How full disclosure of clinical trial data will benefit the pharmaceutical industry
Disclosing clinical trial data is a step in the right direction towards transparency, which benefits both the public and the pharmaceutical industry.
Calls for greater transparency in the healthcare sector have increased in recent years. People want to shine a light on everything, including political donations, government contracts, regulatory decisions, corporate tax payments, profits and environmental impacts. People want greater transparency largely because they have lost confidence in the way institutions do things and want to understand how they can and should operate. Many people do not believe institutions serve the average citizen’s interests or the common good but, rather, cater to the private interests of a few wealthy elite or politically connected people.
In the area of healthcare, 74% of people believe drug companies routinely put profits before patients, and therefore before public and global health needs. To help understand the depth of potential inequalities and problems, to identify best practices and appropriately restore trust, institutions and stakeholders are turning to transparency efforts for assistance.
Several initiatives have tried to make institutions more data-driven, accountable, participatory, collaborative and trustworthy. These include the US Food and Drug Administration’s (FDA) Transparency Initiative, President Barack Obama’s Transparency and Open Government initiative, the Extractive Industries’ Transparency Initiative to govern the use of natural resources, such as gas, oil and minerals, Ernst & Young’s tax transparency initiative to address concerns that companies are not paying their fair share of taxes, and the Sunlight Foundation’s efforts to track political donations.
Improving the lack of transparency in the healthcare innovation sector, particularly in the pharmaceutical industry, is critical to advancing patient, public and global health. It is essential to creating evidence-based medicines and innovating new cures and therapies and will ensure we do not waste funding and resources in the innovation process, conserves costs, and protects research participants and patients. We also need transparency in the pharmaceutical industry for the same reason other industries currently need it: to restore trust in the sector.
Undisclosed clinical trial results
Clinical trial data contain vital information on the safety and efficacy of drugs, and prescribers should have access to these results to ensure they can prescribe appropriately. But there are concerns that information about new medicines is not always publicly available or it can be misleading. One study, for instance, showed that only a median of 57% of clinical trial results for a new drug are made publicly available. Other studies have shown that the majority of published new drug trials tend to be those with positive outcomes,,.
Many doctors follow prescription guidelines in their medical practice, which are based on reviews of publicly disclosed trials. Therefore, if trial disclosures are incomplete or biased, the integrity and reliability of prescription guidelines can become suspect, as might the idea of drug treatments being evidence based. Similarly, in the United States, hospitals, insurance companies and pharmacy benefits managers who are responsible for processing and paying prescription drug claims decide which medicines, vaccines, devices and biologics to include in their formularies based on reviews of publicly disclosed clinical trial information.
There are still many diseases and conditions in need of cures and treatments. In the case of clinical research, the industry cannot learn valuable lessons from past research if trial results are kept under wraps.
Advancing public health and protecting research subjects
Another problem with undisclosure is that it can contribute to research duplication, consequently driving up unneccesary costs. A lack of transparency can also have implications on research subjects, in that it is unethical to enrol people in trials for molecules with known but undisclosed toxicities or harms, as was the case with the TGN1412 disaster in the UK in 2006, where six patients involved in a phase I clinical trial of a drug being developed for leukaemia, rheumatoid arthritis and multiple sclerosis were admitted to critical care. A similar molecule had been tested in the past, but the results had not been publicly disseminated.
In fact, one of the main reasons we allow experimentation and drug testing on humans is because of its potential to contribute to generalisable knowledge and future cures. If trial information remains hidden behind corporate or academic veils, it is hard to generalise knowledge, especially if trials never make it to phase II or III.
Provided patient confidentiality, costs and other ethics issues are addressed, there are few reasons to justify opacity around clinical research, particularly for approved products being used by patients. Of course, different levels of transparency carry different costs. For example, disseminating summaries of clinical trial results on ClinicalTrials.gov — a registry and results database of publicly and privately supported clinical studies conducted around the world — is less costly than publishing results in a medical journal. It is also far cheaper than creating a new platform to share patient level data for trials. Notwithstanding the varying costs, many stakeholders are investing in patient level data sharing platforms, including companies, foundations and other groups.
