The impact of the COVID-19 pandemic will be felt in the pharmaceutical and healthcare industry for decades to come. With shifting care models, new and improved assessment processes, and the development of innovative medicines, it is evident our industry is rapidly evolving.
So, what does 2022 hold for pricing and market access (PMA)?
We believe that these are the 5 key PMA trends that could shape the industry in 2022:
- EU Net Price Disclosure: Industry Verses Payers, But Who Will Come Out On Top?
- The Rapid Development of a Joint EU HTA. What does this mean?
- The Expansion of Cross Border Collaboration for Competitive Pricing Negotiations and Accelerated Access of High-Cost Therapies
- Effective P & R Processes to Ensure the Future of Gene Therapies in the EU
- Continued and Increasing use of Post-Marketing Evidence Collation to Justify Value in Market Access
EU Net Price Disclosure: Industry Verses Payers, But Who Will Come Out On Top?
As a result of the ever-growing budget constraints faced by payers, the pharmaceutical sector has witnessed growth in the number of confidential managed entry agreements negotiated between payers and manufacturers. This is supported by a 2020 survey carried out from the EURIPID executive committee which identified that out of 22 EU states, all reported the use of confidential discounts.
To counterbalance the manufacturer’s market power on patent products, this has spiked recent calls for net pricing transparency, particularly by the WHO and some individual EUY states. For example, in 2020 the WHO published the Oslo Medicines Initiative which outlines a new vision for collaboration between governments and industry to provide equitable and sustainable access to innovative medicines across the EU. Similarly, the Italian medicines agency, AIFA, updated its dossier requirements earlier this year which request that manufacturers provide information on product prices in other EU countries and the existence of any negotiated discounts and/or confidentiality agreements.
So, what do we expect will happen over the course of next year? Will other EU states follow AIFA or will industry fire back to maintain the current status quo?
- As a result of the increasing political push towards transparent net prices, we expect that the WHO will power up efforts to convince the pharma industry that disclosed prices will lead to a reduction in the cost of medicines
- We may also see the WHO gearing up the implementation of the Oslo Medicines Initiative and pushing towards greater collaboration and transparency between healthcare governing bodies and industry
- However, it is likely that industry players will push back, citing that the risk of uniform pricing is too great and will undermine current differential pricing which helps to ensure that higher income countries pay more for the same drug than lower income countries
- Nevertheless, because of mounting political pressure and increased budget constraints faced by payers, it seems inevitable that we will begin to see a shift in payers requesting more and more information related to net price disclosure within HTA dossiers.
- There is potential that the status quo between payers and manufacturers may shift as a result. However, over time, it is likely that things will settle back to normal as manufacturers play the ever-evolving game of meeting increasing payer demands to secure access to lucrative EU markets.
The Rapid Development of a Joint EU HTA. What does this mean?
A joint EU HTA, a long-term goal of the EU to align the HTA processes of all member states into a single process, now seems to be closer than ever. Over the next decade, the regulation will make it compulsory for manufacturers to submit a European-level HTA dossier for joint clinical assessment. A clear roadmap has been set out, with key stakeholders designated (e.g., EUNetHTA21 responsible for developing advanced HTA methodology) and obligations for member states.
It is anticipated that these joint HTA assessments will reduce the duplication of work for both local HTA agencies and pharmaceutical companies, resulting in faster patient access. Although no decision has officially been made now, the provisional agreement states that a Member State Coordination group will be created, requiring each EU country to delegate a representative to it and its subgroups.
The Coordination Group will develop and adapt methodological and procedural guidance on joint work as well as develop specific methodology where appropriate for medicinal products, medical devices and in vitro diagnostic products (IVD). The subgroups will involve experts from national and regional HTA agencies who will be responsible for conducting the clinical assessments as part of HTA assessments or scientific consultations. The European Commission will also establish a stakeholder network, which will support the work of the Coordination Group and its subgroups.
Moving forward, The European Parliament plenary vote will likely take place in December and the EU HTA Regulation will likely come into force by Q1 2022. It is expected that the coordination group and the first subgroup on methodology by will likely be set up by the end of 2022 and the stakeholder network will hopefully be operational from 2023. In addition to this, the Coordination Group will start drafting guidance documents over the next two years. The implementation of EUnetHTA joint health technology assessments will be a continuous process which will take time and that is why, they will be implemented in phases:
- 2025: Joint HTA assessments will be initially conducted for cancer medicines, ATMPs and Medical Devices/IVDs
- 2028: Joint assessments will be extended to orphan medicines
- 2030: All centrally authorised medicinal products will undergo joint HTA assessments
- The implementation of joint HTAs will be initiated and one of the first steps associated with that will be the entry into force of the European HTA Regulation in Q1 2022
- The adoption of an international HTA would entail some structural reorganisation in pharmaceutical companies as they would need to start thinking who will be responsible for the planning and development of these dossiers
- Until now, HTAs have been managed by local affiliate teams, whereas moving forward, centralised joint HTAs are likely to be managed by global teams
- Depending on the clinical and economic requirements of the joint HTA, pharmaceutical companies may need to change their evidence generation strategy, which would necessitate re-planning of future clinical programs, providing training on the joint HTA requirements to global teams etc.
