Making Clinical Trials More Patient-CentredНоябрь 2015
The recent EURORDIS Round Table of Companies Workshop brought together over 100 participants to discuss how to make clinical research more patient-centred through patient-reported outcomes and patient-centred outcome measures.
Making clinical studies more patient-centred means that they are designed to be as relevant as possible to patients and therefore the most efficient at generating evidence to prove that a therapy will improve lives.
What are patient-centred outcome measures?
To determine clinical safety and efficacy of a medicine, clinical trial results must reflect relevant ‘outcome measures’, also referred to as ‘endpoints’ or ‘outcomes’.
Clinical trials do not always measure outcomes that patients consider important or relevant, potentially resulting in a medicine that does not entirely reflect the needs of patients.
Patient-reported outcomes (PROs) involve the collection of data provided directly by patients regarding their health condition. This data is more often qualitative than quantitative (as opposed to very quantitative data often collected during a clinical trial, such as blood test results).
This information is then used to inform patient-centred outcome measures (PCOMs). PCOMs set out what should be measured and reported in clinical trials with the aim of determining whether or not the therapy in question will improve patients’ lives. Many trials, on rare diseases in particular, still do not include standardised outcome measures. Insufficient attention to the selection of clinical trial outcomes leads to a waste of generated data, research efforts and inefficiencies in drug development and in regulatory review processes.
PCOMs aim to place patients, their families and carers at the heart of decisions concerning the most valuable criteria (clinical trial outcomes) in health assessment, rather than leaving assessments to clinicians only.
The EURORDIS Round Table of Companies (ERTC) workshop served as a forum for discussion between all stakeholders present (regulators, pharmaceutical and biotech companies, patient representatives and research bodies) with the aim of reaching a common understanding of PROs and PCOMs.
It is aligned with the work done by the International Rare Diseases Research Consortium (IRDiRC), which has issued recommendations highlighting the importance of PCOMs and PROs. These recommendations have led to the creation of a task force gathering top experts to consider what action needs to be taken specifically in the field of rare diseases.
The following conclusions were reached at the ERTC Workshop:
- There should be collaboration between companies, researchers and regulators so that PCOMS/ PROs are developed to be standardised across the board. Developing standardised core sets of PCOMs would improve the quality of clinical studies or trials.
- There is a need for a clear scientific consensus on the definition of PCOMs and PROs that is accepted by all stakeholders. First steps have been taken in defining these concepts; read the IRDiRC Preparatory Document for Workshop on PCOMs.
- Workshop participants discussed the challenges around PROs and PCOMs, including: the cost of collecting qualitative data from patients; the issue that qualitative data expressed by patients can be subjective and more difficult to measure than quantitative data; and the fact that because rare disease populations are very small there are fewer patients who can participate in the development of PROs/ PCOMs.
For information regarding the next ERTC meeting entitled ‘Multi-stakeholder Symposium on Improving Patient Access to Rare Disease Therapies’, taking place 24 — 25 February 2016, please email email@example.com.
Eva Bearryman, Junior Communications Manager, EURORDIS