COMMENTS FROM THE ASSOCIATION OF MEDICAL RESEARCH CHARITIES ON THE DRAFT
BILL (DATED 15.9.15)
Summary
We support the principle that patients should have access to the best available treatments
as quickly and easily as is safely possible. New initiatives that improve innovative treatments
should be encouraged, provided they do not impact negatively on patient safety, existing
innovation or research.
We appreciate the inclusive approach and a genuine effort to engage with stakeholders,
including medical research charities, which you have made in developing this Bill. However,
we still don’t see the need for new legislation in this area. More importantly we are
concerned that the Bill in its current form might have significant unintended consequences.
Premise of the Bill and litigation
1.1 From discussion with experts in the community we still don't think that the Bill
is necessary. In the context of the Bill, any reassurance about perceived fears of litigation
needed to change doctors' practice would be best achieved via changes to GMC guidance.
1.2 Furthermore, the Bill potentially adds complexity and confusion to the law surrounding
medical negligence rather than the intended purpose of reducing perceived fear of
litigation. This is particularly so since the judgement in Montgomery v Lanarkshire Health
Board [2015] UKSC 11 which moves the informed consent test away from the ‘reasonable
body of opinion’ test to a far more subjective patient-focused test. Arguably the test in the
proposed legislation to ‘obtain the view of one or more appropriately qualified doctors’
does seek to change the law of negligence despite the stated intention not to do so.
Scope of treatments covered by the term “innovative medical treatments”
1.3 It is difficult to confirm our position without knowing how this term is defined. We think
it essential that it is defined on the face of the Bill. Does it include (i) unlicensed treatments
and any off-label use or (ii) only licensed treatments used off-label; or something else?
Would products approved via the Early Access to Medicines Scheme be within scope?
Definition of “accepted medical treatments”
1.4 If it is proposed that “the existing accepted range of medical treatments” will be defined
in such a way that the database will include all off-label treatments, the Bill is in danger of
introducing more bureaucracy (and therefore disincentives) to prescribing treatments
which are, or have the potential to be, widely used off-label, e.g. for children, or those
treatments that may have enough evidence to be licensed but remain unlicensed for
commercial reasons (i.e. repurposed off-patent drugs).
Applicability to the patient journey
1.5 Are there any restrictions on the type of patient that would be eligible to receive an
innovative medical treatment?
1.6 Where are the safeguards that an “innovative treatment” would only be considered for
patients whose condition is not adequately treated/managed through existing goldstandard treatment options?
1.7 How would the legislation link with existing end-of-life care frameworks? What are the
safeguards that the Bill will not promote over-treatment of patients with drugs rather than
discussions about palliative care options?
Patients as partners in their own care – will the Bill promote informed shared decision
making with patients?
1.8 When we met, you stated that patients would not have access to the database. Where
are the safety nets for patients? How will they be able to ascertain the prevalence or
otherwise of the use of any innovative treatment being suggested to them?
1.9 Will there be a duty on the prescribing doctor to communicate the opinions of all peers/
colleagues from whom the doctor has sought in order to prescribe the treatment?
1.10 We would like to ask for a greater clarity on patient consent in the Bill so it states that
information will be shared with the patient and discussed fully. This would need to cover the
likelihood of success as well as safety risks.
Qualifications and independence of prescribing and advising doctors
1.11 Does the definition of “appropriately qualified doctor” in section3, subsection 3
effectively rule out General Practitioners? Could this disincentivise GPs from innovating?
1.11 How will patients be assured about the independence/potential conflicts of interest of
the prescribing doctors and those whose views are sought?
1.13 We would also question the proposal that support could come from only one expert in
the field. That is at odds with current practice, for example, in cancer care where
consultants work in multidisciplinary teams. The level of consultation with peers is also
important because not all doctors will be aware of ongoing trials.
The database of innovative medical treatments
1.14 The idea that any innovation could be captured in a database on the face of it seems
like a good idea and does go a small way to addressing concerns raised about information
capture from the previous incarnation of the Bill. We do not believe that legislation would
be needed to establish such a database.
1.15 Furthermore, as framed, it is unclear what it would add above publishing case reports
in the literature and the non-mandatory nature of entries to this database means that there
remains a significant risk that information of both beneficial and harmful effects of
treatment would not be captured. We have reservations about the utility and safety of a
database that is not mandatory. Existing databases which are mandatory, such as the
Systemic Anti-Cancer Therapy Database (SACT), are still incomplete. We anticipate that
there is little chance of this non-mandatory database being populated to any meaningful
level. What is the rationale for not making it mandatory? How will clinicians be incentivised
to record data?
1.16 When we met you indicated that only clinicians would have access to the database but
the Guidance Note issued on 21 September states that doctors are just an example of who
will have access. This is a critical point. Limiting access to clinicians would substantially
reduce the potential for these data to contribute to the wider evidence-base. However, all
the issues about who has access to care.data and for what purposes would also apply to this
database. In particular, small patient numbers mean there is a very high probability that
patient anonymity could not be protected.
1.17 Outcomes of treatments are dependent on many factors including co-morbidities, past
medical history, concomitant medication etc. Hence, to be of any use, the database would
need to contain or be linked to other sources of these data at patient level. We would
question whether funding the development of a new database makes sense. There may be
more robust and cost-effective options for recording information about the outcomes of
innovative treatments e.g. via care.data or its replacement, the Clinical Practice Research
Datalink etc.
Payment for the treatment
1.18 As commissioning is based on accepted treatment pathways, who would pay for
treatments prescribed through this route, and how would they decide which treatments
should be funded and which should not? For example, until recently Parkinson's patients
had trouble accessing the treatment Duodopa. It is now nationally commissioned but before
this patients had to go through individual funding requests to access it, often without
success, even though their consultant prescribed the treatment.
1.19 Could a treatment ever get enough evidence via this route to move to routine
commissioning? Or the converse?
Impact on research
1.20 We remain concerned that the Bill may inadvertently act to discourage patients and
their clinicians from participating in robust research studies, where novel or experimental
treatments - especially unlicensed drugs – are prescribed in settings where there are proper
arrangements for clinical monitoring and ongoing data collection. Even a doctor ‘acting
responsibly’ might choose to prescribe an innovative treatment to a patient rather than
enter them into a clinical trial (particularly as this would guarantee them access, whilst in a
trial they might be in the control arm).
1.21 How will the Bill be drafted so as to safeguard clinical trials? To ensure the primacy of
research might doctors be required to record that they have explored options for clinical
trials before using the “innovative treatment” route?
1.22 If the Bill applies only to licensed treatments, we would have fewer concerns about the
potential impact on research. However, concerns would still exist. For example, it could
easily undermine clinical trials aimed at testing new indications for licensed medicines and some of
the new types of trials planned via initiatives like EAMS and adaptive pathways.
Conclusion
We are unable to support the Bill as it stands and question the fundamental premise upon
which it is based.
We look forward to seeing the Bill team’s responses to our questions and how these are
reflected in drafting changes to the Bill.
AMRC
1 October 2015