Insights on FTD: An externally led patient‐focused drug development meeting
Bibliographic record
Abstract
Abstract Background Understanding the lived experience of those affected by a disorder should be central to the development and evaluation of therapeutics. Disease‐specific Externally Led Patient‐Focused Drug Development (EL‐PFDD) meetings were established by the FDA to hear directly from those living with a condition about experiences, symptoms, treatments, and hopes for new therapies. Such insights are particularly important for frontotemporal degeneration (FTD), a genetically and clinically heterogenous condition that includes behavioral variant FTD (bvFTD), primary progressive aphasias, progressive supranuclear palsy, corticobasal syndrome, and ALS with FTD. Method On March 5, 2021, AFTD hosted an EL‐PFDD for FTD; 382 caregivers, family members, and persons with FTD participated, plus 171 professionals. In preparation, AFTD and the FTD Disorders Registry conducted the FTD Insights Survey, gathering information on clinical and genetic characteristics of respondents; the earliest, current, and most concerning symptoms; and perspectives on treatments and clinical trials. 1,237 persons with FTD, care partners/caregivers, and family members in the US, Canada and UK had completed the survey by October 31, 2020. Result People living with all FTD syndromes described challenges in activities of daily life, including preparing meals, managing finances, driving, and conversing. Symptoms vary within and across disorders, encompassing language, motor function, thinking/decision‐making, interpersonal/emotional functioning, among others. Many people diagnosed have limited awareness of their symptoms (65% bvFTD; 37% other FTD disorders). In genetic FTD, the path to diagnosis can be simpler, yet a lifetime of perceived or known risk can dramatically influence life decisions. The lack of effective, approved therapies – disease‐modifying or symptomatic – was a universal concern. Non‐prescription and off‐label prescription therapies may stabilize symptoms temporarily while prescribed acetylcholinesterase inhibitors can exacerbate symptoms. Many people are willing to participate in clinical research depending on illness stage and burden imposed by trial design. Conclusion The community of those affected by FTD offers insights into this condition as partners in research. Central themes of the EL‐PFDD meeting for FTD will be summarized by AFTD in a Voice of the Patient reported submitted to the FDA. Results of the FTD Insights Survey are available to researchers through the FTD Disorders Registry.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.008 | 0.013 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.001 |
| Bibliometrics | 0.001 | 0.000 |
| Science and technology studies | 0.003 | 0.001 |
| Scholarly communication | 0.002 | 0.002 |
| Open science | 0.001 | 0.008 |
| Research integrity | 0.003 | 0.005 |
| Insufficient payload (model declined to judge) | 0.015 | 0.004 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".