Two girls stand outside a local church in Salar de Uyuni, Bolivia. | 2008 April Pojman, Courtesy of Photoshare


Rome action plan

In November 2017, a High-Level Dialogue on Scaling Up Early Diagnosis and Treatment of Children and Adolescents was convened by His Eminence Peter Kodwo Appiah Cardinal Turkson, Prefect of the Dicastery for the Promotion of Integral Human Development, with PEPFAR, UNAIDS and Caritas Internationalis, and in close collaboration with the World Council of Churches-Ecumenical Advocacy Alliance, the Elizabeth Glaser Pediatric AIDS Foundation (EGPAF) and the WHO. Key principles of the GAP-f set the basis of discussion, which led to an action plan including an impressive list of commitments from industry, regulators, UN agencies and other stakeholders.

The commitments from the Rome action plan promote three key principles:

  1. Focusing on priority paediatric drugs and formulations
  2. Accelerating development, review, and introduction of paediatric formulations
  3. Collaborating to expedite the development and introduction of paediatric products.

Action Plan for Scaling Up Early Diagnosis and Treatment of Children and Adolescents
17 November 2017
See the action plan here | See the action plan monitoring tracker here

High-Level Dialogue to Assess Progress and Intensify Commitment to Scaling Up Diagnosis and Treatment of Paediatric HIV
6-7 December 2018 | Pontifical Academy of Sciences (Vatican City)
See the EGPAF/WHO press release “A new push for timely diagnosis and treatment of children with HIV” here


PADO-TB – List of priority paediatric antituberculosis drug formulations
First Paediatric Antituberculosis Drug Optimization Meeting (PADO-TB 1)

See the PADO-TB 1 meeting report and list of prioritized paediatric antituberculosis drug formulations here

PADO-HIV – Review of priority paediatric ARV formulations
Paediatric ARV Drug Optimization 3 Review

See the summary report from the review process here

PADO-HIV – List of priority paediatric ARV formulations
Paediatric Antiretroviral Drug Optimization (PADO) Meeting 3

See the PADO 3 meeting report and list of prioritized paediatric ARV formulations here

ILF – Policy brief on advance procurement of paediatric drug formulations
The many challenges of developing drugs appropriately formulated for children are compounded by a series of market factors that make these efforts costly and complex, discouraging drug manufacturers – especially generic drug manufacturers – from prioritizing investments in new paediatric treatment options. This brief explores the concept of advance procurement as a method for reducing uncertainty within the paediatric ART market and spurring investment in adapted child-friendly drug formulations.

Access the policy brief here

EMA – Paediatric medicines: Overview
The European Union Paediatric Regulation enables the EMA to stimulate research into the uses of medicines in children and to overview their authorisation.

Access the overview of EMA’s paediatric medicines work here

EMA - European Medicines Agency and European Commission (DG Health and Food Safety) action plan on paediatrics
This action plan takes into account the suggestions made at a multi-stakeholder workshop convened by the European Commission and the European Medicines Agency on 20 March 2018, where ways to improve the implementation of the Paediatric Regulation were discussed.

See the action plan here

PAWG – Toolkit for research and development of paediatric antiretroviral drugs and formulations
Research toolkit for paediatric HIV drug development developed by the WHO convened Paediatric ARV Working Group (PAWG) in collaboration with Unitaid, IMPAACT and PENTAid.

Access the toolkit here | Access an online version with slide sets here

Transitioning to an optimal paediatric ARV formulary: implementation considerations
Key considerations to facilitate effective transition to more clinically appropriate regimens as optimal ARV medicines and dosage forms become available

See the policy brief here

EMA – Concept paper on the need for revision of the guideline on the investigation of medicinal products in the term and preterm neonate
This concept paper discusses the need to update currently available guidelines regarding the non-clinical and clinical development of medicinal products in neonates in accordance with current trends in neonatal terminology, and issues encountered and experience gained during assessment of several Paediatric Investigation Plans (PIP) applications involving neonatal population. Input will be sought from relevant working parties, committees and experts.

See the concept paper here

FDA – Quality attribute considerations for chewable tablets: Guidance for industry
Recommendations on critical quality attributes that should be considered when developing chewable tablets and selection of acceptance criteria that are appropriate and meaningful indicators of product performance throughout the product shelf life.

