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CRITical Thinking is a blog written by staff, directors, and friends of the Collaboration for Research Integrity and Transparency (CRIT), a joint program of Yale Law School, Yale School of Public Health, and Yale School of Medicine. CRIT's mission is to promote health by improving the integrity and transparency of biomedical and clinical research.

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What Would a Pro-Patient, Pro-Public Health Regulatory System Look Like?

February 13, 2018

In June 2017, Yale CRIT hosted an international conference titled "Ensuring Safety, Efficacy, and Access to Medical Products in the Age of Global Deregulation." The following blogpost is the twelfth installment of a blog series with commentaries from the conference participants. The views and opinions expressed in this blogpost are those of the author and do not necessarily reflect the position of Yale CRIT. For more blogposts related to this series, see here or click the tag "YaleCRIT17" below.

Notwithstanding a few real medical breakthroughs, the quality of our medical R&D in terms of addressing health needs of patients and generating better tools for doctors is poor despite much hype on medical advances and breakthrough therapies in the media.1 The majority of new medicines that come to market have no proven added therapeutic value compared to what we have already.2 Moreover, the quality of evidence about their safety and efficacy and the potential benefits and harms for patients is often too poor to establish their potential value in clinical practice, let alone to know whether they will deliver improved health outcomes for the patients.

While we get many new products we don't need, major public health needs remain unmet. These include effective cures for tuberculosis or AIDS, medicines that are designed for use in children, new medicines to treat the growing threat of drug-resistant infections or prepare us to tackle outbreaks of emerging infectious diseases, or effective treatments for many so-called neglected diseases that affect millions of people that happen to live in poor countries, not considered a lucrative enough market.

We need to redesign our medical R&D to deliver missing medicines for unmet public health needs, not just new or different medicines for already met medical needs. Medicines are public health tools, not market commodities for which the goal is to flood the market and increase consumption. If a medical need is adequately met with existing products (including second line treatments and other needs), it is a formidable waste of resources (financial and human) to develop other products to "compete" in the market or offer more choice, especially given that much of the research is paid for with public money, and that most of the products are ultimately purchased with public money, often at very high prices. Moreover, spending resources on medical research for new treatments with marginal benefit, crowds out efforts towards genuine medical breakthroughs, as is happening in the area of cancer.3

So what would a pro-patient, pro-public health regulatory system look like?

We need to define the role of regulatory agencies in pursuing public (health) interest, serving the dual mission of ensuring the quality and medical benefit of medicinal products and quality of the supporting evidence, as well as help directing R&D towards filling priority public health needs.

The first aspect requires that regulatory agencies operate in a way that is seen to be:

  1. Transparent (including on data, processes, decision making, experts and Conflict of Interest management);
  2. Independent (government funded, even if through fees or taxes levied from pharma companies, and with strong Conflict of Interest policies and practices in place, including about revolving doors);
  3. Accountable to the public based on their contribution to improving people's health by delivering missing medicines for unmet public health needs in a timely way, not just based on the numbers of new products approved in a given year.

The second aspect requires that, in addition to their traditional 'policing' role in overseeing the quality, safety and efficacy, regulatory agencies take a proactive role in re-shaping the R&D process from what is essentially a market and supply-driven process today to a needs-driven R&D process. Based on public health priority needs identified by ministries of health and WHO, regulators should identify how they can shape a conducive environment in terms of both processes and the evidence-base required for products providing genuine therapeutic benefit to be made available to patients. This includes setting target product or regimen profiles, outline which studies and evidence they need to see to establish added therapeutic value, and advise on adequate methodologies to be adopted.

Instead of being gate-keepers waiting at the end of the pipeline for what "the market" delivers to them for review, regulatory agencies could shape the R&D process at the front end by outlining what the public health systems need in terms of missing therapeutic advances, and set out the evidentiary requirements that therapy developers will have to live up to.

Finally, it is important to define a concept of "essential regulatory standards", which must be flexible and balance benefits and risks/harms within a specific context. This means taking into account the status of the health system including their ability for pharmacovigilance, the epidemiology and health status of the population, the availability and accessibility of alternative therapeutic options, available resources for access, etc. What is desirable or acceptable in a certain context is not necessarily the same everywhere, and all the time. This need for regulatory flexibility is a difficult concept to operationalize given the subjective nature of risk and requires very strong public health safeguards, but seems the only way to ensure that appropriate balance between ensuring timely access to therapeutic benefits while safeguarding people for unwarranted harms.

Dr. Els Torreele is the Executive Director of Médecins Sans Frontières' Access Campaign (Doctors Without Borders) and a Free University Brussels Fellow.

1  D Light & J Lexchin, "Pharmaceutical research and development: what do we get for all that money?" BMJ 2012; 344:e4348 http://www.pharmamyths.net/files/BMJ-Innova_ARTICLE_8-11-12.pdf ; E Torreele, "Only a radical overhaul can reclaim medicines in the public interest". PloS online 2015, http://blogs.plos.org/yoursay/2015/10/13/talking-about-drug-prices-acces... N Price, "Problems of Innovation-Deficient Pharmaceutical Manufacturing." NEJM 2013; http://www.nature.com/nrd/journal/v11/n3/abs/nrd3681.html; Public Health, Innovation and Intellectual Property Rights, report of the Commission on Intellectual Property Rights and Health, 2006, http://www.who.int/intellectualproperty/documents/thereport/ENPublicHeal...
2  Prescrire International 2015; 24(159): 107-110. "New drugs and indications in 2014. Some advances this year, but many drugs are poorly evaluated, too expensive, or more dangerous than useful", http://bit.ly/1Amp61o; Wemos position paper 2014, "Added Therapeutic Value: European citizens should get their money's worth", http://bit.ly/1tmYDa5;
3  Tito Fojo, Sham Mailankody, Andrew Lo. Unintended Consequences of Expensive Cancer Therapeutics-The Pursuit of Marginal Indications and a Me-Too Mentality That Stifles Innovation and Creativity. JAMA Otolaryngol Head Neck Surg. 2014;140(12):1225-1236 http://bit.ly/2xyaix6