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Research Advocacy - page 4

Important Advocate Issues in Drug Development


All stakeholders in the drug development process are actively involved in balancing risks (side effects) and benefits of each new drug. We will discuss each aspect in the section below.


The benefits of new drugs are measured in several different ways. The final measurement used to determine if a drug is effective is also called an endpoint.

There are different endpoints that can be measures. Each one can be used in different trial designs to measure whether a new drug will benefit patients.


Clinically Meaningful Benefit:
How a patient feels, functions
or survives.


Knowing what endpoint is being evaluated and what it means is important.

Hanging Balance Scales Image  

The choice of outcomes or endpoints typically involves trade-offs or balancing due to conflicting priorities such as speed, completeness, and clinical value.


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  • Overall Survival (OS)
    • The length of time from either the date of diagnosis or the start of treatment for a disease, such as cancer, that patients diagnosed with the disease are still alive. In a clinical trial, measuring the overall survival is one way to see how well a new treatment works.
    • Improvement in overall survival is one measurement everyone can agree is beneficial to patients!
  • Disease-free survival (DFS)
    • The amount of time a participant with localized disease survives without cancer occurring or recurring, usually measured in months.
  • Progression Free Survival (PFS)
    • The length of time during and after the treatment of a disease, such as cancer, that a patient lives with the disease but it does not get worse. In a clinical trial, measuring the progression-free survival is one way to see how well a new treatment works.
    • Whereas survival is always meaningful it is not as clear when looking at the length of time before your cancer gets worse.
      • What length of time is long enough to call a new drug’s effect clinically relevant? Is it 3 weeks or 3 months?
    • What amount of toxicity is acceptable to extend time without disease progression? This is a quality of life issue.
  • Pathological Complete Response (pCR)
    • The absence of any remaining invasive cancer by microscopic examination of the breast tissue sample and lymph nodes (in the case of breast cancer) removed at surgery following the completion of neoadjuvant therapy (therapy given before surgery to remove the tumor).
    • pCR is a new surrogate endpoint. A surrogate endpoint is an indirect measurement of something else that we know is (or can be) clinically relevant like overall survival or progression free survival. Is it a reliable, validated surrogate endpoint of survival or PFS?

The FDA issued a new guideline in 2012 on how pCR can be used in breast cancer.

Do endpoints matter? …………. YES
    • Is the endpoint clinically relevant to patients?
    • Is the endpoint improving the life of the patient?
    • Will the patient live a longer life? Or a better life?


This is where advocates/patients/consumers
can add a lot to the conversation!

Is there a perfect endpoint?
Success Arm Image  

Again this is a balance and is unique to each trial and each drug being studied

  • Survival is definitely the best endpoint for patients.
  • Sometimes other endpoints can be just as important to specific populations:
    • For example in a population with cancer that has not spread, survival is the ‘cure’ for that cancer.
    • For patients with metastatic cancer (cancer that has spread), it is considered incurable, so progression free survival is also a meaningful endpoint and in some cases stable disease is also a good result.
  • It can also depend on the type of drug being evaluated:
    • Chemotherapy and non-chemotherapy may have very different affects on an endpoint.
    • Biologic agents, vaccines and immunotherapies can have different effects and need different measurements of effectiveness.


How do you measure risk in a meaningful way for patients to understand?

Typically risk is measured by the physician and recorded as an adverse event. Adverse events are bad side effects that happen to you after you take a drug.

Risk is measured as Adverse Events (AEs):

Risk Rocks Image  
  • Severity is measured as 1, 2, 3, 4 and 5
  • Adjectives are sometimes used like, manageable, not any more than expected, tolerable.
  • All assessed by the physician.

However, there is a new way to measure risk, which is a measurement of not only the adverse event but also how it affects the patients’ life. Different from adverse event reporting, patient reported outcome measures (PROMs) are reported by the patient.

Some people think this way of reporting events provides information that is more important to patients. For example a drug may cause a rash that the doctor reports as a “2” and he is not concerned about. The patient with that rash may not be in pain but so embarrassed by it that he/she does not go out and their QOL goes way down.

  • Patient Reported Outcome Measures (PROM):
    • Relatively new way of measuring risk of a drug.
    • Patients can self-report some side effects like fatigue, nausea, etc.
    • Patients can better report severity of the adverse event and more importantly how the event affects their everyday life.
  • Advantages of what is measured by using PROM’s:
    • Measuring what matters to the patient
    • The patient experience matters when weighing benefits and risks of certain treatment options.
    • This is a way to indicate clinical benefit that is meaningful to patients themselves.
    • Pharmaceutical companies are now including PROMs during their studies for the FDA.

Understanding Benefits and Risks – Aids in Decision Making

In reviewing what we have learned about benefits and risks, how does a patient use the information to determine if a treatment is right for them? There is an area of research that is looking at ways to help patients and physicians make these decisions, by developing ‘Decision Aids’. But in order to do this, there has to be good and reliable information about the risks and benefits. Therefore, it is important to use the appropriate measurement.

  • BENEFIT: What are we measuring and is it beneficial to patients
  • RISK: What are the risks that are important to patients?

Another important aspect is how much risk will a patient assume to gain how much benefit of a treatment option?

Decision making on the part of the physician and patient is important and very complex.

The best decision is made when everyone is fully informed
of the actual benefits and risks.

Why is it important to involve advocates?

  • Assessing risk/benefit is very subjective and needs to be done consistently and reliably to actually predict benefits and risks that ARE IMPORTANT to patients.
  • Advocate involvement at all levels – from research through clinical trials to approval – ensures the patient perspective is not forgotten or overlooked.



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