See the guidance here

FDA – Pediatric HIV infection: Drug product development for treatment - Guidance for industry
General recommendations on the development of antiretroviral products for the treatment of HIV infection in paediatric patients (birth to younger than 17 years of age)

See the draft guidance here

2018 Optimal Formulary and Limited-Use List for Paediatric ARVs
Fifth edition of the Optimal Formulary and Limited-use List supporting the transition to optimal WHO-recommended regimens for infants and children

See the list of optimal and limited-use paediatric ARV formulations here

APWG – Updates, forecasts and other documents
ARV Procurement Working Group Documents

See various updates from the ARV Procurement Working Group here

Bringing it all together: The Global Accelerator for Paediatric Formulations (GAP-f)
Accelerating the development and uptake of the most needed drug formulations for children, AIDS 2018
Penazzato & Domanico – July 2018 | Amsterdam, the Netherlands

The Global Accelerator for Paediatric Formulations (GAP-f): Accelerating the development and uptake of the most needed drug formulations for children
11th World Meeting on Pharmaceutics, Biopharmaceutics and Pharmaceutical Technology
Morin, Cohn, Vicari, Domanico, Lewis, Watkins, Pascual, Lee, Sugandhi, Eisenhawer, Pérez Casas, Auton, Siberry, Giaquinto, Penazzato, On behalf of GAP-f partners -- March 2018 | Granada, Spain

Accelerating development and introduction of critical tools to diagnose and treat HIV in children and adolescents: Overview of the issues and potential solutions
High-Level Dialogue on Scaling Up Early Diagnosis and Treatment of Children and Adolescents
Penazzato -- 17 November 2017 | Vatican City, Italy

The Global Accelerator for Paediatric Formulations (GAP-f): Ensuring children have accelerated access to optimal drug formulations
9th European Paediatric Formulation Initiative (EuPFI) Conference
Morin -- 20 September 2017 | Warsaw, Poland

See the slide set here

Hepatitis C virus infection in children and adolescents
Indolfi, Easterbrook, Dusheiko, El-Sayed, Jonas, Thorne, Bulterys, Siberry, Walsh, Chang, Meyers, Giaquinto, Wirth, Chan & Penazzato
The Lancet Gastroenterology & Hepatology, 2019, Published online first

Treating young children co-infected with tuberculosis and HIV
Turkova & Bamford
The Lancet HIV, 2019, 6(1): PE4-E6

Catalysing the development and introduction of paediatric drug formulations: a new global collaborative framework for action
Penazzato, Watkins, Morin, Lewis, Pascual, Vicari, Lee, Hargreaves, Doherty & Siberry
The Lancet HIV, 2018, 5(5): 259-e264

Completion Rate and Safety of Tuberculosis Infection Treatment With Shorter Regimens
Cruz & Stark
Pediatrics, 2018, 141(2)

Challenges of using new and repurposed drugs for the treatment of multidrug-resistant tuberculosis in children
Schaaf, Garcia-Prats, McKenna & Seddon
Expert Rev Clin Pharmacol, 2018, 11(3):233-244

Shortening the decade-long gap between adult and paediatric drug formulations: a new framework based on the HIV experience in low- and middle-income countries
Penazzato, Lewis, Watkins, Prabhu, Pascual, Auton, Kreft, Morin, Vicari, Lee, Jamieson & Siberry
J Int AIDS Soc, 2018, 21(S1), e25049

Prevention and treatment of HIV infection in neonates: evidence base for existing WHO dosing recommendations and implementation considerations
Clarke, Penazzato, Capparelli, Cressey, Siberry, Sugandhi & Mirochnick, WHO Paediatric Antiretroviral Working Group
Expert Rev Clin Pharmacol, 2018, 11(1):83-93

Meeting the goal of concurrent adolescent and adult licensure of HIV prevention and treatment strategies
Hume, Lewis & Nelson
J. Med. Ethics, 2017, 0: 1-4

Recommendations for Optimizing Tuberculosis Treatment: Therapeutic Drug Monitoring, Pharmacogenetics, and Nutritional Status Considerations
Choi, Jeong, Koh & Lee
Ann Lab Med, 2017, 37(2): 97-107

Prioritizing the most needed formulations to accelerate paediatric antiretroviral therapy scale-up
Penazzato, Palladino & Sugandhi, on behalf of the PADO 3 Meeting participants
Curr Opin HIV AIDS, 2017, 12:369-376

Optimizing Research to Speed Up Availability of Pediatric Antiretroviral Drugs and Formulations
Penazzato, Gnanashanmugam, Rojo, Lallemant, Lewis, Rocchi, Saint Raymond, Ford, Hazra, Giaquinto, Belew, Gibb, & Abrams, for the PAWG
Clin Infect Dis, 2017, 64(11):1597-1603

Optimization of the strength of the efavirenz/lamivudine/abacavir fixed-dose combination for paediatric patients
Bouazza, Cressey, Foissac, Bienczak, Denti, McIlleron, Burger, Penazzato, Lallemant, Capparelli, Treluyer & Urien
J Antimicrob Chemother, 2017, 72(2):490-495

Advances in Drug Discovery and Development for Pediatric Tuberculosis
Hoagland, Zhao & Lee
Mini Rev Med Chem, 2016, 16(6): 481-497

