Capacity building needs a political will and
institutional support – Dr. Babar Saeed Khan
Rare diseases are rarely treated & orphan drugs are not available-Javed Akram
Pre-Conference Workshop on Clinical Trials during SNARE SUMMIT
LAHORE: A number of Pre-Conference Workshops were organized during the First Rare and Genetic Diseases Summit 2025 organized by the newly formed Society for Novel & Rare Diseases held here from September 4th to 6th 2025. One of these workshops was on Clinical Trials which was jointly chaired by Prof. Masood Hameed Khan former founder Vice Chancellor of Dow University of Health Sciences along with Prof. Masood Sadiq Vice Chancellor of University of Child Health Lahore, Prof. Moazur Rehman and Prof. Farhan Essa.
Prof. Masood Hameed Khan former VC DUHS along with Prof. Moazur Rahman and Prof. Masood Sadiq chairing the Pre-Conference Workshop on Clinical Trials during the First Rare and Genetic Diseases Summit held at Lahore from September 4-6th, 2025.
Prof. Javed Akram President of SNARE was the first speaker. He pointed out that for him it was a dream come true working with families with rare diseases. In Muslim countries including Pakistan the consangeous marriages account for 73-78%. These rare diseases are rarely treated and orphan drugs are not available. In this conference we are involving all the stake holders. Haqooqul Ibad, he further stated, were much more important. There is a billion dollar clinical trials global market. China is leading in the number of clinical trials being conducted followed by United States, Europe. Iran, Malaysia and India has done a lot and numerus clinical trials are being performed in these countries.
There are lot of opportunities for individuals and institutions. During the Covid pandemic, we had nineteen clinical trials in Pakistan. The PROTECT trial had eight treatment arms in which thirteen institutions were involved. We were trying to find a cost effective treatment. We have had lot of innovations. There are no biological plants. We have many patents but we cannot go in commercial production for which pharma industry with biological plants has to come forward but so far we have failed to get them interested. We have lot of burn patients and severe burns need skin grafting. The imported skin grafting material is very expensive. We produced Pak Derm local skin grafting which was tested and found equally effective. However, in the absence of any biological plant, we cannot produce it on commercial scale. We need to promote investigator driven clinical trials, he added.
Syed Munawar Ali Head of Clinical Operations Associate Director RDS Pakistan IQVIA discussed the Clinical Trials Do and Don’ts in the light of best global practices. He referred to ICH-GCP guidelines, Data integrity, Safety, Quality by design. Speaking about the key stake holders he mentioned Sponsor CROs, Principal Investigators, Research Team, Participating community, Institutional Review Boards/Ethics Committee, Vendor labs, image providers, Independent Safety oversight, researchers and investigators. Some of the Dos which he mentioned included feasibility, connectivity, transparency, documentation, source data, IP management, proper storage and accountability.
Do not force patients to join clinical trials, do not back date, later, leave gaps and do not ignore minor deviation safety alerts. Sponsor CROs must protect resources, ensure oversight presence, training and trials master file. They must not overload sites and do not neglect completeness. Trial Master File is the backbone of Clinical Trials. Speaking about the Dos and Don’ts of Regulatory bodies he mentioned publication of clear guidelines, adopt digital submission, risk benefit interpretation, acquire services of experienced trial experts.
Society for Novel and Rare Diseases organized the First Rare and Genetic Diseases Summit 2025 at Lahore from September 4-6th 2025. Group photograph taken after the pre-conference workshop on Clinical Trials shows some of the participants with speakers and executive of SNARE.
Dr. Babar Saeed Khan Director Clinical Trials Unit at National University of Medical Sciences discussed capacity building for clinical trials. This, he pointed out, is a continuous process. One must develop and strengthen skills, resources and infrastructure. In order to survive, adapt and thrive. Be prepared to respond to challenges. It also needs a political will and institutional support. He also highlighted the importance of regulatory framework, Ethical Boards, Trial registration. It is also important that one should be open to challenge innovations. He also talked about treatment policies, Standard Operating Procedures, Quality assurance mechanism. We need knowledge sharing platforms. At present their dare about eight to ten Clinical Trial Units functioning in the country. Human resource is essential for which we need to motivate professionals, it also needs a political will and institutional support. He also highlighted the importance of leadership and Governance, Partnership and Collaboration. We must make full use of technology, Digital Platforms and Artificial Intelligence Big Data. Some of the challenges that we face include workforce and patient advocacy groups. We need to look at the emerging diseases and precision medicine and adaptive clinical trials, he added.
Dr. Mahwish Ansari Deputy Director Pharmacy Division in the Drug Regulatory Authority of Pakistan (DRAP) discussed the regulatory pathways to clinical trials. She pointed out that we need strong regulatory pathways to ensure safety and transparency, credibility and ethical values. She also referred to the Thalidomide tragedy which resulted in thousands of babies being born without limbs. We need to follow ICH GCP guidelines. WHO has also come up with guidelines for Clinical Trials. EMA, FDA, MHRA have their own modules of guidelines. We have now covered the medical devices as well as health products in the regulatory framework. DRAP prepared ISO 14155 guidelines for medical devices. DRAP Act was introduced in 2012 while Biosafety rules were framed in 2017.
We have prepared Clinical Trials guidelines, GCP guidelines. However, she admitted that our regulatory policy is compromised and we mostly rely on FDA, HMRA guidelines. We take their help and rely on their decisions. She also discussed trial level vs site level monitoring, combination of both is important. She was of the view that we must focus resources where risks are higher and strengthen the GCP interpretation framework. All the stake holder’s engagement and awareness is essential. We have moved towards digitalization of application processing. Updated GCP guidelines including those for medical devices and health products are now available.
