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Tools for 2020 success: 10 trends you should know in orphan drug, cell and gene therapy

2019 set the tone for innovative therapies in the cell and gene therapy space along with huge interest from Big Biotech. With an expected double digit growth trajectory in the orphan drug market, 2020 is going to be a busy year. Here are the top trends from P4A’s experts to help you navigate the year ahead and make a success of it.

1.2020 will the test year for ATMP commercialisation in the US

Sophie Schmitz, Managing Partner: The last 18 months have seen several ATMPs (Advanced Therapy Medicinal Product) gain FDA approval, namely Kymriah, Yescarta, Luxturna, Zolgensma and Zynteglo. What the manufacturers of these therapies have experienced regardless of their technology type, is an inherent lag in the time it’s taken to achieve reimbursement and uptake. So why is that?

In the US, initial CMS (Centre for Medicare and Medicaid Services) reimbursement rates of 50% for CAR-T costs have been unsustainable based on the relatively high cost of therapy and patient management, which has caused a major barrier to access and backlogs of patients waiting for therapy. From this year CMS rates will increase to 65%, but whether this is enough to manage the reimbursement gap remains to be seen.

Developing holistic pricing and access solutions that work downstream with insurers and providers to include associated hospital expenses should be a priority for any ATMP manufacturer looking to commercialise in 2020.

2. New approaches to Market Access Leadership

Akshay Kumar, Partner :To succeed in an environment where the biggest challenge is balancing affordability with enabling patient access, biopharma will have to look at transforming their market access function. Is this the year that we are going to see the emergence of the role of a Chief Value Officer – who ensures that patient access is at the heart of every decision made by C-Suite? In the very least, market access leaders who are comfortable with disruption and uncertainty, are willing to encourage innovation and take calculated risks, as well are those who are able to embed a culture of change in their organisations, will be best placed to succeed. They will have to have the ability to forge partnerships with payers, providers and patient advocacy groups and will drive cross-functional collaboration within their organisations. These leaders will be key to ensuring their organisations are building a competitive advantage by bringing innovative solutions to drive patient access for transformative therapies.

3. Innovative tech to bolster value-based contracting:

Joanna Fernandes, Associate Consultant: With the rise of one-time transformative cell and gene therapies, payers and biopharma are looking to resolve the conflict between uncertain long-term value and price. Linking payment to drug performance has been introduced as a potential solution. However, outcomes-based agreements are difficult to operationalise and implement. In 2020, we will see an upsurge in innovative digital technologies, like wearables and blockchain, to help lessen the burden in implementing these contracts. With technological advances like BioStamp and Google contact lens, regular monitoring of blood pressure, brain activity, seizure risks, and glucose levels is already possible. Usage of wearables in clinical trials has begun, with Medidata Solutions employing Fitbit to measure quality of life in Type 2 diabetes patients, and GlaxoSmithKline utilising wearables for 25 Amyotrophic Lateral Sclerosis patients. Emergence of independent third parties providing services to manage these agreements for industry and payers may also occur as a result.

4. Improvements in the R&D process:

Ciaran Cassidy, Analyst: Over the last few years there have been calls for biopharma to critically assess their pipelines and drop products that add little patient, payer and provider value much earlier in the R&D process. 2020 could be the pivotal year that sees the emergence of new R&D processes aimed at creating innovative drugs that look beyond regulatory approval milestones and instead define success as “speed and quality of patient access”. This will require the presence of cross-functional teams who can develop evidence generation strategies that are riskier for regulatory approval but creates higher value evidence for patients, providers and payers at the time of launch.

5. Basket trials: the increasing challenges payers will face in 2020

Vedika Avadhan, Analyst: The principle of grouping patients with a range of histology under the same treatment protocol, is referred to as a basket trial. With the number of basket trials increasing, this will be a growing challenge for regulatory authorities to prioritise whether these types of trials should be approved. Despite the translational benefits of implementing a basket trial, UK’s NICE hasn’t recommended Bayer’s tumour-agnostic drug Vitrakvi for routine NHS use as it was not cost efficient. This comes shortly after Germany’s IQWiG rejected Vitrakvi for insufficient data. Despite NHS England stating that last year it would prioritise tumour agnostic cancer drugs (in the same year that it has CAR-T cell therapies for cancer), the future still looks challenging for ‘game changing’ drugs from basket trials, such as Vitrakvi, to lobby for regulatory authorities to be open towards adaptive clinical trials.

6. Potential pricing pressure as non-profit organisations enter into gene therapy space

Erfan Akbraian, Analyst: Non profit organisations are increasingly investing in the development of gene therapies. The Bill & Melinda Gates Foundation and the US National Institutes of Health announced plans to invest $100 million each over the next four years towards the development of gene therapy cures for HIV and sickle cell anaemia (SCA). Others such as US based Odylia Therapeutics are investing in gene therapies for inherited retinal disorders.

