Accelerating and sustaining the life of an advanced therapy through rare insight

Written by Trish Shepherd, Global Head of Business Development, OPEN Health on Wednesday 11th August 2021

This article was first published in Rare Revolution Magazine, Issue no. 013, Autumn, 2019. You can find the full magazine HERE

The healthcare landscape has seen many revolutions over the past decades. Each innovation or system is brought to us by pioneers with the hope of improving human health, tackling the unmet needs that prevent people from living life to the full. Trish Shepherd on the importance of capturing real world insights.

Each new advancement has paved the way for the next. Consider cancer: only two decades ago a diagnosis of cancer was considered a death sentence. Since the introduction of more targeted therapies coupled with sophisticated sequencing strategies and improved early detection, the outcomes are now changing, with more people surviving cancer than ever before1.

Yet unmet needs still exist in rare oncology as well as in a host of rare genetic diseases. Of approximately 7,000 rare diseases2, most are still seeking palliative, restorative or curative therapies. The complex nature of each disease along with the limited number of patients has historically compromised funding and research. Thankfully, the pioneering spirit continues, coupled with the advent of new technologies, which is giving rise to a new generation of healthcare and biotechnology companies.

With 34,000 patent applications filed over the last five years and with more than 1,000 clinical trials3 exploring the potential of new gene therapies or advanced therapeutic medicinal products (ATMPs), hope remains finely balanced. Those in the industry know all too well that success in the lab does not always translate into meaningful change in the real world. So, the question remains, how many assets will successfully navigate the regulatory gauntlet through to product launch and ultimately to a successful commercial platform?

We need to look no further than the withdrawal of Glybera, first approved in 2015 in Europe and the muted uptake of Strimvelis, approved in 2016, to see the challenges ahead. Two viable products with patient populations seeking much-needed therapy that have struggled to establish a strong commercial presence. Yet the tides are turning, and more recent entries are looking to establish strong viable candidates for the long term. Yescarta, Kymriah, Luxturna, and Zynteglo – to name the most notable are each testing the waters
with differentiated medical, patient and commercial strategies.

With so many patients still waiting, we ask what factors will help sustain the momentum to ensure the next wave of ATMP assets succeeds? OPEN Health is a family of expert practices working in partnership to enhance market access and medical communications to improve patient outcomes  – and we believe insight can make all the difference. Insights come in many shapes and sizes and can be sourced in a variety of ways. Currently working with over 50 rare and genetic disorders, we have cultivated a unique blend of experience and insights that can be shared with companies who have started their journey towards commercialisation.

1. Moments matter
Knowing the moments that matter most to patients, their parents or their carers is vital. It goes without saying that such insights carry significant value in rare diseases, because stopping therapy or having a negative first experience would have, proportionally, a greater impact compared to a non-rare disease. While companies pioneering ATMPs today are significantly more in tune with the patient journey from a clinical perspective, they are still seeking more support. Our health psychologists work with companies to help unpack the emotional as well as the physical burden of living with a rare disease. These moments can be leveraged at each hurdle towards commercialisation from clinical trial through to educating specialists about how these new therapies can be embraced with the best results. In rare disease clinical trials, every patient retained is a pivotal step towards marketing authorisation. The solutions we are exploring today connect our patient-centred outcomes teams with our patient engagement and creative teams to deliver initiatives that drive patient retention in clinical and real-world studies.

2. Data gaps can be filled
Regulators recognise the urgency for new ATMP assets to reach patients, so the number of patients treated, and the duration of clinical trial periods are reduced compared to therapies of old. This leads to a shortage of data, and in this industry this creates a conundrum. Payors, clinicians and patient advocates need data to drive confidence. The client partners we support are looking to establish long-term data registries in advance of launch to complement early clinical trial experience with real-world evidence. A deep understanding of the patient journey shapes the way we engage with key stakeholders to fill the data gaps and measure demonstrable outcomes that will cement the commercial foundation in the future.

3. Corporate reputation needs to be nurtured and protected
Companies need to work with general and industry media to maintain a positive and balanced image to ensure that they remain approachable and answerable. It's vital to invest in media relationships to ensure a company's mission to support patients and families is not overshadowed by regulatory and financial detail. Through responsible education and open communication, companies can convey to important stakeholders the complex science behind their mission, relating academic data to the real human need behind it all. Nurturing these relationships will aid responsible reporting and drive transparency around the very challenging process it takes to bring a product to market. These relationships can help protect a company's mission for the long term, even when hurdles mean early assets fall short of expectations.

4. When the system is not working, challenge it

The true value of a medicine does not come in the price alone but in the impact it can deliver for the individual, the healthcare system and the broader healthcare economy. We work with clients to help explore new measures that can and should be considered when preparing for the market access of, often, one-time therapies. Yes, we fill data gaps and draft value propositions, but this is not enough to drive success in a regulatory world that is not built for advanced therapies. To navigate new regulatory schemes requires deeper payor insights, starting with the true appreciation of the unenviable position it is to have to make decisions on whole-population value at a time of significant pressure on healthcare resources. We seek different conversations with payors which is helping us to explore new routes and strategies fit for this emerging era of medicine.

