Goal Attainment Scaling: delivering patient-centred clinical trials using personalised outcome measures
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The “cutting-edge” approach of Goal Attainment Scaling has the power to give the patient a voice in decision-making and to deliver truly patient-centred clinical trials, says Chere Chapman, co-founder and CEO at Ardea Outcomes, a Canadian clinical research organisation (CRO)
Written by Geoff Case, RARE Revolution Magazine
Ardea Outcomes’ mission to personalise clinical outcome measures began in the mid-1990s, Chere Chapman explains. The company’s co-founder Dr Kenneth Rockwood, a geriatrician, was struggling to understand what the life of his patients was really like and what was most important to them, especially in Alzheimer’s disease, but also in many other areas in geriatrics, such as rehabilitation from falls or hip fractures.
A gerontologist, Professor Paul Stolee, professor emeritus, University of Waterloo, with whom Kenneth had collaborated for many years, introduced him to Goal Attainment Scaling (GAS), a methodology first developed in California in the 1960s for planning care in mental health.
Paul explained how this approach would enable Kenneth to better measure the impact of interventions something which he had himself been studying in the context of specialised geriatrics.
“If you ask people what’s important, and whether or not those things are improving, they will tell you, and goal attainment scaling is a rigorous approach to figuring that out.”
– Chere
Unlike other patient-centred measures and patient-reported outcomes such as structured questionnaires, Goal Attainment Scaling is personalised to the patient—everybody’s goals can be different. This was “novel” in the 1960s, Chere says, but remains a “pioneering” approach to this day, perhaps surprisingly so, she adds, given the growing emphasis on patient centricity.
When Kenneth began to use and study Goal Attainment Scaling, initially applying it within dementia, he became so “enamoured” with it, Chere says, that he set up a company, in 2000, to encourage its use in clinical trials.
Chere says: “A lot of people had used Goal Attainment Scaling before and since, in rehab and in clinic settings, but to use it in clinical development settings was a very exciting and novel approach, which I think was way ahead of its time—no one was really talking about patient centricity in the same way then as they are now.”
The Goal Attainment Scaling process
Goal Attainment Scaling begins with the patient meeting with an expert clinician—a doctor, nurse, social worker, physiotherapist—and working together to set personalised treatment goals, usually three to five. These are “SMART goals” that are Specific, Measurable, Achievable, and Relevant to the person and the intervention, and doable within the Time of the trial.
Each goal has an accompanying scale, which is “very individualised and very clear”, says Chere. The scale goes from −2 to −1 to zero and then to +1 to +2. “Minus one is where the patient is now (the baseline), and zero represents the attainment of the goal. Plus one would be something better than the goal, and plus two better than that, and likewise for minus one and two,” Chere explains.
In each follow-up visit the clinician and patient review the goal scales and rate where the patient is on the scale at that time. . Over time this approach allows the efficacy of the treatment or intervention to be measured because the goals can be combined to give a “summary Goal Attainment Scaling score”.
Change can be measured for the individual, but individuals’ scores can also be combined to measure whether the whole group is improving, enabling the comparison of a treatment arm and a placebo arm, for example, to see whether a treatment or intervention is working.
A great advantage of Goal Attainment Scaling, compared to other approaches, is that the data show not only whether a drug is working, but also how. Investigators can see whether, for example, mobility, cognition or behavioural goals are driving the treatment effect. “This is a huge amount of extra information compared with other approaches,” Chere points out.
Goal Attainment Scaling’s relevance in rare disease
Goal Attainment Scaling has a particular relevance in rare disease, in which outcome measures may not exist or may not work well. “It’s very common across rare disease that the outcome measures that do exist don’t work very well,” Chere says. “In some cases, they have ‘ceiling effects’ and ‘floor effects’, so the kids who have the most severe symptoms don’t get picked up on standardised questionnaires so the progress that they’re making is not measurable.”
In these cases, Goal Attainment Scaling gives a way to measure progress without having to create new outcome measures or adapt existing ones.
