Catherine Acquadro, MD
Scientific Advisor, Mapi Research Trust
The celebration of the 40 years of Mapi is a wonderful opportunity to ponder the past and dream about the future.
So, I set out to explore with enthusiasm (and tenacity!) the 51 issues of the QOL/PRO Newsletter (all single-numbered issues, combined ones, and special ones). This revealed quite a few surprises, helped me make many rediscoveries, and, unlike “The Boss” (Bruce Springsteen) and his 57 channels,1 I have greatly enjoyed the program. Above all, I have realized that the goal we originally set for the QOL Newsletter as a means of communications between researchers, industry, and regulators has been achieved with flying colors. When I was hired as a trainee in the spring of 1991 to create the QOL newsletter, I could not imagine how far we would go and how great and exciting the adventure would be!
Over these 23 years, 1062 pages were printed, 604 different articles were published, and 959 authors from 42 different countries have entrusted their words to us.
From 1991 to 2014, we publicized the launch of ten new journals in the PRO field, in particular the ISOQOL journal Quality of Life Research, Health and Quality of Life Outcomes, as well as the Patient – Patient-Centered Outcomes Research; a proof of the vitality of the field. We announced the publication of 122 books, the launch of 19 websites, and 7 databases (among them PROQOLID and PROLabels). We advertised dozens of generic and disease-specific PRO instruments that became legacy measures and the activities of numerous groups and organizations that have influenced PRO science and related regulatory affairs. We provided researchers around the world with a forum to present their work: researchers from Australia, China, India, Russia, Uruguay, Turkey, to name a few.
Along these years, the PRO Newsletter has been closely linked to the major steps of the parallel development of Patient-Centered Outcomes research and Mapi itself.
The first issue of the PRO Newsletter in 1991 included the first publication about a new 36-item “Health Status Survey,” a year in advance of its more thorough documentation in the APHA journal Medical Care. Coincidentally, the International Quality of Life Assessment (IQOLA) Project was formally launched the same year. The main objectives were to translate and adapt the widely used MOS SF-36 Health Survey Questionnaire in up to 15 countries and validate, norm, and document the new translations. The IQOLA name was coined by Bernard Jambon, the Newsletter’s founder, during the inaugural meeting of the project. Subsequent PRO Newsletter articles in 1992 introduced the IQOLA Project (Issues #3 and #5), which was a public-private partnership sponsored by the pharmaceutical industry that included individuals and organizations from around the world, including Mapi, in a major role coordinating IQOLA activities. This project encouraged us at Mapi to launch similar collaborative cross-cultural researches with, in particular, the Sickness Impact Profile (SIP) and the Psychological General Well-Being Index (PGWBI), and to develop later on our own Linguistic Validation activity.
One of our most interesting and challenging collaborative projects, which was partly funded by the EC Fifth Framework Program, was the International Health-related Quality of Life Outcomes Database (IQOD) Program (officially launched in May 2001). Its aim was to standardize the administration and the interpretation of several PRO instruments (WHQ, PGWBI, and MLHF), and establish reference values per country, population, and disease severity for these instruments in order to allow cross-national comparisons of scores. See Newsletters #21, #23, #27, #29 and #30 for more details.
The Special Issue (SI) of March 1998 (#SI 1) was a report of the first meeting on Quality of Life and Regulatory Issues organized by Mapi and held in Vienna during the 1997 ISOQOL meeting. The main objective of the meeting was to bring together quality of life researchers and representatives of European regulatory agencies in order to allow the authorities to express their thoughts about QOL evaluation within the specific framework of registration and reimbursement of pharmaceutical drugs. The main outcomes of this meeting were the creation of the ERIQA Group (see #21, #24, #27, #38, and #39) and a long lasting collaboration with Laurie Burke which culminated in the four PRO Harmonization Meetings from 2000 to 2002 (see #26 and #27), of which three were held at the headquarters of the Food and Drug Administration (FDA), and in the release of the draft FDA PRO guidance in 2006. We also reported an open-registration meeting intended to facilitate inclusive discussion, dissemination, and operationalization of the anticipated guidance document and organized by the Mayo Clinic College of Medicine and the FDA Center for Drug Evaluation and Research (CDER) (see #36). In issue #47, we provided a full report of the FDA workshop entitled “Measurement in Clinical Trials: Review and Qualification of Clinical Outcome” that was held on October 19, 2011. The main elements of the workshop were (a) the proposed categorization of COAs; (b) the need for quantitative methods to investigate content validity; and (c) the clear push of the FDA for collaborative work and for the Drug Development Tool qualification of COAs.
