Science and Technology CommitteeWritten evidence submitted by Parkinson’s UK

Do the European Commission’s proposed revisions to the Clinical Trials Directive address the main barriers to conducting clinical trials in the UK and EU?

1. Parkinson’s UK welcomes the proposed changes made by the European Commission to the Clinical Trials Directive. The proposed Clinical Trials Regulation appears to help overcome some of the barriers to conducting clinical trials in the UK and EU and addresses key criticisms of the Directive, such as reducing the administrative burden for clinical trials, clarifying the scope of clinical research so it is not interpreted differently across Europe, and introducing tight deadlines so clinical trials will not be delayed.

2. Introducing the concept of a low-interventional trial is an important step to adopting a risk based approach in clinical trials legislation. This would allow trials of medicines already authorised for use with minimal risk to go ahead more efficiently. This risk proportionate approach would recognise that the requirements associated with application and monitoring processes of a trial can be reduced for medicines with well-known safety profiles without compromising the safety of participants.

3. It is still unclear how the Regulation will reduce the requirements for trials of marketed products used for a new purpose, which are not included in the low-interventional trial category. Trials of these products are particularly important for Parkinson’s research. Drug repositioning (finding new uses for previously authorised drugs) has great promise for quickly bringing new treatments to people with Parkinson’s. Trials are currently underway to examine the effects of isradipine (an anti-hypertensive drug) and desferrioxamine (a drug to decrease blood iron levels) on Parkinson’s. As these are drugs that are currently in routine clinical use in the UK, it is vital that any beneficial effects are translated as fast as possible into the clinical arena.

4. A single application portal with a single application dossier is particularly attractive to streamlining and harmonising the application process for clinical trials. This will reduce the administrative work of sponsors who would otherwise have to submit the same documentation to all the Member States separately.

5. Efficient operation of the IT systems associated with a single European portal will be crucial to the success of all of the measures set out in the Regulation. The Commission should outline how it will go about creating and implementing the IT systems associated with the Regulation.

6. We strongly support the provision that the patients’ views must be sought in this process—it is crucial in order to assess the relevance of the trial to patients’ needs, and to obtain an accurate risk-benefit assessment. Patients, who ultimately bear the personal risks of participation in research, have the right to be involved in assessing its risks. They may be more willing to take up higher risks for different benefits, such as quality of life. For example, Parkinson’s UK surveyed our Research Support Network to ask if having to have a lumber puncture (spinal tap) would stop them from taking part in a drug trial. Whilst 50.4% of respondents agreed that it would put them off, 29.8% responded that they would still take part despite this invasive procedure. One respondent commented, “A lumbar puncture is nothing compared to slogging it out with Parkinson for 11 years plus”. Whilst another respondent said “Depends on whether the drug trial would be likely to make substantial strides towards a cure”. Patients with serious conditions will very often have a different perception of risk compared to that of investigators or regulators.

7. Patient involvement is also shown to contribute to better protocol design and the identification of new issues that researchers may not have considered. These can include for example practical questions such as treatment schedules or transportation that may affect patients’ participation and drop-out rate.

8. We welcome the provision for the publication of summary results of trials, but this should in be more strongly and precisely framed. We recommend that clear standards should be developed for what the summary needs to contain eg descriptions of the methodology, the way the researchers eliminated or minimised biases, blinding and randomisation arrangements etc. These standards should be developed with the involvement patient organisations and researchers, to ensure they address all groups’ information needs.

What is the role of the Health Research Authority (HRA) in relation to clinical trials and how effective has it been to date?

9. The process of obtaining research permissions from NHS Trusts has been identified as a significant barrier to research projects and trials in the UK, introducing delays and increasing costs. This process remains the responsibility of NHS providers which can result in submitting separate applications at each site. We welcome action taken by the NIHR and HRA to streamline this process.

10. We welcome the role of the Health Research Authority (HRA) to promote a unified and fair approach for approving research and producing standards for compliance for researchers.

11. As the HRA has only been in place since December 2011, we feel it is too soon to identify how effective it has been to date with regards to enabling clinical trials to take place.

12. Metrics will need to be developed to measure the effectiveness of the HRA. Researchers and patient organisations should also be consulted to gain an understanding of the level of the HRA’s effectiveness.

What evidence is there that pharmaceutical companies withhold clinical trial data and what impact does this have on public health?

13. It is crucial to research that there is transparency regarding how trials are conducted. Ensuring results are published provides essential information to future research, helping researchers learn and make further advances. Data from trials must be published to help future research build on results and improve treatments based on new discoveries. For example, recently the British Biotech company Phytopharm announced their new Parkinson’s drug Cogane failed in Phase 2 clinical trials. In week 28 participants taking Cogane showed no benefit compared to those taking a placebo. Whilst these results are disappointing, they have been crucially been made public for scientists, professionals and people affected by Parkinson’s so they are available for others to learn from.

How could the occurrence and results of clinical trials be made more open to scrutiny? Who should be responsible?

14. Pharmaceutical companies should have a legal responsibility to publish all trial data regardless of findings. However, it is important that the data is published in a useful format. For example, ensuring confidentiality issues are adhered to and published results should be clear and accessible.

15. The EU Clinical Trials Regulation should include measures that ensure registration and reporting of trials takes place.

16. The HRA should work alongside other bodies to ensure transparency.

Can lessons about transparency and disclosure of clinical data be learned from other countries?

17. We are currently unaware of other countries having improved transparency protocols. As we all come under the EMA umbrella, our national agencies fall under the same level of European law regarding disclosure. We are not aware of any that insist on the release of more data than is required by law.

February 2013

Prepared 16th September 2013