“Whatever the case may be, and whether the improvement in the person is big or small, as long as it’s meaningful to this particular patient, then you can use this method.”
– Chere
Typically, many clinical trials exclude people because they want the population in the trial to be as homogeneous as possible. Goal Attainment Scaling, however, allows people with comorbidities or other issues, regardless of age, to be included in a trial. This means that “real world evidence is almost infused into the clinical trial from the outset,” Chere says, giving investigators a much clearer indication of how a treatment will work in real-world conditions.
The theoretical rationale for using Goal Attainment Scaling to measure outcomes in clinical trials for rare diseases has been explored by researchers at The Critical Path Institute and academics from the Netherlands.
You can find out more using these links:
Value Health | Approaches to the Assessment of Clinical Benefit of Treatments for Conditions That Have Heterogeneous Symptoms and Impacts: Potential Applications in Rare Disease
Rare disease case studies
Ardea Outcomes has worked with many rare disease groups over the years. Recent projects include work in Prader-Willi syndrome, with TREND Community, and in the developmental and epileptic encephalopathies (DEEs), with The Inchstone Project.
TREND Community is a community-powered digital analytics company that turns the conversations of rare, chronic and emerging disease communities into actionable insights. Its mission is to close the gaps in the medical and scientific understanding of rare and chronic conditions and spark scientific progress.
Maria and Christopher Picone, the founders of TREND Community, began their rare disease journey when their daughter was diagnosed with the rare genetic disorder Prader-Willi syndrome (PWS), which causes a wide range of physical symptoms, learning difficulties and behavioural challenges. Today, the company works closely with many rare disease communities, including the Prader-Willi syndrome community.
A multitude of clinical trials spanning more than two decades have studied the safety and effectiveness of drug therapies to treat PWS. Anecdotal reports suggest that many of these drugs were effective in ameliorating burden of disease, yet only one has crossed the finish line.
PWS is a highly complex therapeutic area, and existing standardised clinical outcome assessments (COAs) consistently fail to capture meaningful change in this population. Therefore, there was an urgent need to create COAs that work.
We’ve been working with Ardea Outcomes for some time now to develop a customised goal inventory for Prader-Willi syndrome that could be used to facilitate goal attainment scaling in clinical trials. It’s ready now, and we’re looking for a pharmaceutical company to collaborate with us in implementing this tool in their clinical studies to better capture meaningful patient-centered outcomes…
Find out more
You can find out more about TREND Community here: https://trend.community
Ardea Outcomes is a collaborator on The Inchstone Project, run by DEE-P Connections, which is developing outcome measures for severe Developmental and Epileptic Encephalopathies (DEEs). Collaborating with consumers, industry partners, clinicians, and academics, we are adapting measures for individuals with DEEs who have severe impairments and whose progress isn’t captured by standardised assessment tools. Goal Attainment Scaling (GAS) was piloted in SNC2A-related neurodevelopmental disorders as part of this project, to better understand how the tool might be used with this population.
Countless new targeted or gene-based therapies are nearing trials for many rare epilepsy communities, but these could be jeopardised if there are no tools to capture small but significant improvements in populations who routinely fall three to five standard deviations below the mean and are not currently measured. This caregiver-led project, with a diverse team of experienced and driven researchers, felt an urgent need for outcome measures that would measure the smaller improvements our children make over time—the inchstones, not milestones.
The Inchstone Project aims to support trials of disease-modifying therapies (DMTs) for DEEs by providing sufficiently sensitive measurement tools to demonstrate therapeutic efficacy. An initial pilot study with SCN2A-DEE patients revealed the limitations of existing tools, particularly for those with severe impairments. Novel assessments and caregiver surveys guided the development of more sensitive and inclusive measures aiming to support clinical trials and demonstrate the effectiveness of new treatments for DEEs.