Dr. Ume Sughra Director Al-Shifa Research Centre from Rawalpindi was the next speaker who shared details of her personal journey with clinical trials. Our Clinical Trials Unit, she said, was inaugurated on 16th February 2021 and it was approved for clinical trials on March 3rd 2023 as a generalized clinical trial unit. We have been quite busy and conducted numerous clinical trials including participation in Covid trials. We have a dedicated team of experienced researchers, she added.
Mr. Shaukat Ali Jawaid Chief Editor Pakistan Journal of Medical Sciences discussed issued related to publication of clinical trials. He highlighted the importance of publishing clinical trials, research findings, he said, must be published otherwise no one is going to believe it. Moreover, it is also important where these clinical trials are published. Clinical trials contribute to medical knowledge and practice and there is a pathway to publication in high impact factor journals. Key criteria for high impact publications include rigorous methodology, ethical and transparent reporting besides clinical and scientific relevance.
The editors look at whether there was any novelty in the trials and does it fill some gap in literature. Is there any uniqueness of the research question and what will be its influence on clinical practice, guidelines and future research? Make sure to clearly define the objective. As regards study design and methodology, Randomized Clinical Trials are gold standard. It is important that the design matches the research question. Methodology should include sample size calculation, details of randomization and blinding besides details of comparison and control groups.
Ethical approval with reference number and date should be ensured. Documentation related to informed consent is important. Clinical Trials must be registered. Ensure up-to-date statistical methods, analysis, address the issues related to missing data and provide details of sub-group sensitivity analysis. For reporting clinical trials, follow CONSORT Guidelines and it should include flow diagram. Supplementary material incudes protocol, site plan and datasets.
Manuscript Structure: It includes Title and structured abstract, Introduction, Methodology, Results, Discussion and Conclusions. It is important to use precise and jargon free language and disclose conflict of interest and funding sources. During data interpretation mention statistical vs clinical significance, study limitations and biases if any besides clarifying generalizability of your findings. While selecting the journal for publication make sure that your study matches the journal scope, its readership and focus. Always look at the journal’s history of trial publications. Some journals do provide pre-submission review, ensure to get peers colleagues feedback. Submission of manuscripts must accompany a strong covering letter emphasizing the importance of the study.
Continuing Mr. Shaukat Ali Jawaid mentioned that authors must expect major revisions and ensure to professionally respond to reviewer’s comments and suggestions. Ensure transparency in data sharing, sometimes the journal editors may ask for raw data as well. Before submission, do check that trial registration number with date is mentioned in the abstract as well as in the main text. You have followed the CONSORT guidelines as well as ICMJE guidelines for authorship. Conflict of interest and funding sources have been mentioned. Do include EC approval documents and consent document.
Presentation of the manuscript he stated is extremely important. The title page of the manuscript must ensure that the title is concise and informative, mention RCT if applicable. Author names and affiliation details, details of the correspondence author, word count, number of tables, figures and trial registration details. Structured abstract should include Background and Objective, Methods (design, setting, participants), results should cover primary and secondary outcomes, conclusions and trial registration number.
Giving further details about the information which introduction section should include he mentioned background and rational of the study, clearly state study’s objective and hypothesis. Methods section should cover trial design, inclusion and exclusion criteria, intervention details, randomization, blinding, sample size, statistical analysis and how you have handled the missing data.
The Results section should have CONSORT flow diagram, baseline characteristics, estimates of primary and secondary outcomes, adverse events and sub-group analysis. The Discussion section should include summary of key findings, comparison of results with existing literature. Always ensure to discuss limitations and generalizability, practical and research implications followed by conclusions.
The manuscript should also include authors individual contributions and acknowledgement if any. Figures and Tables should be clear and must convey the message in standalone positon. All abbrevations used in the text must be defined and ensure adherence to authorship guidelines by Internaitonal Committee of Medical Journal Editors (ICMJE). He concluded his presentation by stating that always start with a strong, well designed study, follow ethical reporting and statistical standard. Be transparent and precise, choose the right journal and be prepared for revisions. He also highlighted those medical personalities who have contributed a great deal to promotion the discipline of medical journalism in Pakistan.
During the discussion and concluding remarks by the chairpersons Prof. Farhan Essa emphasized that informed consent should be proper since literacy rate is very low in Pakistan. Data is a major issue; we need proper documentation. Prof. Masood Hameed referred to the start of Certification in Clinical Trials at Dow University of Health Sciences to produce human resource which is extremely important. Healthcare professionals must be trained in biostatistics, epidemiology and research. Screening of diseases before and pre pregnancy should be looked into. Prof. Javed Akram emphasized on easing the regulatory pathways. Availability of orphan drugs should be ensured. Responding to a question it was stated that for personal use of the patients, these orphan drugs can be imported but one has to keep proper records.
Replying to a question about authorship Mr. Shaukat Ali Jawaid stated that unfortunately gift authorship is very common which is an intellectual corruption and a menace which must be checked. I do not allow more than four authors in a single study in our journal and if the authors insist, they are asked to get a letter from the Ethics Committee.
Unfortunately, predatory journals are also thriving and recently more than three hundred faculty members did get promoted by publishing their manuscripts in a predatory journal which was not recognized by the PM&DC since 2018. There are different Mafia groups active in Pakistan and it also includes a Medical Mafia Group which promotes and patronizes such unethical publications.
