Personalized Medicine-Expensive & Pharma Can't Keep Up-Charities Tackle Rare Diseases

The Economist March 14th, 2020 pp11-13 Technology Quarterly|Personalised medicine

The pharmaceutical industry “Kill or cure?” “New drugs are costly and unmet need is growing. Pharma needs new ways of doing things”

With personalized medicine comes complexity especially in developing cancer drugs. This because so many different genes can impact cancer growth. “As drugs, targeted against those different mutations have multiplied, so have the options for oncologists to combine them to fit their patient’s needs”. This type of customization is also relevant to diabetes, Parkinson’s disease and no doubt other diseases.

Personalized Medicine is also impacting the discovery and treatment options for rare diseases. As of today, 7,000 rare diseases have been identified in America and inline with being rare there are fewer than 200,000 potential patients in aggregate. Because each rare disease has a unique molecular mechanism, a drug trial successful for one subtype of disease may not work for another. At some point, with continuing discovery of rare disease, there may be more diseases than treatments. Currently, 90% of rare diseases have no approved treatment.

Given the market size, personalized treatments for rare diseases are expensive. In addition, the inefficiency of biopharma discovery is pushing up costs. Today, “…the number of drugs developed for a given amount of R&D spending has fallen inexorably, even as the amount of biological research skyrocketed”. Part of the failure rests with the biopharma industry as a whole and with individual company management. They have failed, in some cases, by not understanding and targeting the underlying disease mechanisms and in other cases they have pursued the wrong projects. Now because gene editing techniques allow a step-by-step process or a base-by-base approach, discovery should be more efficient. Following this approach more drugs should be developed at lower cost.

In business you can make or buy. On the buying side of drug development, Novartis paid $8.7bn to buy a small firm, Avexis, largely to acquire “Zolgensma, a gene therapy for spinal muscular atrophy (SMA). At $2,100,000 per patient, Zolgensma will be “the most expensive drug ever brought to market”. Drug companies claim such treatments are economical “over the lifetime of the patient”. Most affected children typically die by age four but with successful treatment they may have a normal lifespan. So far 200 patients have been treated to reverse this agonizing and deadly disease. Note there are 6,000 American children with SMA.

Given these examples, we correctly get the impression that making drugs for treating rare disease may not be profitable for biopharma. Consequently, charities are stepping in to fund rare disease treatments using anti-sense-oligonucleotides or ASOs. ASOs target the exact molecular defect and act upstream of protein synthesis by interfering with DNA transcription to RNA or RNA translation to protein. When lives are at risk, individuals with rare disease and parents of children with rare disease and are highly motivated to find treatments. Charities are not bound by profit.

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