Evidence development for medical devices is often focused on satisfying regulatory requirements with the result that health professional and payer expectations may not be met, despite considerable investment in clinical trials. Early engagement with payers and health professionals could allow companies to understand these expectations and reflect them in clinical study design, increasing chances of positive coverage determination and adoption into clinical practice.

An example of early engagement through the EXCITE International model using an early technology review (ETR) is described which includes engagement with payers and health professionals to better inform companies to develop data that meet their expectations. ETR is based on an early evidence review, a framework of expectations that guides the process and identified gaps in evidence. The first fourteen ETRs were reviewed for examples of advice to companies that provided additional information from payers and health professionals that was thought likely to impact on downstream outcomes or strategic direction. Given that limitations were imposed by confidentiality, examples were genericized.

Advice through early engagement can inform evidence development that coincides with expectations of payers and health professionals through a structured, objective, evidence-based approach. This could reduce the risk of business-related adverse outcomes such as failure to secure a positive coverage determination and/or acceptance by expert health professionals.

Early engagement with key stakeholders exemplified by the ETR approach offers an alternative to the current approach of focusing on regulatory expectations. This could reduce the time to reimbursement and clinical adoption and benefit patient outcomes and/or health system efficiencies.

The objective of this research was to evaluate managed access policy in England, drawing upon the expertise of a range of stakeholders involved in its implementation.

Seven focus groups were conducted with payer and health technology assessment representatives, clinicians, and representatives from industry and patient/carer organizations within England. Transcripts were analyzed using framework analysis to identify stakeholders’ views on the successes and challenges of managed access policy.

Stakeholders discussed the many aims of managed access within the National Health Service in England, and how competing aims had affected decision making. While stakeholders highlighted a number of priorities within eligibility criteria for managed access agreements (MAAs), stakeholders agreed that strict eligibility criteria would be challenging to implement due to the highly variable nature of innovative technologies and their indications. Participants highlighted challenges faced with implementing MAAs, including evidence generation, supporting patients during and after the end of MAAs, and agreeing and reinforcing contractual agreements with industry.

Managed access is one strategy that can be used by payers to resolve uncertainty for innovative technologies that present challenges for reimbursement and can also deliver earlier access to promising technologies for patients. However, participants cautioned that managed access is not a “silver bullet,” and there is a need for greater clarity about the aims of managed access and how these should be prioritized in decision making. Discussions between key stakeholders involved in managed access identified challenges with implementing MAAs and these experiences should be used to inform future managed access policy.

Early access schemes (EASs) are approaches used by payers to balance and facilitate earlier patient access to innovative health technologies while evidence generation is ongoing. Schemes require investment from payers and are associated with significant risk since not all technologies will be routinely reimbursed. The purpose of this study was to gain the perspectives of policy experts about the key challenges for EASs and potential solutions for their optimal design and implementation.

Two virtual workshops were convened including (i) UK-based policy experts (England, Wales, and Scotland) and (ii) representatives from multiple healthcare systems (England, France, Sweden, Canada, Poland, and Norway). Participants were encouraged to share their experiences with EASs in their healthcare system and highlight key challenges for policy makers. Discussions were transcribed and analyzed using framework analysis.

Participants agreed that EASs have value when targeted toward innovative technologies with the potential for significant clinical benefit in an area of high unmet need. Participants discussed potential solutions to the challenges faced by payers implementing EASs, including defining eligibility criteria, supporting evidence generation, and approaches to reimbursement.

Participants agreed that EASs are one possible solution for their healthcare systems and have the potential to deliver significant clinical value to patients. However, widespread adoption of EASs is limited due to concerns about the risks for patients and healthcare budgets, further solutions are needed to deliver EASs for targeted therapies.

Many surgical and nonsurgical procedures have been designed for the treatment of snoring due to palatal flutter. All work in some, but not all, snorers. The difficulty lies in making the definitive diagnosis of palatal flutter as the cause of snoring, and in deciding which patients should undergo which treatment, which in some cases are relatively radical.

This study aimed to assess the usefulness of injection snoreplasty in differentiating palatal flutter from other forms of snoring. This was done in the hope of determining which patients would benefit from definitive palatal surgery such as uvulopalatopharyngoplasty and laser-assisted uvuloplasty.

Sixty consecutive patients referred for habitual snoring were treated with sodium tetradycil sulphate during their first consultation visit. No patients were excluded and none refused the treatment. Forty patients received a single 1 ml dose of 1 per cent sodium tetradycil sulphate, and twenty patients received a single 1 ml dose of 3 per cent sodium tetradycil sulphate under topical anaesthesia. Visual analogue snoring scales were completed by the patient and their partner six weeks, three months, six months and 12 months after the procedure.

Forty of the 60 patients showed improvement in snoring and therefore were considered for definitive surgery. Four of the 60 patients found the investigation unpleasant and did not want any further treatment. Of the 40 patients who showed improvement, 29 maintained this at one year. The other 11 underwent uvulopalatopharyngoplasty or laser-assisted palatoplasty. All patients had successful snoring scale outcomes following the surgery.

A significant number of the patients, 62 per cent, were demonstrated to have significant improvement in the short term. Single dose injection snoreplasty seems not only to be an effective investigation but may constitute a safe and simple treatment within the clinic. At the very least, patients in whom the palate appears not to be the problem are prevented from undergoing painful, unpleasant surgery. Our results support the use of injection snoreplasty, both as an investigation and in some patients as a treatment, for habitual snoring.