Over the past 5 years, pharmaceutical companies have increasingly offered patients access to novel agents without charge on compassionate use programmes. This has allowed patients who have exhausted conventional treatment options and do not have access to clinical trials, further treatment lines. Additionally, whole genome sequencing (WGS) has opened up new targets personalised for each patient. At a primary treatment centre in the UK, approximately 100 patients have benefited from this pathway.

Access to these drugs for paediatric patients represents an opportunity for patients to find that elusive cure and offers hope in difficult circumstances. However, it also means that boundaries previously in place around ceilings of care are no longer in place, patients can go through numerous additional lines of treatment. This poses ethical challenges where patients and parents cling onto any chance of a cure and additionally clinicians feel under pressure to offer these untested treatments.

The challenge for pharmacy and the healthcare system as a whole comes in many forms.

Repurposing targeted therapies for children comes with risk and challenge. Medicines may not have been used in children and thus formulations may not be suitable. Dosing information may not be available, which means safe dosing may need to be extrapolated from adult data. Where medicines need aseptic manipulation, stability and quality assurances processes need to be conducted within technical services. Medicines may not be licensed in the UK – therefore product quality and assurance checks need to take place within each hospital pharmacy.

The management of stock is also complex where a pharmacy stock commercial stock and compassionate stock of the same drug, which will look identical. Mechanisms need to be developed to segregate stock and also ensure that stock is used for the correct patients. Traditionally, dedicated procurement teams maintain appropriate levels of stock of drugs in a pharmacy, with electronic systems helping manage stock levels. Compassionate use medicines need to be ordered by clinical pharmacists on a named patient basis, often lead times can vary and supplies can be limited. To add to all this, each patient may require individualised treatment protocols written.

A move to oral targeted therapies from conventional protocol based therapy means that patients are on a very different pathway to that normally seen in paediatric cancer. Predominantly outpatient based and clinic based, these patients often miss clinical pharmacist oversight in this immature pathway, leading to challenges around logistics, supply and clinical pharmacy input.

The resource required for managing a compassionate use access programme is equivalent to running a clinical trial, and yet the evolution of these programmes has been absorbed into the existing clinical pharmacy service.

While great opportunity and benefit to patients is being seen, the impact of the increased availability of compassionate use medicines has led to unsustainable growth without additional resource put into pharmacy teams to manage these complex pathways. Consideration for pharmacy infrastructure is needed be able to maintain a safe and effective service.