The patient’s view on rare disease trial design – a qualitative study
Abstract Background Clinical trials in rare diseases are more challenging than trials in frequent diseases. Small numbers of eligible trial participants, often complicated by heterogeneity among rare disease patients, hamper the design and conduct of a ‘classical’ Randomized Controlled Trial. Theref...
| Main Authors: | , , , , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
BMC
2019-02-01
|
| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | http://link.springer.com/article/10.1186/s13023-019-1002-z |
| _version_ | 1819208488020082688 |
|---|---|
| author | C. M. W. Gaasterland M. C. Jansen – van der Weide M. J. du Prie – Olthof M. Donk M. M. Kaatee R. Kaczmarek C. Lavery K. Leeson-Beevers N. O’Neill O. Timmis V. van Nederveen E. Vroom J. H. van der Lee |
| author_facet | C. M. W. Gaasterland M. C. Jansen – van der Weide M. J. du Prie – Olthof M. Donk M. M. Kaatee R. Kaczmarek C. Lavery K. Leeson-Beevers N. O’Neill O. Timmis V. van Nederveen E. Vroom J. H. van der Lee |
| author_sort | C. M. W. Gaasterland |
| collection | DOAJ |
| description | Abstract Background Clinical trials in rare diseases are more challenging than trials in frequent diseases. Small numbers of eligible trial participants, often complicated by heterogeneity among rare disease patients, hamper the design and conduct of a ‘classical’ Randomized Controlled Trial. Therefore, novel designs are developed by statisticians. However, it is important to be aware of possible design aspects that may jeopardize the feasibility of trial conduct. If the burden of participation is considered out of proportion by patients or parents, recruitment may fail or participants may drop out before trial completion. In order to maximize the chance of success of trials in small populations, it is important to know which aspects of trial design are considered important by patients. Results We have interviewed all ten members of the Patient Think Tank (PTT) of the ASTERIX project, a European research consortium on methodology for clinical trials in small populations. The PTT members are rare disease patient representatives who have completed extensive training in clinical trial methodology. We have analyzed the interviews qualitatively according to Grounded Theory using a thematic analysis, and we structured the topics in four chronologically ordered themes: 1. Involvement in trial design; 2. Opinions on trial design; 3. Trial participation; 4. Phase after the trial. Our main findings are that the PTT-members recommend that patients are involved in trial design from an early stage on, and have influence on the outcomes and measurement instruments that are chosen in the trial, the length of the study, the choice of participants, and the information that is sent to potential participants. Also, according to the PTT-members, patient groups should consider setting up disease registries, placebo groups should be minimized, and more education on clinical trials is advised. Conclusions Rare disease patient representatives who have been educated about clinical trial methodology think it is important to involve patient representatives in research at an early stage. They can be of advice in trial design in such a way that the ratio of potential benefit and burden of trial participation as well as the chosen outcome measures and in- and exclusion criteria are optimized. |
| first_indexed | 2024-12-23T05:40:11Z |
| format | Article |
| id | doaj.art-29ae3bc6cd6d42798bebde9c20d407d1 |
| institution | Directory Open Access Journal |
| issn | 1750-1172 |
| language | English |
| last_indexed | 2024-12-23T05:40:11Z |
| publishDate | 2019-02-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj.art-29ae3bc6cd6d42798bebde9c20d407d12022-12-21T17:58:12ZengBMCOrphanet Journal of Rare Diseases1750-11722019-02-011411910.1186/s13023-019-1002-zThe patient’s view on rare disease trial design – a qualitative studyC. M. W. Gaasterland0M. C. Jansen – van der Weide1M. J. du Prie – Olthof2M. Donk3M. M. Kaatee4R. Kaczmarek5C. Lavery6K. Leeson-Beevers7N. O’Neill8O. Timmis9V. van Nederveen10E. Vroom11J. H. van der Lee12Pediatric clinical Research Office, Academic Medical Center, University of AmsterdamPediatric clinical Research Office, Academic Medical Center, University of AmsterdamPediatric clinical Research Office, Academic Medical Center, University of AmsterdamDepartment of Cognitive Psychology, Vrije Universiteit AmsterdamPSC Patients EuropeLaboratory of Glycoconjugate Immunochemistry, Hirszfeld Institute of Immunology and Experimental Therapy, Polish Academy of SciencesMPS SocietyAlström Syndrome UKEUPATI FellowAKU SocietyStichting Patiëntenstem.nuDuchenne Parent ProjectPediatric clinical Research Office, Academic Medical Center, University of AmsterdamAbstract Background Clinical trials in rare diseases are more challenging than trials in frequent diseases. Small numbers of eligible trial participants, often complicated by heterogeneity among rare disease patients, hamper