Myeloma UK believes that, HTA is a vital part of the clinical path from bench to bedside “system” in medical research. However, many patient organizations focus too much on HTA, and the appraisal of individual treatments, at the expense of focusing on other parts of the system.

Each stakeholder involved in the discovery, development, adoption and diffusion journey (or life cycle as described by the Policy Forum) have specific interests, incentives and rewards, which are not usually focused around a common goal—patients. Patient organizations, if they work smartly, can act as an honest broker to gain better alignment and focus in the system: facilitating very early engagement among relevant stakeholders to discuss/agree unmet needs; advising industry on study design to maximize patient participation and produce results on outcomes of importance to patients; foster collaboration among all stakeholders to develop high quality evidence rapidly that will lead the accelerated uptake of effective technologies. They are perfectly placed to suggest and broker solutions to meet the needs of all the stakeholders, but in a way that ultimately benefit patients.

Marginal improvements to the inputs in to HTA would improve the number of positive recommendations. Improving the design of clinical trials to include quality of life and patient relevant endpoints as well as appropriate comparators for example, could significantly improve the chance of a positive appraisal and ultimately outcomes for patients.

Patient organizations can play an important role in improving these inputs if they engage early and effectively with the pharmaceutical companies who are responsible for designing clinical trials and generating data for regulators, HTA bodies, and payers. Furthermore, patient organizations can take a more proactive approach to the development of evidence for HTA.

The Myeloma UK Clinical Trial Network (CTN), which includes thirty-five hospitals across the United Kingdom and a central clinical trial office, is a good example of this: we established the CTN, in part, to partner with the pharmaceutical industry to generate, by means of investigator-initiated studies (IIS), complementary evidence reflecting real-world clinical practice in the United Kingdom as a means of addressing uncertainties in the regulatory data set. A key part of the CTN strategy is to engage early with NICE Scientific Advice to gain an early opinion on both the design of the Phase III regulatory trial alongside the Phase II CTN IIS.

However, to ensure the best possible chance of success in an HTA, it is important that patient organizations understand the perils and pitfalls of participating. While securing a “yes” could be a fantastic result, it is important to know exactly what to expect.

First, taking part in an HTA requires significant investment of time and energy. After all, you need to really know your stuff before you take part, being unprepared could seriously impact the chances of your involvement having any value at all.

Second, understanding why HTA bodies say no, and seeking to mitigate against that is important. There are many reasons why HTA bodies such as NICE and the SMC say no, and most often, they are well-founded. Common reasons include the use of an inappropriate comparator, indirect comparisons, a lack of transparency in the modeling, a failure to make the economic case, over-optimistic assumptions, and more. Engaging early with industry, as well as HTA bodies themselves, on these factors could significantly improve the prospects of a positive appraisal.

Unsurprisingly, HTA bodies expect patient organizations to communicate the impact and value of a particular treatment to patients in a compelling and evidence-based way. You are there to represent what impact a particular treatment will have on patients’ lives; but this needs to be done in a way that is objective and rational.

On a similar note, emotions, anger, and anecdotes tend not to be effective: appraisal committees at NICE or the SMC will not be persuaded if you bang your fist on the table without good reason. It is better to present your arguments in as clear and objective a way as possible and ensure that the points you make are backed up with evidence.

Another, often overlooked, factor is the need to secure consensus. Dissent among the clinical community about the effectiveness and place in the pathway of a new treatment will make a “No” much more likely. Working closely with clinicians in advance to achieve a consensus could make all the difference between a “yes” and a “no.”

In summary, HTA should be a friend and not a foe for patients and patient organizations. If you engage early, are well prepared, have a clear and objective understanding of the evidence, have achieved consensus among the clinical community, you have the greatest chance of success.

What is more, if you see HTA as a vital part of the wider system of drug discovery through to approval and clinical practice and actively seek to engage at key parts of the this system, you are much more likely to generate even better outcomes. If you do that successfully, ultimately that will benefit the people who matter most: the patients and the families you represent.