Publications by authors named "Francesca Cerreta"

The European Medicines Agency adopted their Geriatric Medicines Strategy more than a decade ago. The strategy aims at elucidating the evidence basis for marketing authorization of new medicines which will be used in the older population, and at ensuring the appropriate communication of findings to the patient and healthcare provider. During the past decade new tools and data sources have emerged to support the strategy goals, and their use should be considered.

View Article and Find Full Text PDF

The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) E7, the guidance for the conduct of clinical trials in people older than age 65 years, dates from 1994. Since then, the inclusion of older people in clinical trials has hardly improved, particularly for the oldest old age group (individuals older than age 75 years), which is the fastest growing demographic bracket in the EU. Even though most medications are taken by this group, relevant endpoints and safety outcomes for this cohort are rarely included and reported, both in clinical trials and regulatory approval documents.

View Article and Find Full Text PDF

Aims: To describe characteristics of applicant, tool, outcomes, regulatory responses and general learnings from European Medicines Agency (EMA) Qualification Procedures on patient-reported outcomes (PROs), observer-reported outcomes (ObsROs) and performance outcomes (PerfOs) finalized between January 2013 and December 2018.

Methods: Descriptive analysis, and qualitative review of the regulatory outcomes of the study procedures.

Results: Seventeen qualification programmes for PROs, 6 for ObsRO tools and 11 for PerfO tools were submitted by consortia, large and small/medium companies.

View Article and Find Full Text PDF

Digital health technology tools (DHTTs) present real opportunities for accelerating innovation, improving patient care, reducing clinical trial duration and minimising risk in medicines development. This review is comprised of four case studies of DHTTs used throughout the lifecycle of medicinal products, starting from their development. These cases illustrate how the regulatory requirements of DHTTs used in medicines development are based on two European regulatory frameworks (medical device and the medicinal product regulations) and highlight the need for increased collaboration between various stakeholders, including regulators (medicines regulators and device bodies), pharmaceutical sponsors, manufacturers of devices and software, and academia.

View Article and Find Full Text PDF

The aging processes alter the body's response to a medicine's pharmacokinetics, pharmacodynamics, and susceptibility to adverse effects. In addition, older adults, especially the oldest age category (85+ years) or those with multiple chronic health conditions, polypharmacy, or frailty, are under-represented in clinical trials of new medicines. Evidence-based prescribing guidelines based on these trials might result in inappropriate prescription, increasing the risk of drug interactions and adverse drug reactions.

View Article and Find Full Text PDF

Older people are often affected by impaired organ and bodily functions resulting in multimorbidity and polypharmacy, turning them into the main user group of many medicines. Very often, medicines have not specifically been developed for older people, causing practical medication problems for them like limited availability of easy to swallow formulations, easy to open packaging and dosing instructions for enteral administration. In 2020, the European Medicines Agency (EMA) published a reflection paper 'Pharmaceutical development of medicines for use in the older population', which discusses how the emerging needs of an ageing European population can be addressed by medicines regulation.

View Article and Find Full Text PDF

Background: Functional outcome measures used to assess efficacy in clinical trials of investigational treatments for rare neuromuscular diseases like Duchenne muscular dystrophy (DMD) are performance-based tasks completed by the patient during hospital visits. These are prone to bias and may not reflect motor abilities in real-world settings. Digital tools, such as wearable devices and other remote sensors, provide the opportunity for continuous, objective, and sensitive measurements of functional ability during daily life.

View Article and Find Full Text PDF

Changes that accompany older age can alter the pharmacokinetics (PK), pharmacodynamics (PD), and likelihood of adverse effects (AEs) of a drug. However, older adults, especially the oldest or those with multiple chronic health conditions, polypharmacy, or frailty, are often under-represented in clinical trials of new drugs. Deficits in the current conduct of clinical evaluation of drugs for older adults and potential steps to fill those knowledge gaps are presented in this communication.

View Article and Find Full Text PDF

Background: In 2016, an expert working group was convened under the auspices of the European Society for Clinical and Economic Aspects of Osteoporosis and Osteoarthritis (ESCEO) and formulated consensus recommendations for the conduct of clinical trials for drugs to prevent or treat sarcopenia.

Aims: The objective of the current paper is to provide a 2020 update of the previous recommendations in accordance with the evidence that has become available since our original recommendations.

Methods: This paper is based on literature reviews performed by members of the ESCEO working group and followed up with face to face meetings organized for the whole group to make amendments and discuss further recommendations.

View Article and Find Full Text PDF

Aims: The aim of this study was to elucidate drug prescription patterns in older European people with the objective to support regulatory contextualisation of (1) the suitability of enrolment criteria for new clinical trials; and (2) the understanding of the potential interactions/incompatibilities of newly authorised medicines with those most frequently used by older people.

Methods: Medicines agencies in Portugal, Poland, Slovakia and England were approached to provide a list of the 10 most frequent prescriptions in 2016 for systemically used medicines per active substances (i.e.

View Article and Find Full Text PDF

However, semantics aside, we think that DXA can indeed serve as a reference standard for measuring muscle mass. Obviously, CT and MRI are advanced techniques that can and have been used to obtain important information such as muscle size/volume and more recently amount and distribution of intra- and intermuscular adipose tissue. Also individual muscles can be assessed separately.

View Article and Find Full Text PDF

Purpose: To describe whether for the cardiovascular and antithrombotic medicines approved in the period 2006-2016 (a) the pivotal trial was designed with an upper ageexclusion criterion, (b) the age distribution of the participants in the registration trials reflected expected use, (c) post-authorisation studies were planned, and d) the age distribution of participants was clearly presented in the approval documents.

Methods: The data provided to EMA in support of centralized marketing authorisations of were analysed.

Results: Two out of 19 protocols excluded patients over 80 and 85 years old.

View Article and Find Full Text PDF

Medicines Adaptive Pathways to Patients (MAPPs) seeks to foster access to novel beneficial treatments for the right patient groups at the earliest appropriate time in the product life-span, in a sustainable fashion. We summarize the MAPPs engagement process and critical questions to be asked at each milestone of the product life-span. These considerations are of relevance for regulatory and access pathways that strive to address the "evidence vs.

View Article and Find Full Text PDF

Background: All proposed definitions of sarcopenia include the measurement of muscle mass, but the techniques and threshold values used vary. Indeed, the literature does not establish consensus on the best technique for measuring lean body mass. Thus, the objective measurement of sarcopenia is hampered by limitations intrinsic to assessment tools.

View Article and Find Full Text PDF

Background: Sarcopenia is increasingly recognized as a correlate of ageing and is associated with increased likelihood of adverse outcomes including falls, fractures, frailty and mortality. Several tools have been recommended to assess muscle mass, muscle strength and physical performance in clinical trials. Whilst these tools have proven to be accurate and reliable in investigational settings, many are not easily applied to daily practice.

View Article and Find Full Text PDF

Ageing comes with an increased propensity in the alteration of human organ and body functions, which can e.g. result in multi-morbidity, frailty, polypharmacy, altered medication safety and/or efficacy, and problems with the practical use of medicines in a real world setting.

View Article and Find Full Text PDF