MEPs Against Cancer
feb 2 2013.jpg
mac group picture.jpg
MEPs Against Cancer Round Table
Summary Report Below
At the core of the discussion lies the idea proposed by several leading European oncologists that the existing Advanced Therapy Medicinal Products Directive (ATMP) is not equipping oncologists involved in vaccine based targeted treatments for certain patients with the correct legislative framework.
Professor Stefaan Van Gool from Gasthuisberg Hospital, Leuven, a world-renown specialist in paediatric oncology provided a proposal for a new European legislation that would complement the existing ATMP directive (not replace) that will enable a more patient-centred treatment approach within European legislation.
Prior to this event, there was a MEPs Against Cancer visit organised to the Immunotherapy Platform in Leuven on May 13
Alojz Peterle MEP and MAC President, Professor Van Gool of KU Leuven, Ilse de Reze of the
Olivia Hendrix Foundation and Professor Phillipe Lewalle Clinical Director of the Jules Bordet Institute.
Fits and Mis-fits: Advanced Therapy for Medicinal Products (ATMP) Legislation
On the 15th October, 2013, the MEPs Against Cancer group facilitated a meeting on the challenges of working on immunotherapy treatments under the Advanced Therapy for Medicinal Products Legislation. At the core of the discussion lies the idea by Professor Van Gool, which is supported by immunotherapy oncologists across Europe, to propose a new European legislation that would complement the existing ATMP directive (not replace).
Rocio Salvador Roldan, DG Health and Consumers, Unit D5 – Medicinal products – authorisations made a presentation on the strengths and weaknesses of the current legislation which is currently undergoing a consultation process. Three types of ATMPs are covered by the legislation:
The legislation also provides for marketing licences for products developed by immunotherapy and a framework for achieving ATMP status for advanced therapy treatments.
Professor Matthias Eyrich of the Wurzburg Children’s Hospital in Germany described problems he has experienced firsthand with working under the remit of ATMP. In Germany, in the field of immunotherapy, there is a huge discrepancy between patient’s needs for innovative treatments and what medics can offer them. As a result, they have been sending on average 30 patients per year to Leuven University Hospital to be treated by Professor Van Gool. The use of ATMP in Germany can be applied under clinical trials and through use of a legal article (known as paragraph 4b) which allows for them to be carried out if production and treatment is in the hands of one physician, a production license is be issued and if individual benefit is be visible. However data from this treatment is not allowed to be used for scientific analysis. In Germany, hospital exemption is only permitted on the authority of the federal level decision and is therefore very difficult to obtain.
According to Professor Eyrich the shortcomings to the ATMP regulation include the current rules for early phase developments that do not allow a timely translation in this field making Europe is uncompetitive in this area. In more advanced phases (I/II, optimization) current rules also do not allow academic hospitals to react flexibly and meet patients needs for innovative treatments: the timeline from the bench to bedside too long. He also stated that Hospital Exemption (HE) is too imprecise and highly diverse interpreted across EU, it also precludes data analysis, the genuine interest of academic centres. ATMP means that patients can be treated for orphan diseases but the legislation makes data analysis very difficult.
Professor Eyrich made the following comments and recommendations for the improvements to the ATMP regulation:
Patient advocate Piet Jurgwal and the father of a survivor treated by Professor Van Gool both gave testimonies to the quality of and efficacy of Professor Van Gool’s treatments. Mr Jurgwal underwent successful treatment with Professor Van Gool after being diagnosed with a stage four brain tumour which his physicians in the Netherlands had been unable to treat. Mr Jonak’s son was treated by Professor Van Gool for his brain tumour and is now working in Germany, drives a car and with his father has set up a patient support group in their area. One of the aims of their group is to encourage the spread of Professor Van Gool’s treatments to prevent unnecessary deaths among people unable to access the same quality of treatment.
Piet (Brain Cancer Survivor) and Jannigje Burgwal
Professor Van Gool outlined his proposal for a new legal framework for Advanced Therapy Treatments (ATT) would be different from ATMP in several ways summarised in the box below.
Prof. Dr. Mark Waer, Honorary Rector of KU Leuven opened his session by expressing support for the ATT proposal as he believed it would speed up approval processes and close the gap between the lab and the patient. He also sees a need for harmonisation of legal initiatives in the EU and opened the question of adapting the hospital exemption clause in ATMP to solve some of these problems. He also referred to the need to enable centres to exchange data from ATMPs across borders in order to maximise the results of treatments
Patricia Blanc, the Executive Director of Imagine for Margot, an NGO set up in the memory of her daughter who died of brain cancer started the expert panel with a call for more clinical trials for children in Europe. The current legislative framework creates too much paperwork which takes up time better spent on treatments. She added that the regulation on clinical trials has currently progressed, and feels strongly that the ATMP should do the same.
Professor Paolo Casali iterated that the key issues in cancer today are personalised oncology, multidiciplinary care and rare cancers. Within these areas four points are of vital importance:
a) Research methodologies and their regulatory implications. There is a need for randomised trials for rare cancers and regulatory bodies to be sensitive to new technologies.
b) With regards the compassionate use and off-label use of drugs, large discrepancies exist across Europe depending on the national legislation. Many children are treated with off-label drugs.
c) The relationship between academia and the industry arises along with the question ‘what if company is not interested?’ This poses the problem of orphan drugs and hinders the possibilities to develop new drugs.
d) Networking within the remit of the new cross-border healthcare directive will bring some opportunities but it is not yet clear whose rules the networks will come under or who will fund them. Problems of subsidiarity may also arise.
Arnd Hoeveler (DG Research) iterated that advanced therapies are not limited to cancer but all kinds of diseases. The Commission is supporting new innovative and regenerative medicine by mixing different approaches. ATMP is still an early regulation; with room for improvement while science is quickly evolving. There is a strong focus on offering incentives for SMEs and he recognised that maybe the academia and charity organisations do need more support however we need to avoid medical tourism and pay particular attention to safety aspects. He also suggested that the ATT proposal should consider whether EU legislation is actually needed in this area and whether the issues should be dealt with by the member states.
Professor Lewalle of the Jules Bordet Institute agreed with the proposal of Professor Van Gool, but also reminded participants that if regulation is too heavy it could risk killing academic research. He added that pharmaceutical companies cannot be relied on to develop treatments for rare cancers therefore regulations should be made to allow the academic world to be partners in immunotherapy. Custom made rules need flexibility and less regulation. If the bar is set too high, people will leave or give up. If it is set too low, it will be ineffective. Europe needs to advance in this area in a collaborative fashion to allow for an innovative and competitive environment that is best for the patient.
Alojz Peterle MEP, Prof. Dr. Mark Waer (Honorary Rector of KU Leuven), Ilse de Reze (Olivia Hendrickx Research Fund), Johnathan Stabenow (Assistant to Ivo Belet MEP), Emma Woodford (ECL), Professor Nadine Ectors (KU Leuven), Professor Paolo Casali (Milan Cancer Insititute) and Professor Stefaan Van Gool (KU Leuven) discuss follow up to the MAC Round Table on ATMP with members of the European Commission.