Summary by Zoya Marinova
Looking back at the most popular discussion forums led by experts in pharmaceutical law and regulations across the week, Zoya Marinova highlights the key points raised.
This popular forum explored European challenges in drug pricing and accessibility. The panel included:
Sophie Pelé initiated the discussion by highlighting the challenges related to transparency of medicine pricing mechanisms at the European level.
Transparency in the EU legal framework is defined in the Transparency Directive 89/105; however, the pricing mechanism is not harmonised on the EU level.
In this context, Christian Jervelund described the traditional price-fixing mechanism, in which negotiations often focus on the price, whereas the outcome is assumed based on clinical trial data.
However, when payers focus only on the price, they are taking a huge risk in terms of outcomes, especially for rare diseases.
As an illustration of the discussed processes, Ariana Greco presented considerations regarding the drug pricing of an Alnylam’s orphan product based on RNA interference.
Challenges during product development include its long duration and gaps in knowledge regarding the disease, patient population and endpoint selection for clinical trials.
Therefore, success during the market stage becomes particularly important for biotech companies.
Next, Pelé explored how value transparency can facilitate conditional pricing, faster access to medicines and adaptability and foreseeability by both parties.
To operationalise value-based agreements, there is a need to understand the risk for all stakeholders.
Jervelund emphasised the need to bring the outcome back into the price negotiation. To help assess the actual risk, transparent models have been developed that simulate how different outcomes will affect the cost.
Further, Jervelund underscored the importance of changing the incentive system from focusing solely on the price to better use of resources.
Next, Greco presented challenges leading to delayed time to access.
They include skepticism in discussions with payers, infrastructure limitations, privacy concerns, administrative and individualisation burdens and difficulties in measuring, tracking and assigning causality to outcomes.
She believes that a partnership with a new way of thinking on both sides is needed to achieve value-based agreements.
Jervelund also stressed the importance of partnerships to track efficiencies during a patient’s treatment, which may identify important cost reductions.
The panelists were asked by moderator Jordi Faus whether they believe the increased reliance on real-world data by regulators will be an asset with regards to pricing.
They agreed that real-world data use may greatly impact pricing, and that the interpretation of the real significance of endpoints will be key.
Click here to watch the full discussion on demand.
In this discussion, legal experts discussed the new rules and guidance for AI, eHealth and mobile apps within the healthcare industry under the EU MDR. The panel included:
Giorgio Rizzello started by emphasising the significance of artificial intelligence (AI) in our life and its potential to transform society.
Notably, the EC has focused on AI by launching its AI Strategy in 2018 and both the EC and European Parliament (EP) have provided further guidance.
In this context, Toby Bond presented examples of current AI applications in healthcare, relating to diagnostics, disease monitoring, surgery and non-clinical applications.
The panelists noted the complexity of AI diagnostic tools development, the vital importance of collaborations between AI technology providers and health care providers (HCPs) and the significance of modules developed around data sharing.
Legal issues that may arise around AI development include data privacy, cybersecurity, technology procurement and collaboration issues. Moreover, one should also consider the medical devices regulation (MDR).
The moderator Jordi Faus asked the panelists how they view the regulatory framework for digital health offerings.
Both believe that the existing framework relates differently to different players in the field. Rizzello emphasised the indispensability of guidelines by regulatory authorities to prepare for the new regulatory framework.
The next question by the moderator Mr. Faus concerned the threats and opportunities which AI brings into the healthcare sector.
Rizzello believes AI will facilitate data sharing and interpretation and will mitigate the scarcity of healthcare professionals, data and time.
He envisions challenges with regards to trust and reputation.
Bond thinks a three-way balance is needed: addressing risks from a regulatory perspective and maintaining trust in the system while not stifling its development. He envisions massive opportunities in healthcare digitalisation.
To him, a challenge is respecting the European values in AI development while staying competitive on the global market. Notably, the EU is pushing forward to an European Data Strategy to strike this balance.
Finally, Faus asked the panelists how they see the impact of the upcoming MDR implementation on the digital health sector, and how they envision the market in the next 5–10 years.
Rizzello described the numerous collaborations between small start-ups and large MedTech companies, rapid innovation and emphasis on medical technologies. He expects robotics and AI to be embedded in the MedTech sector in 5–10 years.
Bond believes that the MDR will bring more challenges to the market. He expects massive adoption of AI technologies within 10 years and new legislation related to AI liability, updating and reassessment.