In the UK, Great Ormond Street Hospital was provided a capital injection of £2.1 million into child health research projects across the UK, with one of the projects being gene therapy for children suffering from seizures. The aim is the develop more effective and less harsh treatments for children.

The increasing interest of non-profit organisations (NPO) in the cell and gene therapy space is likely to permeate into an impact on prices but when there is a development breakthrough and commercialisation.

7. US insurers to focus more on value

Max Rex, Associate Consultant: 2020 is an election year in the US, and few commentators can claim to have an idea of what the result will be. Although Democrats and Republicans cannot see eye-to-eye on most policy issues, one thing which there is at least some agreement on is the need reign-in pharmaceutical prices. We can expect the rhetoric on drug price controls and improving quality & affordability of healthcare to be major election issues on both sides of the aisle.

However, we have heard this talk before and this has not translated into major changes in federal policy, and we could be in a similar situation now. This makes now the opportune time for a third party to emerge that helps assign a value to therapies. ICER has been gaining traction recently as an increasing number of US plans are looking at their decisions to ensure they are receiving value for money when they fund a drug. Many see them as a suitable body that is most likely to position themselves as this arbiter of value, but this will depend on them moving beyond cost-effectiveness. This could involve factoring in elements around healthcare delivery quality and affordability in their decisions as these better reflect payer KPIs.

8. Increased importance of alternative payment and access models for ATMPs

Christina Poschen, Senior Consultant 2019 has seen a significant number of high profile-ATMPs and specifically gene-therapy launches with e.g. Zolgensma and Zynteglo. With that came the need for payers and regulators to adapt and reassess their reimbursement system to react to sudden high one off and often up-front costs. Manufacturers responded by trialling a variety of payment models to ease the cost burden.

These activities are expected to gain even more significance in 2020. Predominantly, three access models have emerged: annuity based payment, outcomes based payment and cross border access models. Annuity models have had little to no uptake as in the case of Zolgensma in the US whereas outcomes based agreements have been growing in populatrity. Even markets that traditionally did not engage in these models such as Germany have seen a raise in agreements for e.g. Kymriah . This trend will continue through the year ahead as manufacturers and payers are searching for ways to make the healthcare system sustainable and future proof for 2020 and beyond.

Cross border access models have also gained popularity in Europe, but have faced challenges and access delays. As therapies become more and more targeted and availability limited to certain facilities and treatment centres, cross border models will be increasing used to ensure patient access. However, stakeholders need to collaborate to implement and use available policies to ensure patients have access to innovative and state of the art therapy regardless of where they live.

9. Genomic data collaboration and privacy issues

Jack Rawson, Senior analyst : 2020 will see a greater focus on collaboration and privacy around genomic data in the EU. More sequences are being collected, at an ever-increasing rate. These sequences will increasingly be utilised by healthcare systems. The beginning of the year saw Germany sign up to the EU’s One Million Genomes Initiative, which includes 22 countries aiming to provide researchers one million sequenced genomes by 2022. This aims to address the problem of a lack of centralisation in genomic data.

There are various datasets existing in many EU countries and linking them will have great impact in facilitating precision medicine and public health. Such developments are particularly important in rare diseases and cell and gene therapy, and policymakers are increasingly aware of this.

As knowledge around the genetics of human disease increases, privacy issues will also become more important. The EU will work towards its 2-year deadline for the application of new regulations on genetic testing. Policymakers will develop guidelines that will be adopted across member states. This comes on the back of major international EU-funded research projects investigating genomics and genomic data-sharing.

10. Pan-European and mutinational HTA collaborations to rise

Nicola Allen, Associate Director: Nearly two years have passed since the European Commission (EC) proposed legislation for the future of European Health Technology Assessment (HTA) cooperation. The proposed legislation includes a joint clinical assessment, but the debate continues on whether member states will still be able to conduct national clinical assessments to inform their decision. 

While the work continues to refine the EC proposal for pan-European HTA cooperation, multinational collaborations at the sub regional level are increasingly gaining traction. In the short term, we can expect to see more country clusters emerging and consolidating activities, such as the recently signed memorandum of understanding for the Nordic collaboration, FINOSE (Finland, Norway and Sweden).

Furthermore, we can also expect these country clusters to extend their influence and eventually cross continents. For example, last month the BeNeLuxA initiative (Belgium, the Netherlands, Luxembourg, Austria and Ireland) hosted an exchange for 11 countries (including Canada) to discuss options beyond existing collaborations. Transatlantic collaborations have already proved successful. In November last year, ICER (The United States) published the results of a project with CADTH (Canada) and NICE (England) to adapt methods for assessing potential cures and other high impact short term therapies. 

Also, the relaunched NICE International which intends to establish relationships between the UK and other countries sharing knowledge to help overcome global health and social care challenges. These initiatives are likely to further accelerate HTA collaborations towards a sustained approach.

If you would like to know more about these trends, please get in touch with us at contact@partners4access.com.


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