Strength in Numbers
R&D innovation is being accelerated through the nurturing of a ground breaking public-private relationship
The HARMONY – Healthcare Alliance for Resourceful Medicines Offensive against Neoplasms in hematologY – has a seemingly simple aim; to use big data to improve the care of patients with blood cancers. By gathering, integrating and analyzing anonymous patient data from high-quality sources, the project – jointly funded by EFPIA and the European Union under the Innovative Medicines Initiative – has united seven pharmaceutical companies with more than 40 universities, research organizations, patient organizations, public bodies and non-profit groups to help define patient outcomes and improve decision making for policy makers, payers and clinicians.
We spoke to EFPIA Project Lead Tay Salimullah, Director, Global Market Access, Cell & Gene Therapies at Novartis Oncology, and EFPIA Co-Lead Pam Bacon, Executive Director, Medical Affairs at Celgene International.
Why is the HARMONY collaboration important?
Salimullah: Some of the challenges we face are so significant that they cannot be solved by a single company. With the emergence of single-arm trials, smaller patient populations, as well as the drive towards combinations and potentially curative therapies, and the lack of survival endpoints at the time of label discussions, we have a situation that could result in a restricted label or access. Ultimately, patients with relapsed refractory forms of blood cancers may have to wait for these life-saving therapies.
One company can’t solve all of this on its own, yet, any discussion on new endpoints have to occur internally and externally – internally, between Access, Medical and Marketing, and externally between public and private partners.
Bacon: Big data is a big topic right now, and we now have the computer power to look at it in a very different way. There are, of course, challenges around data ownership but we hope that projects such as HARMONY can bring different datasets together and apply the power of big data analytics to search out biomarkers, risk factors, etc. that you cannot observe in a single dataset. It offers a much wider potential than working in isolation.
How could the HARMONY project lead to the acceleration of R&D innovation?
Bacon: If you have a large set of data, whether it is genomic data or clinical data, we may be able to identify signals that tell us which patients will do well and which may not. This will then give us a massive head start in knowing which pathways to target, where a product may have most effect, and which patients will gain the most benefit. There is definite future potential in R&D innovation and acceleration once the first analyses emerge.
Salimullah: R&D innovation will ultimately be a side effect of a project like HARMONY, but right now it isn’t the goal. It has been established to accelerate access for patients to new drugs and while one of its aims is to define and align with a new set of outcome measures, another specific aspect is to have direct input from patients, payers and regulators into this. The starting point is to focus on improving patient outcomes and drive healthcare efficiency.
Why is it important for all stakeholders to agree on endpoints and outcomes?
Salimullah: Outcomes are a powerful lever to unlock value-based healthcare systems. The first step to enabling outcomes-focused systems is to define the outcomes that matter to patients, for example, with prostatectomies, it is important to consider outcomes such as incontinence or erectile dysfunction not just survival, which can have a significant quality-of-life impact for patients. Outcomes cannot be defined by one stakeholder group alone, as all perspectives need to be considered (patients, regulators, payers, HTAs, pharma, etc.) for them to be meaningful and relevant.
What impact will the project have on companies’ use of real-world data?
Salimullah: There is a lot of buzz around real-world capability and big data, but this is a really tangible example of a project that you can measure and define clearly. Companies will do more and more of these types of collaborations to build their real-world capability; spreading the risk across several partners makes it more attractive.
Bacon: HARMONY is a big data project including real-world data but is not just a real-world data project, it’s much bigger than that. People can get hung up on using real-world data but the trick is going to be combining the best sources of data – a mixture of clinical trials, genomic studies, preclinical studies and real-world data – to understand how actual patients are treated. HARMONY intends to do all that.
How will these patient insights influence pharma company patient access strategies?
Salimullah: HARMONY will allow us to understand, a) the outcomes that matter to patients, and, b) how treatments respond in the real-world setting. This is critical for our access strategies too as we seek to demonstrate the value and efficiency of medicines to HTAs/payers as well as meet the increasing demand for transparent outcomes data. These insights will also help find new ways to incorporate RWE and outcome measures in regulatory/HTA processes, through collaboration with European stakeholders, and facilitate negotiation of outcomes-based contracts with payers, ensuring ‘right medicine for the right patient at the right time with the right outcome’. Ultimately, this will all pave the way for faster access to innovation for patients with significant unmet needs in hematology.
How will such insights help patients gain faster access to innovative new medicines?
Salimullah: HARMONY will deliver insights from data of thousands patients across Europe, helping to optimize treatment for patients and enabling payers to make more holistic decisions. Only 3% of cancer patients enroll into clinical trials; we are interested in unlocking insights from the other 97%.
What are the challenges of a participating in a public-private partnership?
Bacon: We are a very large consortium, with 51 partners, 44 of which are public institutions or specialist companies, as well as seven pharmaceutical companies. Industry often collaborates with academia but normally we are working on our projects, or we are working on theirs. The challenges is going to be making this work with such a large number of partners to coordinate, but also, as this is a public-private joint project, it is essential to get the right balance in the partnership. It is important to have a common understanding about the tasks and roles of each and everyone in the project.
Salimullah: While collaboration is becoming more popular, a public-private partnership allows us, as an industry, a neutral platform to have a discussion within the wider access and regulatory ecosystem. When they see seven pharmaceutical companies working together on a common goal, that allows us to build trust as they realize this is an industry-wide agenda.
Tay Salimullah and Pam Bacon will be speaking at eyeforpharma’s 3rd annual Access Europe Summit in Amsterdam on 25-26 April. For more information, visit https://goo.gl/ZAM8yV
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