The pharmaceutical package and intellectual property reform to overhaul the regulation of incentives, shortages, and compulsory licensing, among other issues, was presented by the European Commission (EC) in April 2023. As stated by the EC, the reform aims to improve access to health technologies across the EU, ensuring patients have timely and equitable access to safe, effective, and affordable medicines[1]. It also combines with other purposes related to supply, innovation, and competitiveness.
The package is now under discussion at the ENVI Commission in two different files: one related to regulation and the other related to an EU Directive. Other issues regarding compulsory licensing are under JURI’s Commission. It is indeed an ambitious reform that needs to be coherent, although it is a great window of opportunity to review current bottlenecks and gaps. To fulfill these purposes and learn from current inequities, it seems the time for public interest, access, and affordability of medicines, vaccines, and diagnostics.
So far, we have proposals coming from the different rapporteurs of these files. Some of the measures proposed are adequate, such as the transfer exclusivity voucher in Chapter III, which has been amended by the rapporteur, MEP Wolken. Transferable exclusivity vouchers would not function only in practice as an additional year of market monopoly; it is also a very limited tool to foster innovation in the antibiotic R&D landscape. The alternatives proposed in the rapporteur’s draft report are more interesting, namely the European Medicines Facility and the Union push and pull scheme. It is important now that these initiatives include a stronger access dimension, incorporating into its design both regional and global access plans, including but not limited to the establishment of manufacturing supply agreements, intellectual property sharing agreements, and knowledge and technology transfer, as necessary, in order to promote a shared global health R&D effort.
Regarding orphan medicinal products, it is important to highlight the need to link the orphan validity and designation to the principle of “sufficient return to justify the necessary investment” (ROI) that the rapporteur has put back in the draft report. This must be a mandatory requirement to be met and regularly evaluated, not an optional one. Furthermore, we believe that orphan designation must be linked to additional transparency and access measures.
It is also important to include as much support as possible for non-profit organizations, particularly those involved in the development of ATMP under hospital exemption, as well as those involved in R&D of health problems of global concern, including those listed under the “WHO Blueprint List of Priority Diseases” or currently endorsed Neglected Tropical Diseases in the portfolio of WHO. This support can be reflected by including these cases specifically as part of the Priority Medicines (PRIME) scheme. In exchange for this support, the regulator must ensure access conditions, such as manufacturing supply agreements, intellectual property sharing agreements, and knowledge and technology transfer, as necessary.
Regarding shortages, positive elements have been included in the draft report. Transparency is of the utmost importance in these cases, and this has to be a cornerstone of the prevention and management plans and of reporting the causes of shortages. Strong sanctions have to be put in place to avoid situations in which preventable and unjustified ruptures of supply occur due to pure commercial reasons.
Pharmaceutical Directive: a step back for equity and access to medicines
The draft report prepared by MEP Weisse is very disappointing, and a huge step back in terms of accessibility and equity for the EU. It has aligned with pharmaceutical industry views, and we now see a text that aims to increase the incentives without strong access and affordability conditions attached to them. It is also controversial to insist and enlarge on the use of these monopoly protections when there is no evidence that this is a measure in its current form incentivizes R&D.
Regarding the amendments on the Hospital Exemption (HE), the rapporteur is proposing a very restrictive definition of “non-routine basis” to limit its potential use while at the same time limiting the validity of the HE to one year. Additionally, its use would be possible in only a limited number of cases where no therapeutic alternative, clinical trial, or compassionate use program is available.
Our understanding of how the HE should be is quite different. HE has proven an excellent tool to make advanced therapy medicinal products accessible for patients in a timely manner, at a lower cost, while ensuring quality, safety, and efficacy. This has been the case with academic CARTs in Spain, which have coined the “Spanish Model” that includes GMP CPF accreditation, as well as pharmacovigilance plans and other elements that are very similar to those of a centralized procedure. The HE creates an important ecosystem with two relevant outcomes: on the one side, increased patient access to ATMP that could be otherwise not available for them; on the other side, it promotes innovation, R&D, and regulatory capabilities at academic and health institutions. This happens while lowering the cost 2/3 to that of a commercial CAR-T therapy. It is, therefore, a critical case of local production created by and for the public interest.
In that sense, the harmonization intended by the EC should take place, improving upon the high standards and success of the Spanish Model. Additionally, transparency measures around public funding of HE and reimbursement prices shall be incorporated, along with a registry, to strengthen collaboration and transparency among hospitals and non-profits.
Compulsory Licensing Regulation:
MEP Vázquez has recently finalized his draft report on the proposal for a regulation on an EU-wide CL, which includes a few aspects to highlight. Salud por Derecho welcomes the inclusion of disclosure of trade secrets and know-how as part of the compulsory licensing mechanism proposed. This need has been traditionally stressed by CSOs, particularly in the case of innovative vaccines during the COVID-19 pandemic, and we hope the EU will advocate for it in the negotiations taking place around the Pandemic Accord.
Additionally, the parliament’s position should include the need to extend the application of the CL to unpublished patents (patent applications might be confidential within the first 18 months since the filling), extend the suspension to market and data protection in the event of a CL (as referred in article 80.4 of the proposed directive mentioned before) to market exclusivities, and try to enlarge the scope of the regulation as needed to incorporate other public health threats or needs related to accessibility, availability or affordability of medicinal products.
[1] https://health.ec.europa.eu/medicinal-products/pharmaceutical-strategy-europe/reform-eu-pharmaceutical-legislation_en
