Strasbourg, 31 October 2023                                                                       CDBIO (2023) 29

STEERING COMMITTEE FOR HUMAN RIGHTS

IN THE FIELDS OF BIOMEDICINE AND HEALTH

(CDBIO)

EQUITABLE AND TIMELY ACCESS

TO APPROPRIATE INNOVATIVE TREATMENTS AND TECHNOLOGIES

IN HEALTHCARE

Note from the Bureau

Summary of the methodology adopted by the Drafting group,

outcomes, conclusions, and possible future approaches

Prepared the Secretariat

Adopted methodology

The discussion paper CDBIO (2023) 25 on “Equitable and timely access to appropriate innovative treatments and technologies in healthcare” has been prepared by the drafting group entrusted by the CDBIO at its 3^rd plenary meeting in June 2023, to examine how the general and procedural principles of Recommendation CM/Rec(2023)1 also apply to the decision-making processes ensuring equitable access to innovative treatments and technologies.

With this purpose and in accordance with the scope defined in Article 2 of Recommendation CM/Rec(2023)1 on “Equitable access to medicinal products and medical equipment in a situation of shortage”, the drafting group focused on access to innovative treatments and technologies used for the medical care of patients with serious or life-threatening health conditions, certified through an appropriate regulatory process while considering that the access to innovative treatments and technologies in healthcare is usually restricted by limited supply capability or budgetary constraints.

In its work, the drafting group considered “innovative treatments and technologies,” those adopting approaches that depart significantly from conventional medical practices. They may incorporate broad cutting-edge scientific discoveries and breakthroughs in engineering, with the aim of providing appropriate new therapeutic possibilities and better quality of healthcare. As compared to conventional treatments and technologies, innovative treatments and technologies may suffer from a lack of statistical robustness, higher prices, and may require more extensive interdisciplinary decision-making processes and re-evaluations of their evidence. In this context, lobbying efforts and societal pressure may create challenges and raise unrealistic expectations from patients.

The discussion paper CDBIO (2023) 25 has been elaborated considering a wide variety of healthcare products, processes, and medical procedures, not limiting the scope of the work to the case of expensive medicinal products (ref. CDBIO (2023) 25 APPENDIX I: Illustrative overview of innovative treatments and technologies).

In addition, the drafting group mapped and briefly analysed the work already done by other international organisations, such as the WHO, OECD, and the EC, to provide an overview and avoid duplications of work.

Outcomes and conclusions

The examination carried out by the drafting group indicated that the general and procedural principles included in the articles of Recommendation CM/Rec(2023)1 are applicable also to innovative treatments and technologies.

In addition, although the analysis was carried out within the specific scope of the Recommendation, the drafting noted that the general and procedural principles may also be applicable in the context of access to innovative treatments and technologies that are used outside of the strict scope of Recommendation CM/Rec(2023)1, for instance for treatment of health conditions other than severe or life-threatening, or outside of situations of scarcity.

In view of these general conclusions and considering the specific characteristics of innovative treatments and technologies, the drafting group also pointed out that some principles may be especially relevant or may raise challenges for their application for innovative treatments and technology in the healthcare context.

The table below summarises the conclusions of the drafting group.

General principles
Non-discrimination (Article 4)	The article is applicable, and no differences have been identified for the application of this principle.
Attention to systematically disadvantaged individuals in relation to health (Article 5)	The article is applicable, and no differences have been identified for the application of this principle.
Prioritisation based on medical criteria (Article 6)	The article is applicable. A major challenge can be raised in case of lack of robust data on safety and efficacy.
Appropriate support and removal of barriers (Article 7)	The article is applicable. Significant barriers may exist that are more pronounced as compared to conventional treatments and technologies.
Respect for the dignity of persons excluded from access (Article 8)	The article is applicable, and no differences have been identified for the application of this principle.

Procedural principles
Accountability (Article 9)	The article is applicable. It is of particular importance in the context of innovative treatment and technology.
Reasonableness and relevance (Article 10)	The article is applicable. This principle may pose specific challenges in the context of priority-setting standards for access to innovative treatment and technology.
Inclusiveness (Article 11)	The article is applicable. This principle is more critical in the context of innovative treatment and technology.
Consistency (Article 12)	The article is applicable. This principle may be more challenging in the context of innovative treatments and technologies considering that policies may need frequent and ongoing adaptation in the light of rapid technological advancements and a changing evidence base.
Transparency and communication of decisions (Article 13)	The article is applicable. This principle is especially relevant in the context of innovative treatment and technology, considering the ethical and societal challenges that they may raise, the inflated patient and public expectations, and their budgetary implications.
Review (Article 14)	The article is applicable. This principle is more critical for innovative treatment and technology since innovative treatment and technology may be introduced for medical use under scientific uncertainty and with only limited availability of clinical data.

Possible future approaches

The drafting group suggested to consider the following possible approaches to support member States in ensuring “equitable access to appropriate innovative treatments and technologies in healthcare”.

a.    A new Recommendation might provide the opportunity to emphasise how the characteristics of innovative treatments and technologies may impact decision-making and equitable access. However, the drafting group does not suggest following this approach. The main reason is that the principles defined in Recommendation CM/Rec(2023)1 and its articles do not need to be modified when applied to innovative treatments and technologies in healthcare.

b.    A position paper, white paper or report may be considered by CDBIO to describe in more detail how Recommendation CM/Rec(2023)1 is applicable to the context of equitable access to innovative treatments and technologies, while also elaborating on the challenges and the solutions. The following consideration may be relevant for this approach:

                      i.        Obligation on the part of states could be helpful to ensure that innovative treatments and technologies are adequately considered and assess for their overall clinical value and appropriateness for patients in the national healthcare system. Innovative treatments and technologies may be available in some countries but not in others, not because of their different value but because of national health priorities, socio-cultural factors, regulatory approval processes, healthcare infrastructure, and resources.

                     ii.        Differentiating between experimental and innovative treatments and technologies may be complex. For instance, concerning certain treatments and technologies (e.g., immunotherapy, regenerative medicine, and neurotechnology), the boundaries between experimental and innovative may be dynamic, and their access regulated under different pathways (i.e., accelerated assessment, conditional marketing authorisation, compassionate use, clinical trials). Table II of the Concept note CDBIO (2023) 6 shown elements influencing equitable access to an innovative treatment during life cycle stages.

c.    Other efforts could be taken to promote equitable access to innovative treatments and technologies together with other bodies in and outside of the Council of Europe, noting, however, that providing guidance on reimbursement may not fall within the remit of the CDBIO.