Handful of new children’s meds on the horizon. But will they be accessible to children in LMICs?
New analysis: Just 7% of pharmaceutical companies’ R&D is for children under 12, despite significant gaps in paediatric treatment options.
- Out of more than a thousand R&D projects assessed in the 2021 Access to Medicine Index, only 69 are for children under 12.
- Yet there are examples of treatments that could prove game-changers for children in low- and middle-income countries (LMICs).
- Children in LMICs cannot wait at the back of the queue. Planning ahead to ensure access to new medicines is critical. 31/56 late-stage projects have access plans.
Despite significant advances in driving down child mortality rates in recent decades, almost 5.2 million children under five still die every year from preventable and treatable diseases, most of whom are in low- and middle-income countries (LMICs). Far too often the youngest members of society are at the back of the queue when it comes to receiving treatment, due to the lack of availability of appropriate medicines.
The obstacles are well known. Many medicines come as hard pills or bitter syrups that are difficult to swallow, few treatments are tested in clinical trials in children, and the small and fragmented nature of the paediatric market provides little incentive for industry investment.
As a result, it takes far longer for child-friendly medicines to reach the market than adult versions and there are major gaps in the paediatric drugs pipeline. Indeed, only 69 – or less than 7% – of the 1,073 research and development (R&D) projects underway at leading pharmaceutical companies target children below the age of 12, according to an analysis from the 2021 Access to Medicine Index. It is, however, encouraging that at least 53 of the 69 paediatric R&D projects identified by our analysis are designed for children under the age of five, since this is the group in which the majority of paediatric deaths occur.
Closing gaps in access to medicine for children: how R&D and delivery efforts can be ramped up
What the analysis shows: paediatric pipeline breakdowns; comparison of companies' late-stage coverage of access plans; examples of potentially game-changing products and; tangible actions to help ramp up R&D and delivery efforts.
Some new treatments could prove game-changers for children
But pharmaceutical companies now have a chance to turn the tide, with the arrival of a number of game-changing new medicines in late-stage development or having gained market approval. These treatments could dramatically improve the health of children in LMICs, provided they reach the patients that need them most.
These initiatives show what is possible when pharmaceutical companies address the challenge of paediatric healthcare head-on. However, the current response across the industry is patchy, with GSK, Johnson & Johnson and Sanofi the most active in the field.
- Eli Lilly’s pioneering glucagon nasal powder (Baqsimi®) is the first dry nasal spray that can treat severe hypoglycaemia
in children with diabetes over the age of four. It is a form of glucagon given as a puff in the nose, enabling easy administration. This is the first nasal glucagon to enter the market.
- Bristol Myers Squibb’s dasatinib (Sprycel®) is the first oral suspension formulation for paediatric patients from the age of one with acute lymphoblastic leukaemia.
- Astellas and MSD* oral suspension of fidaxomicin (Dificid®) for treating Clostridioides difficile, a common cause ofdiarrhoeal disease, in children aged six months. Across LMICs, one in ten deaths in children younger than five years is attributable to this illness.
- Dolutegravir (Tivicay®) by GSK (through ViiV Healthcare) is the first dispersible tablet formulation of dolutegravir to treat HIV in children weighing at least 3kg and from four weeks of age. This treatment, for use in combination with other antiretroviral agents for the treatment of HIV, is taste-masked and can be dispersed in water, making it easier to administer to children.
Only half of treatments entering the market show signs of access
But the litmus test for all the paediatric products in development lies not only in securing market approval. That is just the first step. Pharmaceutical companies must also have concrete plans in place to make them accessible and affordable in all the markets where they are needed – including small LMICs that are frequently overlooked.
For example, nearly half of the 56 paediatric projects that have reached Phase II and onwards – when access planning should be taking place – are still not supported by access plans. Worryingly, this includes seven newly approved products.
“It is good news that new medicines for paediatric cancer and diabetes, amongst others, are entering the market. But it is concerning that many children in lower-income countries will not be able to access these products rapidly. Our research shows that access to medicine does not happen by itself - it requires concrete plans to ensure supply and address affordability for specific groups, among other issues. That planning starts with the pharma companies that hold the reins for delivery,” said Claudia Martinez, Index Research Programme manager.
More work needed to widen the scope of paediatric medicine
Clearly, there is considerable scope for both the industry and multilateral donors to widen the scope of their paediatric endeavour. To date, much of the focus has been on infectious diseases like HIV and malaria, where donor involvement, market-shaping activities and partnerships have successfully prompted corporate action. But there has been less attention paid to non-communicable diseases that also pose serious threats to children’s health, such as epilepsy, diabetes, cancer and sickle cell disease – the last of which overwhelmingly affects individuals living in sub-Saharan Africa.
Currently, the three disease areas that are focus of most paediatric R&D projects are lower respiratory infections, HIV and cancer. The attention to children’s cancers is welcome, although there is still a huge amount of work to do to level the gulf in care between rich and poor, since childhood cancer survival rates are just 20% in low-income countries compared to 80% in high-income ones. Meanwhile, the amount of work underway for neonatal conditions like sepsis is alarmingly low, with only five projects in development.
A chance to turn the tide on life-saving medicines for children
Pharmaceutical manufacturers have shown they can do more – for example, by signing up to the Rome Action Plan, a compilation of commitments by pharmaceutical companies and global health stakeholders to accelerate the development of paediatric HIV/AIDS and TB products. Gilead, GSK (as ViiV Healthcare), Johnson & Johnson, MSD*, Roche and Sanofi have signed up to the action plan.
Broadening the scope of paediatric medicine R&D to more disease areas and improving accessibility will take close coordination between companies and other stakeholders. To show where action is possible, the new analysis describes a range of steps that pharmaceutical companies, product development partners, governments and the global health community can take including:
- Simplify and incentivise the paediatric R&D process
- Become drivers of access through access planning
- Build on the advances in HIV/AIDS and malaria
- Leverage financing, for example, from government funding, development assistance and philanthropic finances
"Ramping up access for children is technically difficult, though it can and must be done. Which is why it is urgent that companies, regulators and governments prioritise ways to remove barriers, share risks and shape an environment for faster innovation and faster access," Jayasree K. Iyer, Executive Director of the Access to Medicine Foundation.
About the finding
This finding is part of the 2021 Access to Medicine Index. The Index analyses 20 of the largest research-based pharmaceutical companies with products for high-burden diseases in low- and middle-income countries. It ranks them on their efforts to improve access to medicine, identifies best practices, highlights where progress is being made and uncovers where critical action is still required. The Index is endorsed by more than 100 investors, collectively managing assets worth over USD 14 trillion. It is published every two years by the Access to Medicine Foundation, an independent non-profit organisation funded by the UK Government (UK AID), the Bill & Melinda Gates Foundation, the Dutch Ministry of Foreign Affairs, the Wellcome Trust and AXA Investment Managers.
*Merck & Co, Inc (Kenilworth, NJ USA)