the design and conduct of a ‘classical’ Randomized Controlled Trial. Therefore, novel designs are developed by statisticians. However, it is important to be aware of possible design aspects that may jeopardize the feasibility of trial conduct. If the burden of participation is considered out of proportion by patients or parents, recruitment may fail or participants may drop out before trial completion. In order to maximize the chance of success of trials in small populations, it is important to know which aspects of trial design are considered important by patients. Results We have interviewed all ten members of the Patient Think Tank (PTT) of the ASTERIX project, a European research consortium on methodology for clinical trials in small populations. The PTT members are rare disease patient representatives who have completed extensive training in clinical trial methodology. We have analyzed the interviews qualitatively according to Grounded Theory using a thematic analysis, and we structured the topics in four chronologically ordered themes: 1. Involvement in trial design; 2. Opinions on trial design; 3. Trial participation; 4. Phase after the trial. Our main findings are that the PTT-members recommend that patients are involved in trial design from an early stage on, and have influence on the outcomes and measurement instruments that are chosen in the trial, the length of the study, the choice of participants, and the information that is sent to potential participants. Also, according to the PTT-members, patient groups should consider setting up disease registries, placebo groups should be minimized, and more education on clinical trials is advised. Conclusions Rare disease patient representatives who have been educated about clinical trial methodology think it is important to involve patient representatives in research at an early stage. They can be of advice in trial design in such a way that the ratio of potential benefit and burden of trial participation as well as the chosen outcome measures and in- and exclusion criteria are optimized.http://link.springer.com/article/10.1186/s13023-019-1002-zRare diseasesPatient involvementQualitative study |
| spellingShingle | C. M. W. Gaasterland M. C. Jansen – van der Weide M. J. du Prie – Olthof M. Donk M. M. Kaatee R. Kaczmarek C. Lavery K. Leeson-Beevers N. O’Neill O. Timmis V. van Nederveen E. Vroom J. H. van der Lee The patient’s view on rare disease trial design – a qualitative study Orphanet Journal of Rare Diseases Rare diseases Patient involvement Qualitative study |
| title | The patient’s view on rare disease trial design – a qualitative study |
| title_full | The patient’s view on rare disease trial design – a qualitative study |
| title_fullStr | The patient’s view on rare disease trial design – a qualitative study |
| title_full_unstemmed | The patient’s view on rare disease trial design – a qualitative study |
| title_short | The patient’s view on rare disease trial design – a qualitative study |
| title_sort | patient s view on rare disease trial design a qualitative study |
| topic | Rare diseases Patient involvement Qualitative study |
| url | http://link.springer.com/article/10.1186/s13023-019-1002-z |
| work_keys_str_mv | AT cmwgaasterland thepatientsviewonrarediseasetrialdesignaqualitativestudy AT mcjansenvanderweide thepatientsviewonrarediseasetrialdesignaqualitativestudy AT mjduprieolthof thepatientsviewonrarediseasetrialdesignaqualitativestudy AT mdonk thepatientsviewonrarediseasetrialdesignaqualitativestudy AT mmkaatee thepatientsviewonrarediseasetrialdesignaqualitativestudy AT rkaczmarek thepatientsviewonrarediseasetrialdesignaqualitativestudy AT clavery thepatientsviewonrarediseasetrialdesignaqualitativestudy AT kleesonbeevers thepatientsviewonrarediseasetrialdesignaqualitativestudy AT noneill thepatientsviewonrarediseasetrialdesignaqualitativestudy AT otimmis thepatientsviewonrarediseasetrialdesignaqualitativestudy AT vvannederveen thepatientsviewonrarediseasetrialdesignaqualitativestudy AT evroom thepatientsviewonrarediseasetrialdesignaqualitativestudy AT jhvanderlee thepatientsviewonrarediseasetrialdesignaqualitativestudy AT cmwgaasterland patientsviewonrarediseasetrialdesignaqualitativestudy AT mcjansenvanderweide patientsviewonrarediseasetrialdesignaqualitativestudy AT mjduprieolthof patientsviewonrarediseasetrialdesignaqualitativestudy AT mdonk patientsviewonrarediseasetrialdesignaqualitativestudy AT mmkaatee patientsviewonrarediseasetrialdesignaqualitativestudy AT rkaczmarek patientsviewonrarediseasetrialdesignaqualitativestudy AT clavery patientsviewonrarediseasetrialdesignaqualitativestudy AT kleesonbeevers patientsviewonrarediseasetrialdesignaqualitativestudy AT noneill patientsviewonrarediseasetrialdesignaqualitativestudy AT otimmis patientsviewonrarediseasetrialdesignaqualitativestudy AT vvannederveen patientsviewonrarediseasetrialdesignaqualitativestudy AT evroom patientsviewonrarediseasetrialdesignaqualitativestudy AT jhvanderlee patientsviewonrarediseasetrialdesignaqualitativestudy |