Including the caregiver in access submissions
Partnering with caregivers can help build a more powerful case for re-imbursement if pharma can gather the right evidence
While pharma now understands the merit of placing the patient at the centre of all it does, the industry typically gives too little consideration to another important stakeholder - the caregiver.
Better products and outcomes are two benefits of doing so. Another is the potential to make a stronger case for a therapy with payers. Because caregivers often prescribe or administer a medicine, they can provide valuable insights to strengthen evidence in access submissions.
“Often, in rare diseases the patient’s abilities are compromised,” says Raquel Cabo, VP of market access and patient services at Ovid Therapeutics, a biotech company that develops drugs for rare neurological conditions. “They may have intellectual or development disabilities so we really rely on the caregiver to advocate for them and give insights into the patient experience.”
It is the caregiver, according to Cabo, who can really help provide a true understanding of what the unmet needs are, what the burden of the condition is on the patient and on the wider family, to build a greater understanding of the true benefits of a new therapy.
“Really focusing on understanding those experiences in a holistic way from early diagnosis is crucial,” she says.
But identifying opportunities to capture data outside of traditional endpoints in clinical trials is often tricky given how difficult it is to demonstrate outcomes such as adherence and patient wellness within the framework of a clinical trial, says Kyle Hvidsten, vice president, head of global health economics and value assessment, at Sanofi.
“The key challenge before us is what we can test and demonstrate before approval, what we need to improve in clinical practice to demonstrate that added value and how that aligns with reimbursement decision-making.”
Looking for signals
Pharma must find ways of extrapolating from the clinical evidence factors such as what a treatment will mean for a patient’s cognitive and social function, their productivity at work and what the impact will be on their wellness and so on, says Hvidsten.
“At the point of moving from pre-clinical to clinical, when we are looking for a signal between the treated group and the control group, we need to understand the impact of a treatment on the patient and caregiver, usually through interviews, ideally before Phase 2a, so we can develop and test measures in early development that we can then use as a basis for evidence in full development.”
Cabo agrees that a key means of starting to capture and understand the clinical meaningfulness of the carer experience is experience questionnaires or surveys added on to Phase 2. “Will this make it onto your regulatory label? No, but it does offer a different perspective and a qualitative view that helps support your main endpoints,” says Cabo.
The challenge here is that payers have not historically considered the value of a medicine outside of the strict clinical parameters and a formal structure for the inclusion of value-added evidence does not yet exist.
Early discussions with HTAs to establish acceptable parameters in this respect is crucial, says Cabo. “It is really important to have early conversations with payers to establish whether or not they would consider new or adapted endpoints as valid and if they will take extra information on clinical meaningfulness into consideration when evaluating a product.”
It is the ideal opportunity, she says, to make the most of the information to be gleaned from caregivers to give an additional perspective on the benefits of a product.
The value of RWE
Real-world evidence also comes into its own when it comes to making the case with payers and the pandemic restrictions, says Hvidsten, provided a perfect opportunity to demonstrate this. “We have multiple treatments for Gaucher’s Disease,” he says. “Our initial treatment, Cerezyme, requires infusion, whereas our more recent one, Cerdelga, is oral. In the age of COVID this has been of tremendous benefit.
“The real-world evidence has shown us that in Gaucher’s disease, and in many diseases, the value of simpler administration for both patient and caregiver is huge. Frankly, payers could be reluctant to see that if they are looking at things purely in terms of clinical parameters versus cost.
“But when we raise how this has a number of real-world benefits, not only improving adherence but also changing the way patients could be cared for, this becomes much more meaningful to them.”
Demonstrating these benefits is now the challenge. Melisa Troshani, patient access public affairs head at Novartis, says partnering with all stakeholders as early as possible in the development process is key to shifting the model from one focused on pricing to a more holistic view.
Pharma is well-placed to take on a coordinator role to bring together the views of all stakeholders – patients, caregivers, policymakers, payers and regulators, she says. “If you have a customer obsession approach,” she says, “and gather insights from all the actors we will find a solution together. Pharma must be seen as a trusted partner.
“A co-creation model will help us be stronger in front of payers,” she says. “We must listen to the needs of caregivers. This will enable the right decisions to be made at each and every stage. To build trust we have to build the journey together, so patients and caregivers feel valued in the process.”