By Marco de Morpurgo, Nicola Landolfi and Chiara Perotti
Last year was a pivotal year for the Life Sciences industry. As we had foreseen, 2019 witnessed significant technological and regulatory progress. Digitalization of the medical sector has impacted the way pharmaceutical and medical device companies approach patients, with smoother communication means that bring to zero the physical distance between patients, healthcare professionals, and the industry. In parallel, cell and gene therapies – which require sophisticated and precision development techniques – are shaking the market, increasingly establishing themselves as the “medicine of the future”. In this continuously evolving environment, Life Sciences regulation has tried (and inevitably struggled) to keep step with innovation and respond to the needs of the Life Sciences sector.
Year 2020 has just begun. What are some of the key developments that we expect to take place in the Life Sciences sector in the next twelve months?
- New technologies and increased patient centricity
The Life Sciences industry is increasingly placing patients at the center of its operations and business model. There are many reasons for this, including the feasibility to deliver more personalized treatments to patients as well as pressure from policymakers to deliver effective treatments while ensuring that healthcare systems are financially sustainable. In this scenario, new digital technologies are a key factor allowing the transition towards a patient driven system. Perhaps the best example is the changing role of patients in clinical trials: remote monitoring, virtual studies, electronic surveys on drug tolerance show how patients can get closer to their treatment path, while allowing sponsors to obtain more – and faster – data that is needed to adjust therapies from the earliest stages of product development.
2020 will be a decisive year for the enhancement of patient centricity, the use of digital technologies in the Life Sciences sector, and their regulation. On the one hand, as the industry needs to get closer to patients, companies will be engaging more and more with them – not only as groups but also individually – from the very beginning of the life cycle of new medicinal products. On the other hand, given the rapidly evolving nature of relevant technologies and analytical methods, stakeholders and regulators will need to take a more collaborative approach and set out a clear road map in order to properly tackle the challenges raised.
In a scenario where the use of digital technologies and social media lacks a clear regulatory framework, we hope that regulators will shed some light during the course of 2020. In this respect, the Italian Medicine Agency (AIFA) is expected to issue its guidelines on promotion of medicinal products shortly.
- Software as medical device under the new Regulation
Regulation (EU) No. 2017/745 on medical devices will become fully applicable on 26 May 2020. Under the new Regulation, software intended to serve a medical purpose will qualify as a medical device in its own right. This means that a number of products that were not considered medical devices under Directive No. 93/42/EEC may now qualify as such under the new Regulation. Moreover, while most software will continue to fall within Class I, some may be upgraded to Class II or even Class III. In particular, medical apps providing diagnosis or influencing diagnosis decisions will fall within Class IIa, while those impacting treatment decisions that may lead to death or cause an irreversible condition in patients will qualify as Class III devices. This up-classification will bring along additional obligations for manufacturers such as the need to involve a Notified Body in the conformity assessment procedure, and higher costs.
Tech companies developing apps relating to fitness, wellbeing and health or medical treatment must therefore consider whether their tools fall within the definition of medical device and where they fit under the new rules. To this end, companies should assess the functionality and purpose that the app intends to serve and any health claim made in relation to it. Specific attention should be drawn to the new risk classification provided by the new Regulation.
The good news for app and software developers in 2020 is that the European Parliament recently approved a corrigendum to the new Regulation granting a transition period for Class I medical devices that are up-classified under the Regulation. The corrigendum will only cover Class I devices that were CE-marked before 26 May 2020 and that require assessment from a Notified Body following the application of the new Regulation. These devices may continue to be placed on market until 26 May 2024 with no need for conformity assessment by a Notified Body, leaving companies more time to ensure that their products are compliant with the new Regulation.
- New AIFA reimbursement approaches for CAR-T therapies
In 2019, AIFA approved for the first time the reimbursement of two CAR-T therapies for cancer. Such CAR-T therapies are now available in Italy and covered by the national health system by virtue of a new reimbursement approach – the so-called payment at results.
The payment at results model aims at mitigating the financial effort of the national health system by requiring pharmaceutical companies to bear the costs incurred for patients who did not respond positively to the treatment. This model is similar to the “payment by results” approach, as it makes reimbursement conditional on achievement of the agreed results. However, in case of payment at results, reimbursement is not provided in a one-shot solution, but it is deferred in several – typically three – instalments.
Cell and gene therapies are establishing themselves as the medicine of the future and making an important step forward in patient care. However, these therapies are typically very expensive for the national health system. This requires a systemic rethinking of the current reimbursement system in order for the public sector to be able to sustain the high costs generated by these innovative treatments.
In 2020, AIFA will face the increasing challenge of striking a balance between safeguarding the universal approach of the Italian national health system and granting access to advanced therapies such as gene and cell therapies. We therefore expect AIFA to follow the payment at result approach for newly approved cell and gene therapy products during 2020, and possibly to propose further innovative approaches to medicinal product reimbursement that grant patients access to cutting-edge therapies while ensuring sustainability of the national health system.
Check out other trending topics for 2020 at this link.