Spinal Muscular Atrophy (Europe) PSP question verification form

The purpose of this Question Verification Form is to enable Priority Setting Partnerships (PSPs) to describe clearly how they checked that their questions were unanswered, before starting the interim prioritisation stage of the process. 

The JLA requires PSPs to be transparent and accountable in defining their own scope and evidence checking process. This will enable researchers and other stakeholders to understand how individual PSPs decided that their questions were unanswered, and any limitations of their evidence checking.  

Name of the PSP

Spinal Muscular Atrophy (Europe)

Please describe the scope of the PSP

Treatment, management and care (incl. mental health, nutrition), wellbeing and participation (topics that address the holistic needs of an individual. The PSP will exclude from its scope questions about diagnosis and newborn screening.

Please provide a brief overview of your approach to checking whether the questions were unanswered

To determine whether the questions identified in the survey have been answered by existing research, we conducted a structured literature review using databases such as PubMed, Cochrane Library, Embase and Google Scholar. The process began by defining the criteria for what constitutes an "answered" question, focusing on peer-reviewed publications, systematic reviews, clinical guidelines and meta-analyses. We developed a search strategy using keywords and phrases relevant to each summary question, applying filters to refine the results. After an initial screening of titles and abstracts, a full-text review of the selected studies was performed to assess their relevance and quality using standardised tools such as GRADE. Key information was extracted from these studies and compared with the summary questions to determine their status. The results were documented in a report highlighting which questions were answered, which were partially answered or remain unanswered, and identifying gaps for future research. 

Please list the type(s) of evidence you used to verify your questions as unanswered

To check whether the survey questions were unanswered, we used several types of tests. This included conducting a comprehensive review of peer-reviewed journal articles, systematic reviews, meta-analyses and clinical guidelines from reputable medical and scientific databases such as PubMed, Cochrane Library and Embase. We also considered evidence from clinical trials and consensus statements from professional organisations. The focus was on identifying studies that directly address the questions of interest, ensuring that they are based on rigorous research methodologies. By critically evaluating these sources, we determined the extent to which each question had been explored or remained unexplored in the existing literature.

Please list the sources that you searched in order to identify that evidence

To identify evidence on whether the survey questions have been answered, we searched the following key sources:

1. PubMed/MEDLINE: A comprehensive database of biomedical literature, including research articles, clinical trials, and reviews, which is essential for medical and health-related questions.
2. Cochrane Library: Known for its high-quality systematic reviews and meta-analyses, Cochrane provides reliable evidence on the effectiveness of healthcare interventions.
3. Embase: A biomedical and pharmacological database that includes extensive coverage of international journals, offering a broader range of research articles and conference abstracts.
4. Google Scholar: Provides access to a wide array of scholarly articles across various disciplines, including grey literature and theses, which may not be covered in other databases.
5. CINAHL (Cumulative Index to Nursing and Allied Health Literature): Specializes in nursing and allied health literature, which is valuable for questions related to healthcare practice and policy.
6. ClinicalTrials.gov: A registry and results database of publicly and privately supported clinical studies conducted around the world, useful for identifying ongoing and completed trials relevant to the questions.
7. Professional Organization Guidelines: Recommendations and consensus statements from organizations such as the American Academy of Neurology (AAN) or the National Institute for Health and Care Excellence (NICE), which provide authoritative guidance based on existing evidence.
8. Scopus: A multidisciplinary database that includes peer-reviewed journals, conference proceedings, and patents, offering a wide scope of scientific literature.

What search terms did you use?

Used search terms ‘SMA’ or ‘spinal muscular atrophy’ and ‘systematic review’.

Please describe the parameters of the search (eg time limits, excluded sources, country/language) and the rationale for any limitations

Included all reviews published in the last 5 years 2019-2023.

Searched PubMED and Cochrane Library.

Names of individuals who undertook the evidence checking

Kristina Staley was the Information Specialist for the PSP

On what date was the question verification process completed?

June 2023

Any other relevant information

N/A