This discussion revealed insights on the latest rules around compassionate use of unapproved and off-label products. The panel included:
The panelists examined the hospital exemption, the rise in the use of the compassionate mechanism, recent developments regarding unapproved and off-label products and compounding and associated legal challenges.
Catherine Longeval stated that the hospital exemption is the only exception to marketing authorisation (MA) for advanced therapy medicinal products (ATMPs). Hospital exemption for an ATMP can be obtained under strict conditions and implementation requirements.
Its intended benefits are ongoing medical research, gaining clinical experience and addressing unmet clinical needs.
Potential issues include undermining MA of ATMPs with the same indication or market exclusivity of ATMPs with orphan status, impact on cohort size for clinical trials, absence of information exchange and lack of clinical data.
Longeval emphasised that the COVID-19 pandemic has resulted in increased compassionate use, “temporary exemptions” from MA requirements and off-label use.
Positive aspects of compassionate use include facilitating patient access to new medicines and gaining clinical experience, whereas the diverging and complex national regulations are challenges.
Hilary Jones noted the very high volume of requests for compassionate use during the COVID-19 pandemic and emphasized that compassionate use should not slow down the clinical trials.
The panelists discussed also recent developments regarding unapproved and off-label use. The “temporary exemptions” use is not subject to civil or administrative liability except in cases of a defective product.
Jones summarised that in a pandemic a variety of different regulatory mechanisms operate for the supply and use of unapproved products.
They depend on each country’s national law, approach and needs, and necessitate case-by-case discussions and robust communication with the national competent authorities, EMA and EC.
Longeval presented the WHO and EC guidance on off-label use of medicines for COVID-19. The panelists reminded us that suspected adverse events must be reported.
The next topic of discussion was compounding (pharmacy preparation).
Compounding is regulated at the member state (MS) level and has regained significance during the pandemic due to the strain on healthcare budgets and medicine shortages. Challenges that can arise with compounding include GMP compliance, pharmacovigilance and no patient information or even consent.
Finally, the panelists shared their thoughts on challenges faced by the industry with regards to unapproved products.
There are questions how the use of unapproved products ultimately affects medicine access and availability. Moreover, the non-compliant use of exemptions has the potential to stifle innovation.
In this discussion, industry experts discussed legal compliance in promotional and marketing activity across digital channels and virtual congresses.
This session covered disease awareness campaigns, promotion through digital channels and virtual medical and scientific congresses.
Ilja Moree emphasised that disease awareness campaigns (DACs) should not reference medicinal products, even indirectly, and should not have promotional intent.
Practical tips for DACs include being balanced and objective, using factual and neutral wording and scientific language, not referring to data for a particular product, driving the DAC preferably by a medical function, not targeting exclusively future prescribers and obtaining internal approval as a non-promotional material.
Adela Williams reviewed promotion through digital channels, stressing their significance during the COVID-19 pandemic.
She noted that promotion through digital channels is subject to the same regulations as any other type of promotion, but its use raises some specific challenges.
Notably, transparency is essential. The EFPIA has suggested to consider the objective and type of content, intended audience, channel standard set-up and updating and maintenance of the content.
Digital communications with HCPs are generally not permitted, unless the recipient has previously given permission and should be accompanied by prescribing information.
Promotion of prescription-only medicines (POMs) should be limited only to HCPs. Regarding user content, the EFPIA guidance explicitly states that inappropriate comments should be monitored, moderated and/or deleted.
Moree discussed virtual medical congresses, which are especially important during the COVID-19 pandemic.
The EFPIA/IFPMA guidance for 2020 clarified that, in the context of virtual congresses, the code should be considered from the region where the majority of attendees is expected to come from.
Additional considerations for virtual congresses include stating the label by which promotional materials were developed, informing in which countries the product is registered, restricting access to promotional materials to HCPs only and providing the prescribing information.
The audience queried Moree how she views promotion of medical congresses over social media.
She believes that the answer depends on the information provided over social media; as long as the post in social media is not promotional, and the general public cannot access information intended for HCPs only, this should be acceptable.
The next audience question addressed possible avenues to share clinical trial results in light of their problematic publication on Twitter.
According to Williams, balanced and non-promotional information can be provided to shareholders and investors via press releases or on a section of the website dedicated to this target audience.
Moderator Jordi Faus completed the session by underscoring the importance of continuous education in this challenging area of law and the goal of working toward solutions that promote successful patient treatment and disease cure.