- Global HTA teams will likely take some of the responsibilities of the local teams. Good communication between teams will be crucial to ensure no duplication of work
The Expansion of Cross Border Collaboration for Competitive Pricing Negotiations and Accelerated Access of High-Cost Therapies
Collaborations between smaller countries are being created and implemented to support the evaluation of new treatments, especially those of high cost or which treat orphan diseases. The aims of these initiatives are to shorten the time to market, increase the geographical access, and to negotiate for more competitive prices of new products.
Some examples of cross boarder collaborations include:
- BeNeLuxA between Belgium, The Netherlands, Luxembourg, Austria, and Ireland
- FiNoSe between Finland, Norway, and Sweden
- Nordic Pharmaceutical Forum between Iceland, Norway, Sweden, and Denmark
- The Visegrád Group between Czech Republic, Hungary, Poland, and Slovakia
- The Valletta Declaration between Cyprus, Greece, Italy, Malta, Portugal, Spain, Croatia, Ireland, Romania, and Slovenia
So far much of the success for cross-border collaboration has occurred at the HTA level. With noteworthy achievements from BeNeLuxA in the joint assessment of Zolgensma and Spinraza. However, collaboration on pricing negotiations has been limited, this may have arisen from differences in GDP and healthcare spending per capita and hence ability to pay as well as variability in what is deemed cost effective. Exemplified by the end of joint negotiations between Belgium and Netherlands on Orkambi as the countries failed to reach an agreement on what would be a cost-effective price.
- Due to the success of some initiatives, we may expect this to entice other countries to join existing initiatives or to form their own initiatives. For example, earlier this year BeNeLuxA and the Nordic Forum released a joint statement outlining plans for collaboration.
- The number of joint HTAs are expected to rise due to continued and increased pooling of resources between countries. As cross border collaborations mature, we also predict an increasing assessment speed as the joint assessment models are further established.
- Additionally, it is likely that some of the more recent initiatives, such as The Visegrád Group or The Valletta Declaration, could begin pilot joint assessments and pricing negotiations. It will be interesting to watch over these collaborations to determine how successful they will be as it could set the stage for further new initiatives.
- Cross border collaborations are expected to try and align to develop methodologies such as an agreement on a pricing framework which considers the different levels of willingness and ability to pay of the member countries. This may allow for successful collaboration of further processes such as pricing negotiations and more efficient procurement, improving the affordability of innovative therapies.
- Increases in collaboration between countries could mean a reduction in the complexity of submissions for high cost and/or orphan therapies for manufacturers and so we may see timelier patient access for new drugs.
- However, reports have warned that submissions will have to be broad to appease multiple countries rather than tailored to one countries ideals and logistically different countries work in different languages, have different timelines, and have different confidentiality laws. These hurdles could overall lead to slower access. For this to be overcome governments may need to relax some of their national requirements in the future to allow for greater flexibility.
Effective P & R Processes to Ensure the Future of Gene Therapies in the EU
Central to much of the debate around Advanced Therapy Medicinal Products (ATMPs) are their premium price tags, clinical uncertainty, and assessment challenges. However, in a clinical landscape that continues to shift towards personalised medicine the requirement for these innovative technologies seems more relevant than ever.
As outlined in our previous article, the case of Zolgensma’s entry across some EU markets demonstrates that existing drug assessment frameworks are not suited to ATMPs. Further, the mix of approaches taken shows that the EU has made little progress thus far at implementing new performance-based risk-sharing funding models as seen in the US.
Showcasing these market access challenges in Europe is the case of Bluebird’s Zynteglo gene therapy where ultimately a lack of value recognition led to its exit from the European market after failing to secure access across various governments.
Despite the extensive challenges faced for gene therapies looking to secure access, some markets are showing promising innovation. Germany, for example, appears to be at the forefront of intuition with the annuity model utilised for Zolgensma and combined annuity plus risk-sharing model used for Zynteglo, which resulted in approval for both therapies under orphan drug status. Whilst a post launch real word evidence generation was implemented for Zolgensma, a breakdown in pricing negotiations saw Zynteglo’s withdrawal from the German market. The latter highlights the inability of current assessment frameworks to recognise the value of ATMPs, despite approval failure to reach pricing agreements limits patient access to the benefits of innovative treatments.