Find out more
You can find out more about this work here: Therapeutic Advances in Rare Disease | Patient-advocate-led global coalition adapting fit-for-purpose outcomes measures to assure meaningful inclusion of DEEs in clinical trials
Other useful links
https://www.neurology.org/doi/full/10.1212/WNL.0000000000207958
A rigorous and standardised approach
The US Food and Drug Administration (FDA) is creating a set of four methodological guidance documents for patient-focused drug development (PFDD). These documents will guide stakeholders on collecting and submitting patient experience data and relevant information.
The FDA’s fourth and final guidance (Draft Guidance 4) mentions the potential benefit of using Goal Attainment Scaling and other measures as a personalised endpoint in clinical development”. Chere believes that this “nod” from the FDA, along with the enthusiasm of patient advocacy groups to engage with Goal Attainment Scaling and propose it to sponsors, may be helping to de-risk personalised endpoints for the pharmaceutical community.
However, personalised endpoints are still a novel approach and sponsors may be concerned about how they incorporate them into a trial in a rigorous and standardised way, and they may assume that the process will be cumbersome.
Yet, for the last 24 years, Ardea Outcomes has developed products and services that allow Goal Attainment Scaling to be standardised and streamlined. Chere points to three examples.
First, it has a “really robust” Goal Attainment Scaling clinical rater certification programme that ensures that the same high-quality level of data can be collected by different clinicians in different sites around the world.
Second, its careful use of goal inventories—potential goals such as the symptoms and challenges that patients may have, based on input from patients and clinicians—helps to ensure the quality of Goal Attainment Scaling data and facilitates interpretation at the end.
Third, the time burden of implementing Goal Attainment Scaling has been minimised. There is even software, called GoalNav®, that guides clinicians through the process. “Talking to patients for half an hour might seem like a big burden in a clinical trial,” she says. “As it turns out, the clinicians love it and the patients love it too.”
In its consultancy work Ardea Outcomes advises sponsors on how to develop protocols and study plans and everything else that is needed to implement Goal Attainment Scaling in an efficient and rigorous way.
Protecting the voice of patients
“We are in the business of getting patients heard and making sure that they’re at the centre of clinical research and care. And we think goal attainment scaling is the best method in the world to do that.”
– Chere
Capturing patient experiences is an essential part of the drug development process in the 21st century. But while many sponsors engage with this in the “spirit of true collaboration” with patients and patient communities, sadly, in the “worst-case scenario”, others still treat it as a “box-ticking” exercise, Chere says.
For sponsors who really want to gain high-quality data about the lived experience of patients, she recommends Goal Attainment Scaling over other measures.
“Why do we need these different outcomes?” she asks. “Why don’t we just use the same old ones? Why don’t we use the SF-36 survey and the EQ-5D questionnaire and all the different patient reported outcomes? Because they don’t work. And because they don’t work, we’re missing treatment effects that are there, and populations, especially in the rare disease world, are excluded.”
This compelling argument to take a closer look at Goal Attainment Scaling has an extra resonance as we move ever closer to an era of personalised medicine. As Chere says: “We’re trying to develop tailored approaches to interventions for patients, so why wouldn’t we have tailored measurement approaches?”
Chere believes that with few such measures available, it is time for sponsors to take a closer look at Goal Attainment Scaling, which, as a truly personalised outcome measure, remains “cutting edge” three decades on from Ken Rockwood’s bold step into this space in the mid-1990s.
Find out more
Ardea Outcomes is passionate about getting Goal Attainment Scaling into more clinical trials and is partnering with pharma, patient advocacy groups, academic institutions and other CROs to make that happen.
Chere is keen to hear from any other groups that are interested in developing and using the technique. “Whether they are already doing it well or needing a little more input, we are happy to work with them,” she says.
To find out more about this exciting field, visit Ardea Outcomes’ website. The GAS Center of Excellence showcases the world’s most impactful and relevant Goal Attainment Scaling resources, drawing on the work of researchers from around the world.
You can also subscribe to Ardea Outcomes’ free newsletter to receive the latest scientific updates on Goal Attainment Scaling.
Useful links
YouTube | How does Goal Attainment Scaling work?
