By Christina Poschen, Consultant
Since 2004, the European Union’s centralised drug approval is in place, allowing manufacturers to submit just one application to gain marketing authorisation in all European Union Member States. Although there were voluntary agreements and working groups to further harmonize health technology assessment, so far, there are 28 different systems making it a challenging task to seek reimbursement in all countries.
In January 2018, the European Commission adopted a proposal for the regulation of the European Parliament and of the Council on health technology assessment and amending Directive 2011/24/EU. The proposal focuses on four key aspects:
- Joint Clinical Assessment
- Joint Scientific Consultation
- Identifying emerging Health Technologies
- Voluntary cooperation
This briefing focuses on changes to the proposal voted for by the European Parliament in October 2018, with a focus on the Joint Clinical Assessment and Joint Scientific Consultations.
Proposed regulation aim:
- Strengthening the cooperation between European Union Member States
- Avoid duplication of clinical assessments conducted for new medicines, in-vitro diagnostics and medical devices
- Ensure predictability and faster access for patients
Focus on key developments
Joint Clinical Assessment
- A newly established Coordination group will together with nominated experts, conduct a joint clinical assessment report, focusing on the therapeutic value only. Member States are asked to use the report for their price setting, cost effectiveness and budget impact analysis
- The European Parliament voted on a slightly watered-down language which gives Member States the opportunity to conduct their own assessments. This follows requests from countries as Germany and France. Austria which holds the European Presidency this year is currently working on a compromise acceptable for all
- The proposal also diminished the role of patients and consumer groups to provide written comments only rather than being involved in the assessment process itself
- Six months after the regulation enters in to force, the Coordination Group should develop the principles for assessment based on existing EUnetHTA work
Joint Scientific Consultation
- Manufacturers can request a joint scientific consultation with the coordination group for HTA. This can be conducted in parallel with EMA scientific advice, the coordination group will align the timelines
- The consultation focuses on trial design, evidence generation, predictability, quality and efficiency to ensure best evidence
- Following a draft scientific consultation report, the manufacturer can comment on the draft, patients HCPs and experts may also comment
- The European Commission will define the consultation process
Focus and reactions on the provisions for orphan drugs
This proposal is aimed at all products including orphan drugs. The proposal acknowledges the challenges associated with developing orphan drugs:
- The Coordination Group could consider that there is no reason or evidence available for a further benefit assessment beyond the EMA’s
- The proposal offers the opportunity to develop a specific orphan drug pathway to take the limited patient population and a lack of comparator in to account, under the provision that this information need to be publicly available
- The patient safety and scientific quality should not be compromised
A number of consumer groups fear that the provision for orphan drugs open doors for less rigorously conducted clinical trials and spending money on high priced products without sufficient evidence. Ensuring access for all patients to safe and high-quality drugs needs to be the number one priority for manufacturers and assessor and we therefore believe that orphan drugs need to be considered slightly differently to other therapies.
The EU aims at streamlining the clinical assessment of health technologies to facilitate faster patient access and improved predictability for manufacturers. This approach is widely supported by industry and consumer groups. However, a number of proposed changes introduced new uncertainty, the role of patients? The potential for additional national consultations? The role of orphan drugs?
The effort of the Austrian Council Presidency will be crucial to get a timely and mutually acceptable regulation. For the proposal to be accepted, the Council needs a qualified majority of 55% of the Member States must vote in favour representing at least 65% of the European population
Europe votes in May 2019, the European Parliament’s ENVI committee therefore needs to be quick to get details discussed with Member States in the Council before the break for the elections.
During the translation period, the number of joint clinical assessments and joint scientific consultations will be dependent on the number of participating Member States and available resources.
Partners4Access will continue to monitor the progress, for more information and to understand how this could affect your product, please contact:
Christina Poschen at firstname.lastname@example.org]
P4A are specialists in orphan drug, cell and gene therapy access, partnering with the biotechnology industry to support launches worldwide. Focus areas include: orphan drug, cell and gene therapy pricing and market access strategy; bespoke access roadmap development; rare disease foresights; evidence mitigation solutions and access team excellence training. As one of the only consultancies with comprehensive experience in orphan, cell and gene therapies, P4A are excellently placed to help clients successfully achieve access.
Through its initiatives, P4A are able to inform and shape the conversation around orphan drugs.
The team at P4A believe in striking an equilibrium in the rare disease world. That means helping drive access to the most appropriate treatments for patients and physicians, a fair return on investment for manufacturers at an affordable proposition for payers.
For more information, please email P4A at email@example.com.