- We may see collaborative action taking place amongst payers, HTA bodies, patients, regulators, and developers to ensure agreements on clinical trial design, manufacturing, and equality in access both for patients and resource allocation are made. This is fundamental to reduce developer uncertainty and increase efficiency of the Pricing & Reimbursement (P&R) process for gene therapies.
- To improve valuation of benefits for ATMPs, conditional marketing authorisation and reimbursement mechanisms may begin to consider the societal perspectives alongside long-term efficacy and safety in post-authorisation follow-ups.
- To transform current expenditure into investment implementation, as seen thus far in Germany, annuity-based modelling could start to be favoured over outcome-based managed entry agreements (MEAs).
Continued and Increasing use of Post-Marketing Evidence Collation to Justify Value in Market Access
In recent years, the generation of post-marketing evidence has become an increasingly common practice, most often in the form of real-world evidence (RWE) although randomised clinical trials (RCTs) may also be used. A commitment from manufacturers for post-marketing evidence and RWE collation may be a pre-requisite to initial PMA approval and market access, as well as playing a role in potential reassessment or indication expansions.
This year, we have already seen many HTA agencies bring in new policies for the use of post-marketing evidence collation, and RWE generation, which form the basis of our trends. In the UK, NICE announced their new Innovative Medicine’s Fund, which will require two years of post-marketing evidence collation (either RWE or RCTs) as part of a managed access arrangement for eligible products, a model already used by NICE’s Cancer Drugs Fund. RWE is also a central pillar of the NICE methods review, which is currently out for consultation. The review lays out plans for increased use of RWE, and a framework for ensuring it is standardised and of high quality.
Elsewhere, in France, after launching its Action Plan back in 2020 HAS released new guidelines for RWE Methodology. They have committed to issuing conditional reviews, where uncertainties in data exist, that will rely on post-marketing evidence, and aim towards greater transparency in post-launch studies. In Germany, following a 2020 commitment to mandate the collection of post-launch RWE and an IQWiG review into scientific concepts of RWE generation, this year saw the first drugs have post-launch evidence collation mandated by the G-BA, the very first of which was the gene therapy Zolegnsma.
On the subject of Zolgensma, 2021 also saw it launch globally as one of the (if not the) world’s most expensive drug. Its access in Europe and the UK relied heavily on the promise of generating post-marketing evidence, with commitments including data-collection registries, managed entry agreements and payment-by-results plans.
- As seen over the last two years, we expect a continued increase in requests for post-marketing evidence collation from HTA agencies, for both initial price and access discussions and reassessments
- In fact, it is quite possible more agencies will release new plans and protocols for the collation of post-evidence data as seen by NICE, HAS and the G-BA, particularly centring around the methodology and standards of RWE generation
- Further to this, the NICE methods consultation will be finalised, with next steps implemented next year; as it is unlikely significant changes to be made to the draft review, we expect to see RWE become a more central part of the NICE process as their plans to integrate RWE into appraisals, standardise its methodology and ensure high quality, are upheld
- Finally, with over 360 cell and gene therapies currently in the pipeline, we expect to see more appraisals like that of Zolgensma, with mandated post-marketing evidence collation required to support initial price and access discussions, and regular reassessments, to justify continued access and the high price-tag of such products.
- America’s Biopharmaceutical Companies. Medicines in Development, 2020 Update: Cell and Gene Therapy (2020) https://phrma.org/-/media/Project/PhRMA/PhRMA-Org/PhRMA-Org/PDF/A-C/MID-cell-and-gene-therapy-2020.pdf [Accessed 06/12/21]
- Bulaklak, K., Gersbach, C.A. The once and future gene therapy. Nat Commun 11, 5820 (2020). https://doi.org/10.1038/s41467-020-19505-2
- Van Overbeeke, E et al (2021) Market Access of Gene Therapies across Europe, USA and Canada: Challenges, Trends and Solutions, Drug Discovery Today, Volume 26, Issue 2, Pages 399-415. https://doi.org/10.1016/j.drudis.2020.11.024
- Ronco, V., Dilecce, M., Lanati, E. et al. Price and reimbursement of advanced therapeutic medicinal products in Europe: are assessment and appraisal diverging from expert recommendations?. J of Pharm Policy and Pract 14, 30 (2021). https://doi.org/10.1186/s40545-021-00311-0
Do you agree that these are the key trends for 2022? Do you anticipate other topics will be more important?
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