ReviewFrontiers in pharmacology2019
The New Paradigms in Clinical Research: From Early Access Programs to the Novel Therapeutic Approaches for Unmet Medical Needs.
Review in Frontiers in pharmacology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
22 citing papers in PubMed.
- Article
- A model for the clinical management of the introduction of new medicines in a large University Hospital in Spain.Journal of pharmaceutical policy and practice · 2026Article
- Exploring community pharmacy manager/ pharmacist perceptions and responses to China's dual-channel policy for improving access and rational use of innovative drugs: a qualitative study.International journal of clinical pharmacy · 2025Article
- The UK's Early Access to Medicines Scheme 10 years on: an evaluation using publicly available data.JRSM open · 2025Article
- Occupational hygiene risk assessment at light speed-a study for protecting worker health and safety in the biopharmaceutical industry.Frontiers in public health · 2025Review
- Computational identification of potential modulators of heme-regulated inhibitor (HRI) for pharmacological intervention against sickle cell disease.Research square · 2023Article
- A pan-serotype antiviral to prevent and treat dengue: A journey from discovery to clinical development driven by public-private partnerships.Antiviral research · 2023Review
- Meeting the Need for a Discussion of Unmet Medical Need.Healthcare (Basel, Switzerland) · 2022Article
- Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain).Journal of clinical medicine · 2022Article
- A survey on work status and competencies of Clinical Research Nurses in China.Journal of research in nursing : JRN · 2022Article
- The value of real world evidence: The case of medical cannabis.Frontiers in psychiatry · 2022Review
- Non-coding RNAs and their bioengineering applications for neurological diseases.Bioengineered · 2021Review
- Associations between uncertainties identified by the European Medicines Agency and national decision making on reimbursement by HTA agencies.Clinical and translational science · 2021Article
- A Biomarker-Centric Approach to Drug Discovery and Development: Lessons Learned from the Coronavirus Disease 2019 Pandemic.The Journal of pharmacology and experimental therapeutics · 2021Review
- Pharmacological, Technological, and Digital Innovative Aspects in Rhinology.Frontiers in allergy · 2021Review
- Reporting rates of adverse reactions to specialty care medicines exhibit a direct positive correlation with patient exposure: A lack of evidence for the Weber effect.British journal of clinical pharmacology · 2020Article
- Computer-Aided Estimation of Biological Activity Profiles of Drug-Like Compounds Taking into Account Their Metabolism in Human Body.International journal of molecular sciences · 2020Article
- Defining a Regulatory Strategy for ATMP/Aerosol Delivery Device Combinations in the Treatment of Respiratory Disease.Pharmaceutics · 2020Review
- Article
- Precision Dosing Priority Criteria: Drug, Disease, and Patient Population Variables.Frontiers in pharmacology · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Despite several innovative medicines gaining worldwide approval in recent years, there are still therapeutic areas for which unsatisfied therapeutic needs persist. For example, high unmet clinical need was observed in patients diagnosed with type 2 diabetes mellitus and hemophilia, as well as in specific age groups, such as the pediatric population. Given the urgent need to improve the therapy of clinical conditions for which unmet clinical need is established, clinical testing, and approval of new medicines are increasingly being carried out through accelerated authorization procedures. Starting from 1992, the Food and Drug Administration and the European Medicines Agency have supported the so-called Early Access Programs (EAPs). Such procedures, which can be based on incomplete clinical data, allow an accelerated marketing authorization for innovative medicines. The growth in pharmaceutical research has also resulted in the development of novel therapeutic approaches, such as biotech drugs and advanced therapy medicinal products, including new monoclonal antibodies for the treatment of asthma, antisense oligonucleotides for the treatment of Duchenne muscular dystrophy and spinal muscular atrophy, and new anticancer drugs that act on genetic biomarkers rather than any specific type of cancer. Even though EAPs and novel therapeutic approaches have brought huge benefits for public health, their implementation is limited by several challenges, including the high risk of safety-related label changes for medicines authorized through the accelerated procedure, the high costs, and the reimbursement and access concerns. In this context, regulatory agencies should provide the best conditions for the implementation of the described new tools.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.