Towards early inclusion of children in tuberculosis drugs trials: a consensus statement
Nachman, Ahmed, Amanullah, Becerra, Botgros, Brigden, Browning, Gardiner, Hafner, Hesseling, How, Jean-Philippe, Lessem, Makhene, Mbelle, Marais, McIlleron, McNeeley, Mendel, Murray, Navarro, Anyalechi, Porcalla, Powell, Powell, Rigaud, Rouzier, Samson, Schaaf, Shah, Starke, Swaminathan, Wobudeya & Worrell
Lancet Infect Dis, 2015, 15(6):711-720

The background and rationale for a new fixed-dose combination for first-line treatment of tuberculosis in children
Graham, Grzemska & Gie
Int J Tuberc Lung Dis, 2015, 19(1): S3-S8

The procurement landscape of pediatric tuberculosis treatment: a Global Drug Facility perspective
Scott, Gardiner & de Lucia
Int J Tuberc Lung Dis, 2015, 19(1): S17-S22

Pediatric tuberculosis drug market: an insider perspective on challenges and solutions
Usherenko, Basu Roy, Mazlish, Liu, Benkoscki, Coutts, Epstein, Qian, Rafiq, & Scott
Int J Tuberc Lung Dis, 2015, 19(1): S23-S31

From availability to uptake: planning for the introduction of new, child-friendly anti-tuberculosis formulations
Malhotra, Ursu, Ghoneim, Jodrey, Paredes, Brown, Soucy & Barr-DiChiara
Int J Tuberc Lung Dis, 2015, 19(1): S32-S38

A bitter pill to swallow: the need for better medications for drug-resistant tuberculosis in children
Furin, Mafukidze, Brigden, du Cros, Golin, Harausz, Seddon, Ustero, & Garcia-Prats
Int J Tuberc Lung Dis, 2015, 19(1): S55-S60

Paediatric formulations of second-line anti-tuberculosis medications: challenges and considerations
Taneja, Garcia-Prats, Furin & Maheshwari
Int J Tuberc Lung Dis, 2015, 19(1): S61-S68

Accelerating clinical drug development for children with tuberculosis
Murray, McKenna, Pelfrene & Botgros
Int J Tuberc Lung Dis, 2015, 19(1): S69-S74

Optimizing drugs to reach treatment targets for children and adolescents living with HIV
Penazzato, Lee, Capparelli, Essajee, Ford, Ojoo, Pascual, Sugandhi & Lallemant
J Int AIDS Soc, 2015,18(Suppl 6):20270

Pharmacokinetic and Pharmacodynamic Considerations in Antimalarial Dose Optimization
Antimicrob Agents Chemother, 2013, 57(12): 5792-5807

The HCV Treatment Pipeline
Treatment Action Group, 2018

The Tuberculosis Prevention Pipeline for Children
Treatment Action Group, 2018

The Tuberculosis Treatment Pipeline for Children
Treatment Action Group, 2018

New Pathways for Childhood TB Treatment: Lessons from the STEP-TB Project
TB Alliance & Unitaid, 2017

The Pediatric Tuberculosis Treatment Pipeline: Beyond Pharmacokinetics and Safety Data
McKenna, Treatment Action Group, 2016

Optimizing Malaria Treatment in the Community (this is a chapter in a book)
Hawkes & Serghides
Optimizing Treatment for Children in the Developing World, 2015, 251-264

Prescription pharmaceuticals in Canada Off-label use
Standing Senate Committee on Social Affairs, Science and Technology, 2014

Improving Medicines for Children in Canada
Expert Panel on Therapeutic Products for Infants, Children, and Youth, 2014

Comparative, Open Label, Dose and Regimen Optimization Follow-up Study of Intravenous and Intramuscular Artesunate in African Children With Severe Malaria
Kremsner, Köhler & Bolte, Universitätsklinikum Tübingen, 2011

EuPFI – European Paediatric Formulation Initiative
With subgroups focused on administration devices, biopharmaceutics, taste masking/testing, age appropriateness, and excipients

Enpr-EMA – European Network of Paediatric Research at the European Medicines Agency
Network of research networks, investigators and centres with recognised expertise in performing clinical studies in childrens

GRiP – Global Research in Paediatrics
Network of excellence funded by the European Union

GPFC – Goodman Pediatric Formulations Centre (GPFC)
Rosalind & Morris Goodman Family Pediatric Formulations Centre of the CHU Sainte-Justine

LEAP – Long-Acting / Extended Release Antiretroviral Resource Program
Portal funded by the U.S. National Institutes of Health

MAP-IT Model – Model for Assessment of Pediatric Interventions for Tuberculosis
RTI International, TB Alliance, Unitaid

PaedForm – European Paediatric Formulary
Council of Europe

PDCO – Paediatric Committee
European Medicines Agency

STEP database – Safety & Toxicity of Excipients for Paediatrics
European Paediatric Formulation Initiative

USPFI – United States Pediatric Formulation Initiative
In the context of the Best Pharmaceuticals for Children Act (BPCA) and with groups focused on scientific/technical/regulatory barriers, taste/smell/flavour, economics/partnerships, and use/application of new delivery systems