Ten years after the creation of the QOL Newsletter, 2001 saw the materialization of an original initiative supported by Mapi, i.e., the formal registration of the Cochrane Health-related Quality of Life Methods Group (#26). Its creation was initiated during an exploratory meeting held in Lyon on July 6th, 1998 attended by members of the French Cochrane Centre and researchers interested in HRQoL issues (#20). Since then, regular news on its activities have been provided in the PRO Newsletter. The Methods Group is now co-convened by Donald L. Patrick and Gordon Guyatt.
The exploration of the 51 issues of the Newsletter was the occasion to rediscover with emotion the Mapi training program on the use of PRO Instruments in clinical trials. From 1995 to 2004 we organized 34 sessions in collaboration with PRO instrument developers. Those sessions targeted people from the industry. On April 6, 2001, with the help of Sergei Varshavsky, we held a bilingual session in English and Russian in Moscow (see #26) at the occasion of an international conference, “Human and Medicine,” which was followed by an ERIQA meeting with Russian drug agency representatives (Dr. Dmitry Reihart and Dr. Sergei Filiunin). In 2002, we launched a new program called “Workmats” (which became the PROmats in 2004; see #30) which was widely used to train regulators from 2002 to 2006 [French Medicines Agency (AFSSAPS), FDA, European Medicines Agency (EMA), the Belgium “Institut National d’Assurance Maladie-Invalidité” (INAMI), the French “Agence Nationale d’Accréditation et d’Evaluation en Santé” (ANAES), and the Dutch College voor Zorgverzekeringen (CVZ)].
Faithfull to our humanistic beliefs and encouraged by Dick Joyce in doing so, in 2001 we launched a special award, the Catherine Pouget Award (in memory of a colleague who suffered from the lack of understanding and compassion in the attitude of the medical staff treating her during her battle with cancer). This award was intended to encourage young investigators to study the impact of quality of care for terminally ill patients and/or to develop processes to improve one’s quality of life. From 2002 to onwards, we presented the results of the original and empathetic work of several young investigators (see #29 to #31, #33, #35, #37 to #43, #45, #47 to #49).
During all these years, the appearance of the Newsletter changed several times, from green to blue (#33) to celebrate the creation of Mapi Research Trust, with a new design when the QOL Newsletter became the PRO Newsletter (#34), and two other designs with the evolution of Mapi (#38 and #50) and the involvement of new staff.
We witnessed the growth of the field and its evolution over time with the publication of articles (and books) about new developments such as PROMIS (#38, #47) and computerized adaptive testing (CAT) (#49), ePROs (#45 and #51) and the PRO Consortium (#48 to #51).
Many, many other things could be said about these 23 years of publication, but it might be too long. Suffice to say that the PRO Newsletter has chronicled the growth of the PRO field and has provided its contributors and readers with a forum and a source of considerable value. The 1062 pages published so far are like a history book, the history of how the patient’s voice has finally been listened to and integrated in drug development.
I want to finish my recollection with my sincere and heartfelt thanks to two people very dear to my heart: Dick Joyce and Donald Patrick. Dick, for your gentleness, elegance and your profound humanity. Donald, for your attentiveness and impressive courage and tenacity. And of course, for all the books you advised me to read along all these many years of friendship!
To all those who have enabled the QOL/PRO Newsletter to exist, my sincere and respectful thanks.
Happy birthday to Mapi, Bernard, and all the great colleagues I work with every day.
1. Bruce Springsteen. 57 channels (And Nothin’ On). 1992. Columbia.