Balancing Speed and Rigor in Drug Approval

We discussed drug development and approval in phase 1 of the Global Visioneering program. There is a consensus this needs to be sped up.

@iraspastor wrote that the modus operandi remains “study combinations after single interventions”, which isn’t fast enough.

@crointel and @darlenedamm suggested clinical trials could be digitized or simulated in silico.

Before we explore these as potential breakthroughs in phase 3, I’d like to get more clarity around why these – and other innovations – aren’t catching on? If it’s purely a matter of regulation or policy, then it might not be worth moving this forward in the Global Visioneering process. XPRIZE is not a lobby group. But if there are market failures or technological shortcomings or simply a lack of funding, that’s something we could potentially address with a prize competition.

@efoehr, @Lodder545, @Liz_Parrish, @TSeoh, and @sadiew – as pharma experts, I hope you can shed some light on this. Specifically on how to balance speed and rigor in the drug approval process, which seems to me the core of the challenge. (But please correct me if you think that’s wrong!)

@Shabbir, @barati, @Kwenz, and @Youdufkhan79 – I’d also like to ask you to weigh in from a global perspective.

If you haven’t yet, please read About Global Visioneering for a quick description of the program we launched in April.

Thanks, @NickOttens. Just to clarify, the digital clinical trials work is already happening in oncology (a group called XCures is leading one effort and I believe there are other efforts.) In that case, it is just still early stage and takes time and funding to bring people on board. There is no regulatory block in that case. Rather it should speed up regulation as regulators will have access to more data.

In that case, do you think this is something that could still need an XPRIZE? Sometimes a prize competition can still be very useful to speed up wide application of a novel technology, but we want to avoid being overtaken by the market or technology.

Hi @NickOttens - I am new to the group, and still learning how things work, but you would be interested in talking to the team at XCures? I think they would be in a much better position to answer this question (and discuss the topic more broadly.) I can make an intro if you like. Thanks! Darlene

Thank you for the offer, @darlenedamm! Let me check with @Roey if that could be of interest to us? It might be a little early, if we’re not sure yet if this idea will be taken forward in the Global Visioneering process.

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Hi @DidierC, @taboma, @AlexandraW, @danbelsky, @bjcooper, @Terenceericson - What are your thoughts on balancing speed and rigor in the drug approval process? What challenges needs to be addressed?

Thank you for asking. Some of my ideas (not complete)

  1. If one drug is allowed in one country/region with a good health system (European Union, UK, USA), the authorization should be valid in the other countries.

  2. In the process of authorization, there should be compulsory deadlines.

  3. During the process of approbation, the importance of potential lost of Healthy Life Years because of the slowness of approbations must be explicitly indicated (for example X Years of Healthy Life Expectancy for each month of daly.

I suppose many of you know about the book “Daath by regulation” by Mary J. Ruart https://www.amazon.com/Death-Regulation-Robbed-Golden-Reclaim/dp/0963233610 . I do not agree with everything, but is is certainly a book to read to hav ideas about this question (especially in the US)

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Thanks @DidierC for sharing these insights. I was wondering how would you calculate potential lost of Healthy Life Years because of the slowness of approbations. I agree with the other two points.

I further want to understand if the point 1 could be applicable in developing countries; if not what process need to be followed to increase the pace of drug approval there.

Thanks Shashi for the answer.

Concerning the potential lost of Healthy Life years, you could calculate the number of people concerned (it can be the whole aging population sometimes), the number of years of gain of life expectancy hoped/expected (for example, let’s say systematic use of a small dose of metformin from the age of 65 years: 18 months). You take 2/3 or 75 % for the healthy part. And then you should be able to calculate. Not easy for me, I admit, but not complicated for a statistician, I think).

Concerning point 1, it could be applicable to some extent for developing countries. Once agreed in UK, US, Europe, also admitted in these countries. I think, it is already that way in some countries. For the other way around, it will be more difficult since the studies need a good working health network, are expensive, … But some form of cooperation could be created.

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Hi @supratik12 - As you have lead the Deep Drug team, you might have inputs to share on this topic of balancing speed and rigor in drug approval. Thanks.