HTA and Patient Advocacy: An Important New Relationship

By: Mark Krueger, MPH


Patient voice increasing in HTA decision making

Today, patients play an increasingly important role in health technology assessment (HTA).  HTA is the multi-disciplinary field of policy analysis focused on medical, social, ethical, and economic implications of development, diffusion, and use of health technology. In many countries, notably excluding the US, patients are invited to provide their perspective on the value of diagnostic tests, drugs and devices before HTA committees and in many jurisdictions, patient representatives have a voting seat on the HTA committee. Patient representatives are selected from groups that meet a rigorous set of selection criteria.

While Europe is seen as the leader in HTA, each country within Europe has its own HTA process, ranging from complete patient involvement to no formal involvement whatsoever.  For example, in Sweden, patients are fully integrated in the Tandvårds-och läkemedelsförmånsverket (TLV) with four patient representatives on the committee. In the UK, NICE involves patient advocates by inviting them to comment on guidance. The Public Involvement Program encourages the public to get involved in NICE committees and a 30 member Citizens’ Council provides input. However, other agencies such as Austria’s Gesundheit Österreich have no formal system for patient input.

Expectations of patient representatives

Patient representatives on HTA committees are expected to bring the patient view of what is important in a technology, diagnostic, device or medication. Patients’ views, including their preferences, bring both real-life experiences and facts to the HTA discussion. Emotions and personal experience are often thought by experts to be best left at the door. It is important to note that not every patient representative will be an expert in every disease state – but s/he will have a full vote on the HTA committee.

Patient input can have a significant impact on HTA committees.   I recall when a teenage boy living with Duchenne muscular dystrophy described how a new treatment allowed him to move his arm, enabling him to feed himself and use a computer mouse, two things important in his life. The committee then understood that treatment outcomes for young men with Duchenne valued may well be different than those of clinicians. The contribution made by this young man went well beyond more traditional testimony about clinical data and theoretical patient impact.

How can companies get involved?
Enable expert advocates through appropriate training. Companies can’t and shouldn’t do this directly, but there are respectful, compliant ways to enable advocates to ensure that HTA decisions reflect patients’ real needs. Recently, at the Union for International Cancer Control (UICC) meeting, MK&A planned and executed a two-day HTA training program for 30 carefully selected cancer groups. Content included technical sessions, policy discussions and role playing. All were designed to develop a cadre of skilled advocates equipped to participate fully and credibly in HTA deliberations. While the session was organized on behalf of a pharma client, the company did not participate directly.

MK&A’s Expertise in HTA
MK&A has worked in the HTA arena for 20 years, charting patient engagement, talking with HTA authorities, and learning patient groups’ perspectives. We have also created what we believe to be the only map of patient and consumer engagement in HTA discussions and decisions making.

Outside of Europe, the HTA process tends to be more opaque, although there is movement in some countries. In Canada, MK&A recently was actively involved in a series of collaborative dialogues with senior federal and provincial health officials, patient groups, sponsors and others to formalize the patient voice in the evolving framework for managed access for orphan drugs.

In the US, there is no HTA and CER (comparative effectiveness research) sometimes a long way off.  With increased interest and funding from PCORI and involvement by the insurance industry, this may eventually change. Patient advocates’ role in the US in ensuring access is different but also important. Recently, the U.S. Food and Drug Administration convened meetings to understand patients’ views on a particular disease. Perhaps more promising is the submission of the first patient group-led draft guidance on drug development to be received by the FDA with its full cooperation.

The patient voice will continue to have a growing impact on the HTA decision-making process.   If you would like more information about HTA or how your company can responsibly get involved, please contact me at mkrueger@mkanda.com or +1 212 620 2770.




About the Author:

Mark Krueger, MPH

Mark Krueger, MPH is president of MK&A.  He can be reached at +1 212 620 2770 or at mkrueger@mkanda.com.

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