My day-to-day role consists of listening to, and interacting with, patients and learning about their daily lives, experience of their condition, and their hopes for their health.
It’s my job to transmit those perspectives to my colleagues at the company, so that our work on researching, developing and bringing new medicines to market is truly in line with patients’ needs and perspectives.
Of course, this kind of role is still new within the pharmaceutical industry, which means we are still learning about how best to listen to, and serve, those needs. Our company is focused on understanding the real experiences of those affected by neuromuscular and neuro-ophthalmological diseases.
My role helps me and my colleagues put ourselves in the shoes of the patients. For me, this is a little easier than for most people, as my sister has been living with a serious, severe and life-threatening rare metabolic disease for 37 years.
One key insight from that personal experience is that open and transparent communication with patient groups from the earliest stages of drug development is vital.
I believe this ongoing process is helping everyone in our organisation to bring medicines to market that better reflect patients’ needs.
People often ask me about a potential conflict between patients’ requirements and the industry’s need to make a profit. To me, it is possible for patient centricity and profitability to go together – but only if we look to the long term. If a company is focusing on what is most important for patients, we will be able to help as many of them as possible. This will lead to better medical outcomes and quality of life for patients and can also create value for all stakeholders. Or, as our chairman emphasises: “Patients first and the business will follow.”
Rare diseases present unique challenges, but these can be overcome with passion, commitment, collaboration, respect and accountability – our corporate values.
Our mission is to develop treatments for rare neuromuscular and neuro-ophthalmological diseases that currently lack treatment options. These include Leber’s hereditary optic neuropathy (LHON), Duchenne muscular dystrophy (DMD), congenital muscular dystrophy (CMD) and primary progressive multiple sclerosis (PPMS).
We have dedicated more than a decade to researching potential treatments for these often overlooked, life-altering diseases.
My job is to raise awareness across functions about the value of incorporating the patient perspective in every project and activity we set up to drive better patient outcomes. I would describe Santhera as a proactively curious listener to what really matters to patients, their family members and those working on their behalf.
That’s why our motto is ‘their future – our focus’.
More patient-centric leadership positions are needed in pharma to ensure patient views are seen and heard more prominently.
Patient voices will then be able to influence the full breadth of activities within the sector, ranging from research and development to regulatory, medical affairs and market access.
We need to find ways to allow an exchange of experiences and information across internal functions and company affiliates. At Santhera, we recently set up the ‘Patient Engagement Workshop’ with the aim of opening up conversations about what activities are already being done at headquarter and regional level, and then find best practices that can be transferable across our affiliates.
Traditionally, medical doctors were seen as the main customer for the pharma industry. Nowadays, there are different stakeholders: regulators, payers, researchers, investors and, of course, patients. With the rise of the ‘patient revolution’, we need to introduce patient-centric goals and metrics at a corporate and individual level, throughout the company. Only then can we be sure that this new mindset has been fully embraced.
How far does pharma dare to go with this change? Perhaps we could think about including patient opinion leaders in the executive management teams of pharma companies.
Another change is to boost communication with patients much earlier in the development process, because input from patients on their disease journey may give insights to identify better research priorities and to address the design and planning of clinical trial protocols.
There are limited standard operating procedures (SOPs) and recognised good practice guidelines for patient centricity. That’s why we need frameworks to inform when, where and how to integrate the patient voice in a meaningful and consistent way across drug development.
I have tackled this question at Santhera by considering existing resources like the European Federation of Pharmaceutical Industries and Associations (EFPIA) Code of Practice on the Relationship between the Pharmaceutical industry and Patient Organisations, the EURORDIS Code of Practice between patient organisations and the healthcare industry, and the European Patients Academy on Therapeutic Innovation (EUPATI) guidance on patient involvement in industry-led medicines R&D.
I also drew on the Consensus Framework for Ethical Collaboration from the International Alliance of Patients’ Organisations (IAPO) and the Pillars of a patient-centred company set by the European Patients’ Forum (EPF).
I represented our company in the Patient Focused Medicines Development initiative and had the chance to give feedback on the usability and content of the Patient Engagement (PE) Quality Guidance. This tool will measure the level and quality of PE activities within organisations and compare different PE projects and their impact.
There is currently no standard way to assess the impact of patient centricity across drug development. However, some organisations are taking the lead. For example, the Clinical Trials Transformation Initiative (CTTI) developed an approach to estimate the financial value of patient engagement for a typical oncology development programme entering phase 2 or phase 3. DIA also led a study entitled ‘Capturing the Value of Patient Engagement: Summary of Results of the 2016 Study Quantifying the Adoption and Impact of Patient-Centric Initiatives’. The industry has the chance to develop key performance indicators (KPIs) that will provide evidence to show that integrating the patient voice really can reduce costs and better serve patient communities.
Organisations like the CTTI and the Patient-Centred Outcomes Research Institute (PCORI) have been soliciting patient input to define meaningful study endpoints and establishing broad guidelines for patient engagement, respectively.
In the case of rare diseases, several members affiliated to EURORDIS are involved in key European Medicines Agency committees, helping to reshape regulatory affairs and the orphan drug development process.
Furthermore, the European reference networks (ERNs) for rare diseases will change the landscape of many rare diseases by creating a clear governance structure for knowledge sharing and care coordination across the EU. There are also several services that focus on changing outcomes for patients by improving the patient experience through therapy and improving quality of life. For example there are programmes and tools to improve adherence to medication, devices to monitor the patient condition under treatment and training/tools to help physicians prescribe the right drug to the right patient.
I believe we are on the right track to fully integrating the patient experience into the pharmaceutical industry’s working practices – and this can only help to bring more badly-needed treatments for rare conditions to patients.
But we still have a long way to go. It is sometimes easy to lose sight of patients and their needs in our daily working lives. The solution to that is straightforward: tap into your organisation’s resources, and simply stop and listen again to what patients are telling us.
Dr Vanessa dos Reis Ferreira is responsible for working with European patients organisations at Santhera Pharmaceuticals. She has a unique perspective from having been a rare disease advocate that founded a non-profit organisation. She has been a researcher, a lobbyist representing rare disease patients within patient organisations such as EURORDIS, and she joined industry several years ago with the goal of accelerating orphan medicines development.
Dr dos Reis Ferreira is uniquely positioned to ensure patient groups’ insights are integrated into programmes and projects from conception and across departments. She facilitates rare disease patient communities to connect, increases awareness with resources, helps identify and share best practices across countries and improves the capacity-building of different patient groups.
She has been a deputy at the Health Parliament Portugal, developing political solutions to put patients at the centre of decisions on the future of health in that country.
She has aided national and European rare disease patient organisations by: establishing an international patient-driven network for research and orphan drug development; creating the World Congenital Disorders of Glycosylation (CDG) Awareness Day, and organised international and national conferences for rare diseases. She has also developed educational and empowerment programmes targeted at patients, patient groups, researchers and medical opinion leaders.
Dr Ferreira holds a PhD (Sc.D) in Cell and Developmental Biology and an International MBA from IAE de Paris, Sorbonne Graduate Business School.