The Good Pharma Scorecard
To help doctors, formularies, patients and other stakeholders understand how transparent the information is around new medicines and vaccines, Bioethics International, which I founded in 2005, has created the Good Pharma Scorecard (GPS).
GPS is an index that scores and ranks new medicines approved by the FDA, along with their institutional sponsors, on clinical trial transparency. It is an outcome-driven and performance-based assessment. We analysed, scored and ranked the drugs approved by the FDA in 2012 that were sponsored by the 20 largest drug companies (http://bioethicsinternational.org/good-pharma-scorecard-overview/background-and-rating-system/) and the first set of rankings were published in 2015. This was a ranking of 15 drugs, sponsored by ten large companies and involving around 100,000 research subjects. The results include data on drugs for diseases such as HIV, breast cancer, colorectal cancer, rheumatoid arthritis, rare diseases, tuberculosis, and meningitis in children.
The rankings showed that some companies are exceeding baseline transparency requirements. Both GSK and Johnson & Johnson scored 100% on the transparency rankings. However, other companies and drugs have room for improvement.
Per drug, a median of 57% of trials were registered, 20% reported results in a registry, 56% were published, and 65% were publicly available (either published or reported results). The main finding, however, is that there is public information for only two-thirds of trials per drug. Many of the undisclosed trials were the smaller phase I trials, but not always. Almost half of all reviewed drugs had at least one undisclosed phase II or III trial.
The next step is to produce an annual ranking. If successful, each new drug, biologic, and device approved by the FDA (and possibly the European Medicines Agency and other regulators in the future) will be evaluated and ranked for its clinical trial transparency performance. This performance signal will hopefully help identify best practices, as well as where practices need to be improved and how to do it. We plan to add other ethics performance criteria and measurements in the future.
The GPS index should help provide a comprehensive understanding of what it means to be an ethical (or good) pharmaceutical company, signal how they are performing relative to those defined standards, and create a pathway for positive change and improved trustworthiness where needed.
Benefit of enhanced transparency for pharma
There are many ways in which increased corporate integrity and transparency pays off. These include: helping appeal to socially conscious investors; keeping activists and reactive regulations at bay; attracting consumers, partners, and talented employees; boosting the morale of existing employees; and protecting the value and safety proposition around an industry, institution, service or product.
People increasingly want to work for and invest in ethical companies, while studies by George Serafeim and colleagues from Harvard University show that ethics and transparency initiatives have the potential to increase a company’s value, if such initiatives are directed towards material operations. For example, it is important for the diamond industry to ensure its diamonds are ethically sourced and not financing civil wars. Similarly, it is important for fossil fuel companies and transportation industries to manage their environmental impact. These things are arguably less material for a financial firm or healthcare company, but for healthcare innovators, such as pharmaceutical companies, clinical trial transparency is material.
Some pharmaceutical companies recognise the importance of transparency. They are superseding baseline transparency requirements and voluntarily setting new and more robust disclosure standards. Johnson & Johnson and GSK were two of the early leaders in setting up new transparency platforms for the industry, and they scored the highest in our GPS. In subsequent years, it is hoped that more companies will join them and perform better on legal and ethics transparency standards in the next round of rankings. Working together, we can help advance patient and global public health priorities by improving the healthcare innovation sector’s ethics, transparency and trustworthiness.
Jennifer E Miller is an assistant professor in the department of Population health at the NYU School of Medicine, president of Bioethics International, and the creator of the Good Pharma Scorecard.
The author thanks Arthur Caplan, from the division of medical ethics in the department of population health at the NYU School of Medicine, for his comments on this article.
Citation: The Pharmaceutical Journal DOI: 10.1211/PJ.2016.20201274
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