5. The right map can make all the difference 
Each rare condition comes with a unique challenge, requiring a differentiated mix of healthcare providers across a multitude of healthcare systems, with a variety of delivery and manufacturing considerations. We work with clients to map these complex pathways, identifying centres of excellence and leading specialists to ensure these assets are in the best hands. However, this is only half the battle, as people living with rare diseases do not congregate around centres of excellence or production facilities. Once we uncover our map, we go deeper to cultivate insights through co-creation or experiential initiatives that help each stakeholder truly appreciate the lived patient experience. These insights signpost how, when and where we need to connect stakeholders to ensure patients can access support with ease.

6. Informing is not enough, you need to inspire
Gone are the days of sharing information under the guise of education. So much has been discovered about adult learning that these theories must now be considered, even by the world's leading specialists. Education must now go beyond just communicating the science. It has to sustain peer-to-peer conversations. When you consider the limited number of cases presenting, shared experience from diagnosis, referral, treatment and follow-up will serve as a proxy for the various healthcare professionals throughout the patient journey. Our Medical Communications team aims to inspire change through strong thought leader partnership, compelling content, and memorable and engaging delivery. Companies that know how to develop bespoke education, connecting the multi-disciplinary teams across disparate healthcare facilities and systems will ignite these valuable discussions.

7. Branding is challenging

Let's face it, if you are going to market you will need to establish a brand. But the approach to building brand equity in the rare disease space cannot sustain nor justify the investment levels of the blockbuster brands of old. Yes, local relevance is important but never has having a global brand platform alignment been so critical. Marketers will be operating in a new value exchange where the end-user, the patient (or the parent or carer of the patient) will be more knowledgeable and demanding than other therapy area categories. The barometer for a successful brand in this territory will go far beyond healthcare professional awareness and recall of the brand. These close-knit communities will evaluate your brand on the quality of your interactions, support and true engagement. Efficient, insight-led creative campaigns that are developed in partnership with core countries or geographies will help lock down key pillars for your brand from vision and messaging to visual assets. A centralised brand platform will help control costs of excessive local adaptations allowing teams to concentrate investment on relevant engagement.

8. Leadership is a gift

Patient engagement and support will be an obvious investment. Successful patient engagement is the result of great design and planning. It removes friction from a patient's life. It supports clinical outcomes and behaviours by understanding patients' true motivations. And, ultimately; it connects patients to the things that matter most – their lifestyle, healthcare team and the people that care about them. To achieve all of this, we take nothing for granted and ensure all recommendations are pressure-tested with key stakeholders throughout development. One of the greatest lessons we have learned along the way is to start your patient engagement journey with an exit strategy in mind. Counter-intuitive you may think. However, the pharma industry's reputation has been called into question amongst advocacy groups over the years due to abandoned support programmes. Companies willing to explore beyond brand initiatives that can be gifted to relevant third parties at an agreed milestone will not only deliver solutions with greater utility for patients and carers in the real world, but also help strengthen industry and advocacy relations in the future.

9. Be proud yet humble

In the burgeoning ATMP space, many companies and therapeutic targets are new and unknown. It's important in this environment to make a noise about the achievements of a company in order to gain trust and credibility with disparate stakeholders who have minimal experience in this brave new world. Confidence and know-how are key to navigating these uncharted waters, but we must also seek to look forward, learn constantly, and be at the forefront of the market as it emerges. With no precedent to guide us, a broad perspective and sensitivity to trends is fundamental to protecting the future pipeline in this uncertain time where invention has outstripped regulatory frameworks. Or, as one unnamed participant at the JP Morgan Healthcare Conference 2019 had it, "Star Wars medicine in the age of Flintstones reimbursement".

*This article was first published in Rare Revolution Magazine, Issue no. 013, Autumn, 2019. You can find the full magazine HERE


1. The BMJ:
2. America's Biopharmaceutical Companies:
3. Getting Ready: Recommendations for Timely Access to Advanced Therapy Medicinal Products (ATMPs) in Europe report from Alliance for Regenerative Medicine,

It is a challenging task, but we know that pioneers do not give up easily.

With the right support to navigate the hurdles that no doubt lie ahead, more and more ATMP assets will reach the intended patients and we will see yet another revolution in healthcare. I, for one, am very excited about the proposition of taking part in this journey. We invite you to get in touch today to see how the collective experience and rich insights that exist within OPEN Health can get you and your team where you want and need to go. 

At OPEN Health, we bring together deep scientific knowledge, global understanding and broad specialist expertise to improve health outcomes and patient wellbeing.


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