-world evidence. Pharmaceutical manufacturers and others play critical roles in assuring medications are given the opportunity to prove their benefit in the real world.

Chief Medical Director

Richard has focused his career on improving health outcomes, especially for some of the most vulnerable populations. This has been achieved through several avenues, beginning with his continued active role as a treating internist/geriatrician.…